Muscular Dystrophy, DuchennePhase 3NCT03179631
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
A Phase 3 study testing Ataluren in people with Muscular Dystrophy, Duchenne and Muscular Dystrophies, run by PTC Therapeutics at 64 sites in 18 countries.
Trial summary
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy. Read more about this study
- Who is it for?
- Ages 5 years and older, men only
- Conditions the study is for
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic
- What is being tested?
- Ataluren
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 360 participants
- Where
- 64 sitesin 18 countries
- Study dates
- 6 Jul 2017to 25 Jul 2023
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male sex
- Age ≥5 years
- Phenotypic evidence of Duchenne Muscular Dystrophy
- Nonsense point mutation in the dystrophin gene
- Use of systemic corticosteroids (prednisone/prednisolone or deflazacort)for a minimum of 12 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen for a minimum of 3 months immediately prior to start of study treatment
- 6MWD ≥150 meters
- Ability to perform timed function tests within 30 seconds
- Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.
Who cannot join
- Any change in prophylaxis treatment for cardiomyopathy within 1 month prior to start of study treatment.
- Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy.
- Prior or ongoing therapy with ataluren.
- Known hypersensitivity to any of the ingredients or excipients of the study drug
- Exposure to another investigational drug within 6 months prior to start of study treatment, or ongoing participation in any interventional clinical trial.
- History of major surgical procedure within 12 weeks prior to start of study treatment, or expectation of major surgical procedure during the 72-week placebo-controlled treatment period.
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- Requirement for daytime ventilator assistance or any use of invasive mechanical ventilation via tracheostomy.
- Uncontrolled clinical symptoms and signs of congestive heart failure
- Elevated serum creatinine or cystatin C at screening.
Trial locations and contacts
64 sites in 18 countries.
64 sites in 18 countries
United States22 sites
University of Michigan - CS Mott Children's Hospital
Ann Arbor, Michigan, 48109
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Children's Hospital of Michigan
Detroit, Michigan, 48201
Cook Childrens Medical Center
Fort Worth, Texas, 76104
Northwest Florida Clinical Research Group, LLC
Gulf Breeze, Florida, 32561
Texas Children's Hospital
Houston, Texas, 77030
Indiana University Health - Riley Child Neurology
Indianapolis, Indiana, 46202
University of Kansas Medical Center
Kansas City, Kansas, 66160
Children's Hospital of Los Angeles
Los Angeles, California, 90027
University of Minnesota
Minneapolis, Minnesota, 55455
Columbia University College of Physicians & Surgeons
New York, New York, 10032
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507
University of California, San Francisco (UCSF) - Benioff Children's Hospital - Oakland
Oakland, California, 94143
Stanford University Medical Center
Palo Alto, California, 94305
Phoenix Childrens Hospital
Phoenix, Arizona, 85016
University of Pittsburgh School of Medicine
Pittsburgh, Pennsylvania, 15224
Shriners Hospital for Children
Portland, Oregon, 97239
University of California (UC) Davis Medical Center
Sacramento, California, 95817
University Of Utah
Salt Lake City, Utah, 84112
University of Texas Health Science Center at San Antonio
San Antonio, Texas, 78229-3900
Loma Linda University Children's Hospital
San Bernardino, California, 92408
Seattle Children's Hospital
Seattle, Washington, 98105
India9 sites
Panchshil Hospital
Ahmedabad, Gujarat, 380005
National Institute of Mental Health and Neurosciences
Bengaluru, Karnataka, 560029
Postgraduate Institute of Medical Education and Research
Chandigarh, 160012
Apollo Children's Hospital Chennai
Chennai, Tamil Nadu, 600006
Nizam's Institute of Medical Sciences (NIMS)
Hyderabad, Telangana, 500082
Apollo Gleneagles Hospital
Kolkata, West Bengal, 700054
P.D. Hinduja Hospital
Māhīm, Maharashtra, 400016
All India Institute of Medical Sciences
New Delhi, 110029
Christian Medical College Hospital Vellore
Vellore, Tamil Nadu, 632004
China5 sites
General Hospital of Chinese Armed Police Forces
Beijing, 100039
The First Affiliated Hospital of Fujian Medical University
Fuzhou, 350005
Xiangya Hospital Central South University
Hunan, 410008
Children's Hospital of Fudan University
Shanghai, 200032
Shenzhen Children's Hospital
Shenzhen, 518038
Australia4 sites
Perth Children's Hospital
Nedlands, Western Australia, 6009
The Royal Childrens Hospital
Parkville, Victoria, 3052
Queensland Children's Hospital
South Brisbane, Q4101
The Childrens Hospital at Westmead
Westmead, New South Wales, 2145
Brazil3 sites
Hospital de Clínicas da Universidade Federal de Minas Gerais
Belo Horizonte, 30130-100
Universidade Federal do Rio de Janeiro
Rio de Janeiro, 21.941-912
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo - FMUSP
São Paulo, 05403-000
Mexico3 sites
Hospital Angeles Chihuahua
Chihuahua City, 31217
Instituto Nacional de Pediatría
Mexico City, 04530
Instituto Nacional de Rehabilitacion
Tlalpan, 14389
South Korea3 sites
Samsung Medical Center
Seoul, 06351
Seoul National University Hospital
Seoul, 3080
Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam-do, 50612
Canada2 sites
Childrens Hospital London Health Sciences Centre
London, Ontario, N6A5W9
Childrens Hospital of Eastern Ontario
Ottawa, Ontario, K1H8L1
Malaysia2 sites
Hospital Tunku Azizah Kuala Lumpur
Kuala Lumpur, 50586
University Malaya Medical Centre (UMMC)
Pantai, 59100
Russia2 sites
Russian National Research Medical University n.a. N.I.Pirogov, structural branch - Research Clinical Institute of Pediatrics n.a. Academician Yu. E. Veltishchev
Moscow, 125412
"Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre
Saint Petersburg, 194100
Taiwan2 sites
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 80756
National Taiwan University Hospital
Taipei, 10002
Poland1 site
Samodzielny Publiczny Centralny Szpital Kliniczny w Warszawie
Warsaw, 02-097
Puerto Rico1 site
University of Puerto Rico - School of Medicine
San Juan, 00936-5067
Turkey (Türkiye)1 site
Istanbul University- Instanbul Medical Faculty
Istanbul, 34093
About this study
Official title: A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
Read the detailed description
This study is a randomized, double-blind, placebo-controlled, 72-week study, followed by a 72-week open-label period. The purpose is to characterize the long-term effects of ataluren-mediated dystrophin restoration on disease progression. Participants will be randomized in a 1:1 ratio to ataluren or placebo. Participants will receive blinded study drug three times daily (TID) at morning, midday, and evening for 72 weeks, after which all participants will receive open-label ataluren for an additional 72 weeks (144 weeks in total). Study assessments will be performed at clinic visits every 12 weeks during the double-blind period and every 24 weeks during the open-label period.
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases
- Being tested
- Ataluren, PLACEBO
- Keywords
- Duchenne Muscular Dystrophy, Dystrophinopathy, Nonsense Mutation, Premature Stop Codon, Becker Muscular Dystrophy, DMD/BMD, PTC124, Ataluren
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AtalurenDrug
Also called PTC124
10, 20 mg/kg
PLACEBODrug
Also called Matching Placebo
10, 20 mg/kg
| Study group | Role | What they receive |
|---|---|---|
| Ataluren | Experimental | Participants will receive ataluren oral suspension 10 milligrams (mg)/kilogram (kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for 72 weeks in DB treatment period and for an additional 72 weeks in open-label treatment period. Receives: Drug: Ataluren |
| Placebo | Placebo comparator | Participants will receive placebo matched to ataluren oral suspension for 72 weeks in DB treatment period. After completion of DB treatment period, participants will receive ataluren oral suspension 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for 72 weeks in open-label treatment period. Receives: Drug: PLACEBO |
What is being measured
The primary outcome is the main question the study is designed to answer.
DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population
The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Participants with confirmed loss of ambulation at a particular visit were assigned a 6MWD result of 0. Baseline was defined as the maximum measurement of valid Day 1 and Day 2 6MWD values. Least square (LS) mean and standard error (SE) was calculated using the mixed-model repeated measures (MMRM).
Measured: Baseline, Week 72
DB Period: Change From Baseline in 6MWD at Week 72 - Intent-to-Treat (ITT) Population
The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Participants with confirmed loss of ambulation at a particular visit were assigned a 6MWD result of 0. Baseline was defined as the maximum measurement of valid Day 1 and Day 2 6MWD values. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 72
DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - mITT Population
The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Participants with confirmed loss of ambulation at a particular visit were assigned a 6MWD result of 0. Baseline was defined as the maximum measurement of valid Day 1 and Day 2 6MWD values. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 72
DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - ITT Population
The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Participants with confirmed loss of ambulation at a particular visit were assigned a 6MWD result of 0. Baseline was defined as the maximum measurement of valid Day 1 and Day 2 6MWD values. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 72
Secondary outcomes (35)
DB Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 72 - mITT Population
During the test for walking/running 10 meters, the method of walk/run used by the participant was categorized as follows: 1. Unable to walk independently; 2. Unable to walk independently but can walk with support from a person or with assistive device (full leg calipers [knee-ankle-foot orthoses] or walker); 3. Highly adapted gait, wide-based lordotic gait, cannot increase walking speed; 4. Moderately adapted gait, can pick up speed but cannot run; 5. Able to pick up speed but runs with a double stance phase (that is, cannot achieve both feet off the ground); 6. Runs and gets both feet off the ground (with no double stance phase). Participants who could not perform a timed function test (TFT) within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 72
DB Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 72 - ITT Population
During the test for walking/running 10 meters, the method of walk/run used by the participant was categorized as follows: 1. Unable to walk independently; 2. Unable to walk independently but can walk with support from a person or with assistive device (full leg calipers [knee-ankle-foot orthoses ] or walker); 3. Highly adapted gait, wide-based lordotic gait, cannot increase walking speed; 4. Moderately adapted gait, can pick up speed but cannot run; 5. Able to pick up speed but runs with a double stance phase (that is, cannot achieve both feet off the ground); 6. Runs and gets both feet off the ground (with no double stance phase). Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 72
DB Period: Change From Baseline in Time to Climb 4 Stairs at Week 72 - mITT Population
During the test for stair-climbing, the method of climbing used by the participant was categorized as follows: 1. Unable to up climb 4 standard stairs; 2. Climbs 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Climbs 4 standard stairs "marking time", using one arm on one handrail; 4. Climbs 4 standard stairs "marking time", not needing handrail; 5. Climbs 4 standard stairs alternating feet, needs handrail for support; 6. Climbs 4 standard stairs alternating feet, not needing handrail support. Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 72
DB Period: Change From Baseline in Time to Climb 4 Stairs at Week 72 - ITT Population
During the test for stair-climbing, the method of climbing used by the participant was categorized as follows: 1. Unable to up climb 4 standard stairs; 2. Climbs 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Climbs 4 standard stairs "marking time", using one arm on one handrail; 4. Climbs 4 standard stairs "marking time", not needing handrail; 5. Climbs 4 standard stairs alternating feet, needs handrail for support; 6. Climbs 4 standard stairs alternating feet, not needing handrail support. Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 72
DB Period: Change From Baseline in Time to Descend 4 Stairs at Week 72 - mITT Population
During the test for stair-descending, the method of descending used by the participant was categorized as follows: 1. Unable to descend 4 standard stairs; 2. Descends 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Descends 4 standard stairs "marking time", using one arm on one handrail; 4. Descends 4 standard stairs "marking time", not needing handrail; 5. Descends 4 standard stairs alternating feet, needs handrail for support; 6. Descends 4 standard stairs alternating feet, not needing handrail support. Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 72
DB Period: Change From Baseline in Time to Descend 4 Stairs at Week 72 - ITT Population
During the test for stair-descending, the method of descending used by the participant was categorized as follows: 1. Unable to descend 4 standard stairs; 2. Descends 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Descends 4 standard stairs "marking time", using one arm on one handrail; 4. Descends 4 standard stairs "marking time", not needing handrail; 5. Descends 4 standard stairs alternating feet, needs handrail for support; 6. Descends 4 standard stairs alternating feet, not needing handrail support. Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 72
DB Period: Composite of Average Change From Baseline in TFTs at Week 72 - mITT Population
The composite TFT was defined as the average in times to run/walk 10 meters, climb 4 stairs, and descend 4 stairs. Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement (average in times to run/walk 10 meters, climb 4 stairs, and descend 4 stairs) on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 72
DB Period: Composite of Average Change From Baseline in TFTs at Week 72 - ITT Population
The composite TFT was defined as the average in times to run/walk 10 meters, climb 4 stairs, and descend 4 stairs. Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement (average in times to run/walk 10 meters, climb 4 stairs, and descend 4 stairs) on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 72
DB Period: Composite of Average Rate of Change From Baseline in TFTs at Week 72 - mITT Population
The composite TFT was defined as the average in times to run/walk 10 meters, climb 4 stairs, and descend 4 stairs. Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement (average in times to run/walk 10 meters, climb 4 stairs, and descend 4 stairs) on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 72
DB Period: Composite of Average Rate of Change From Baseline in TFTs at Week 72 - ITT Population
The composite TFT was defined as the average in times to run/walk 10 meters, climb 4 stairs, and descend 4 stairs. Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement (average in times to run/walk 10 meters, climb 4 stairs, and descend 4 stairs) on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 72
DB Period: Time to Persistent 10% Worsening in 6MWD at Week 72 - mITT Population
The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Time to 10% persistent worsening in 6MWD was defined as the last time that 6MWD was not 10% worse compared with baseline. Participants who did not have 10% 6MWD worsening were censored at the time of the last 6-minute walk test during the DB period.
Measured: Baseline to Week 72
DB Period: Time to Persistent 10% Worsening in 6MWD - ITT Population
6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where participant is instructed to walk as far as possible, back and forth around two cones, with permission to slow down, rest, or stop if needed. Ambulation was assessed via 6MWD test following standardized procedures. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during 6MWD test. Time to 10% persistent worsening in 6MWD was defined as the last time that 6MWD was not 10% worse compared with baseline. Participants who did not have 10% 6MWD worsening were censored at the time of last 6-minute walk test during DB period. Due to COVID, there were many participants who participated in the protocol-defined Week 72 visit late. Due to this reason, the calculated median for the time to event for the Ataluren arm in the DB period were greater than Week 72.
Measured: Baseline to approximately Week 72 (Due to COVID, many participants attended the protocol-defined Week 72 visit late)
DB Period: Change From Baseline in North Start Ambulatory Assessment (NSAA) Total Score at Week 72 - mITT Population
The NSAA total score in the original scale is the sum of scores from 16 activities (excluding head lift). Each activity was scored as 0 (activity couldn't be performed), 1 (modified method, achieved goal without assistance), or 2 (normal, achieved goal without assistance). The total score ranges from 0 to 32, where higher scores indicate better functioning. If fewer than 13 of the 16 activities were performed, the total score was considered missing. If from 13 to 16 activities were performed, the total score was standardized by (observed total score / number of non-missing activities) x 16. If an activity could not be performed due to disease progression, a score of 0 was assigned. Baseline was defined as the last observed measurement on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 72
DB Period: Change From Baseline in NSAA Total Score at Week 72 - ITT Population
The NSAA total score in the original scale is the sum of scores from 16 activities (excluding head lift). Each activity was scored as 0 (activity couldn't be performed), 1 (modified method, achieved goal without assistance), or 2 (normal, achieved goal without assistance). The total score ranges from 0 to 32, where higher scores indicate better functioning. If fewer than 13 of the 16 activities were performed, the total score was considered missing. If from 13 to 16 activities were performed, the total score was standardized by (observed total score / number of non-missing activities) x 16. If an activity could not be performed due to disease progression, a score of 0 was assigned. Baseline was defined as the last observed measurement on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 72
DB Period: Time to Loss of Ambulation - mITT Population
Time to loss of ambulation was defined as persistent inability to perform the 10-meter run/walk test within 30 seconds at any post-baseline visit and for all remaining visits. Due to COVID, there were many participants who participated in the protocol-defined Week 72 visit late. Due to this reason, the calculated median and 95% CI for the time to event for the Ataluren arm in the DB period were greater than Week 72.
Measured: Baseline up to approximately Week 72 (Due to COVID, many participants attended the protocol-defined Week 72 visit late)
DB Period: Time to Loss of Ambulation Over 72 Weeks - ITT Population
Time to loss of ambulation was defined as persistent inability to perform the 10-meter run/walk test within 30 seconds at any post-baseline visit and for all remaining visits.
Measured: Baseline up to Week 72
DB Period: Time to Loss of Stair-Climbing - mITT Population
Time to loss of stair-climbing was defined as persistent inability to perform the 4-stair climb test within 30 seconds at any post-baseline visit and for all remaining visits. Due to COVID, there were many participants who participated in the protocol-defined Week 72 visit late. Due to this reason, the calculated median and 95% CI for the time to event for the Ataluren arm in the DB period were greater than Week 72.
Measured: Baseline up to approximately Week 72 (Due to COVID, many participants attended the protocol-defined Week 72 visit late)
DB Period: Time to Loss of Stair-Climbing - ITT Population
Time to loss of stair-climbing was defined as persistent inability to perform the 4-stair climb test within 30 seconds at any post-baseline visit and for all remaining visits. Due to COVID, there were many participants who participated in the protocol-defined Week 72 visit late. Due to this reason, the calculated median and 95% CI for the time to event for the Ataluren arm in the DB period were greater than Week 72.
Measured: Baseline up to approximately Week 72 (Due to COVID, many participants attended the protocol-defined Week 72 visit late)
DB Period: Time to Loss of Stair-Descending - mITT Population
Time to loss of stair-descending was defined as persistent inability to perform the 4-stair descend test within 30 seconds at any post-baseline visit and for all remaining visits. Due to COVID, there were many participants who participated in the protocol-defined Week 72 visit late. Due to this reason, the calculated median and 95% CI for the time to event for the Ataluren arm in the DB period were greater than Week 72.
Measured: Baseline up to approximately Week 72 (Due to COVID, many participants attended the protocol-defined Week 72 visit late)
DB Period: Time to Loss of Stair-Descending - ITT Population
Time to loss of stair-descending was defined as persistent inability to perform the 4-stair descend test within 30 seconds at any post-baseline visit and for all remaining visits. Due to COVID, there were many participants who participated in the protocol-defined Week 72 visit late. Due to this reason, the calculated median and 95% CI for the time to event for the Ataluren arm in the DB period were greater than Week 72.
Measured: Baseline up to approximately Week 72 (Due to COVID, many participants attended the protocol-defined Week 72 visit late)
DB Period: Number of Participants With Function Loss of NSAA Items at Week 72 - mITT Population
Function loss was defined as a drop of score from 1 or 2 at baseline to a score of 0 at the specified post-baseline of NSAA items. The missing assessments at post baseline visits were imputed using last observation carried forward (LOCF).
Measured: Week 72
DB Period: Number of Participants With Function Loss of NSAA Items at Week 72 - ITT Population
Function loss was defined as a drop of score from 1 or 2 at baseline to a score of 0 at the specified post-baseline of NSAA items. The missing assessments at post baseline visits were imputed using LOCF.
Measured: Week 72
DB Period: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A TEAE was defined as an AE that occurred or worsened on or after the first dose of study drug and up to 4 weeks after the last dose of double-blind study drug and prior to the first dose of open-label treatment. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Measured: Baseline up to Week 76
Overall Treatment Period: Change From Baseline in 6MWD at Week 144
The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Participants with confirmed loss of ambulation at a particular visit were assigned a 6MWD result of 0. Baseline was defined as the maximum measurement of valid Day 1 and Day 2 6MWD values. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 144
Overall Treatment Period: Composite of Average Change From Baseline in TFTs at Week 144
The composite TFT was defined as the average in times to run/walk 10 meters, climb 4 stairs, and descend 4 stairs. Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement (average in times to run/walk 10 meters, climb 4 stairs, and descend 4 stairs) on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 144
Overall Treatment Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 144
During the test for walking/running 10 meters, the method of walk/run used by the participant was categorized as follows: 1. Unable to walk independently; 2. Unable to walk independently but can walk with support from a person or with assistive device (full leg calipers [knee-ankle-foot orthoses ] or walker); 3. Highly adapted gait, wide-based lordotic gait, cannot increase walking speed; 4. Moderately adapted gait, can pick up speed but cannot run; 5. Able to pick up speed but runs with a double stance phase (that is, cannot achieve both feet off the ground); 6. Runs and gets both feet off the ground (with no double stance phase). Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 144
Overall Treatment Period: Change From Baseline in Time to Climb 4 Stairs at Week 144
During the test for stair-climbing, the method of climbing used by the participant was categorized as follows: 1. Unable to up climb 4 standard stairs; 2. Climbs 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Climbs 4 standard stairs "marking time", using one arm on one handrail; 4. Climbs 4 standard stairs "marking time", not needing handrail; 5. Climbs 4 standard stairs alternating feet, needs handrail for support; 6. Climbs 4 standard stairs alternating feet, not needing handrail support. Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 144
Overall Treatment Period: Change From Baseline in Time to Descend 4 Stairs at Week 144
During the test for stair-descending, the method of descending used by the participant was categorized as follows: 1. Unable to descend 4 standard stairs; 2. Descends 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Descends 4 standard stairs "marking time", using one arm on one handrail; 4. Descends 4 standard stairs "marking time", not needing handrail; 5. Descends 4 standard stairs alternating feet, needs handrail for support; 6. Descends 4 standard stairs alternating feet, not needing handrail support. Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM.
Measured: Baseline, Week 144
Overall Treatment Period: Change From Baseline in NSAA Total Score at Week 144
The NSAA total score in the original scale is the sum of scores from 16 activities (excluding head lift). Each activity was scored as 0 (activity couldn't be performed), 1 (modified method, achieved goal without assistance), or 2 (normal, achieved goal without assistance). The total score ranges from 0 to 32, where higher scores indicate better functioning. If fewer than 13 of the 16 activities were performed, the total score was considered missing. If from 13 to 16 activities were performed, the total score was standardized by (observed total score / number of non-missing activities) x 16. If an activity could not be performed due to disease progression, a score of 0 was assigned. Baseline was defined as the last observed measurement on or prior to the first dose of study drug. LS mean and SE was calculated using analysis of covariation (ANCOVA) with multiple imputation.
Measured: Baseline, Week 144
Overall Treatment Period: Time to Loss of Ambulation Over 144 Weeks
Time to loss of ambulation was defined as persistent inability to perform the 10-meter run/walk test within 30 seconds at any post-baseline visit and for all remaining visits.
Measured: Baseline up to Week 144
Overall Treatment Period: Time to Loss of Stair-Climbing Over 144 Weeks
Time to loss of stair-climbing was defined as persistent inability to perform the 4-stair climb test within 30 seconds at any post-baseline visit and for all remaining visits.
Measured: Baseline up to Week 144
Overall Treatment Period: Time to Loss of Stair- Descending Over 144 Weeks
Time to loss of stair-descending was defined as persistent inability to perform the 4-stair descend test within 30 seconds at any post-baseline visit and for all remaining visits.
Measured: Baseline up to Week 144
Overall Treatment Period: Number of Participants With Function Loss of NSAA Items at Week 144
Function loss was defined as a drop of score from 1 or 2 at baseline to a score of 0 at the specified post-baseline in NSAA items. The missing assessments at post baseline visits were imputed using LOCF.
Measured: Week 144
Overall Treatment Period: Number of Participants With TEAEs
An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A TEAE was defined as an AE that occurs or worsens on or after the first dose of ataluren (regardless of double-blind or open-label) and up to 4 weeks after the last dose of ataluren. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Measured: Baseline up to Week 148
OL Period: Plasma Pharmacokinetic (PK) Concentration of Ataluren
Measured: Predose and 2 hours postdose at Week 144
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jun 2017
- Study start6 Jul 2017
- Primary completion5 Mar 2022
- Study completion25 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Muscular Dystrophy, DuchenneStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This study describes the randomized, double-blind, placebo-controlled, 72-week study and its 72-week open-label extension
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 360 participants (actual)
Sponsor and investigators
- Lead sponsor
- PTC Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Vinay Penematsa, MD, Study director, PTC Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03179631
- Sponsor's protocol ID
- PTC124-GD-041-DMD
- Status last verified
- Feb 2026
- First submitted
- 1 Jun 2017
- Last updated
- 10 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Wu S, Vlodavets D, de Queiroz Campos Araujo AP, Castle J. Ataluren for the treatment of people living with nonsense mutation Duchenne muscular dystrophy: a plain language summary of Study 041. J Comp Eff Res. 2026 Apr;15(4):e250184. doi: 10.57264/cer-2025-0184. Epub 2026 Mar 25.
PubMed 41879641
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension
- ClinicalTrials.gov ID
- NCT03179631
- Acronym / sponsor ID
- PTC124-GD-041-DMD
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases
- Keywords
- Duchenne Muscular Dystrophy, Dystrophinopathy, Nonsense Mutation, Premature Stop Codon, Becker Muscular Dystrophy, DMD/BMD, PTC124, Ataluren
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 360 participants (actual)
- Lead sponsor
- PTC TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 6 Jul 2017 (actual)
- Primary completion
- 5 Mar 2022 (actual)
- Study completion
- 25 Jul 2023 (actual)
- First posted
- 7 Jun 2017 (actual)
- Results
- Posted 10 Mar 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
Detailed description
This study is a randomized, double-blind, placebo-controlled, 72-week study, followed by a 72-week open-label period. The purpose is to characterize the long-term effects of ataluren-mediated dystrophin restoration on disease progression. Participants will be randomized in a 1:1 ratio to ataluren or placebo. Participants will receive blinded study drug three times daily (TID) at morning, midday, and evening for 72 weeks, after which all participants will receive open-label ataluren for an additional 72 weeks (144 weeks in total). Study assessments will be performed at clinic visits every 12 weeks during the double-blind period and every 24 weeks during the open-label period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- This study describes the randomized, double-blind, placebo-controlled, 72-week study and its 72-week open-label extension
| Arm | Type | Receives |
|---|---|---|
| Ataluren | Experimental | Drug: AtalurenParticipants will receive ataluren oral suspension 10 milligrams (mg)/kilogram (kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for 72 weeks in DB treatment period and for an additional 72 weeks in open-label treatment period. |
| Placebo | Placebo comparator | Drug: PLACEBOParticipants will receive placebo matched to ataluren oral suspension for 72 weeks in DB treatment period. After completion of DB treatment period, participants will receive ataluren oral suspension 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for 72 weeks in open-label treatment period. |
Who may qualify
- Ages
- 5 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Male sex
- Age ≥5 years
- Phenotypic evidence of Duchenne Muscular Dystrophy
- Nonsense point mutation in the dystrophin gene
- Use of systemic corticosteroids (prednisone/prednisolone or deflazacort)for a minimum of 12 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen for a minimum of 3 months immediately prior to start of study treatment
Key exclusion criteria (9 listed)
- Any change in prophylaxis treatment for cardiomyopathy within 1 month prior to start of study treatment.
- Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy.
- Prior or ongoing therapy with ataluren.
- Known hypersensitivity to any of the ingredients or excipients of the study drug
- Exposure to another investigational drug within 6 months prior to start of study treatment, or ongoing participation in any interventional clinical trial.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AtalurenAlso: PTC12410, 20 mg/kg | Drug | Ataluren |
| PLACEBOAlso: Matching Placebo10, 20 mg/kg | Drug | Placebo |
Primary outcome
DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population
Time frame: Baseline, Week 72
The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Participants with confirmed loss of ambulation at a particular visit were assigned a 6MWD result of 0. Baseline was defined as the maximum measurement of valid Day 1 and Day 2 6MWD values. Least square (LS) mean and standard error (SE) was calculated using the mixed-model repeated measures (MMRM).
DB Period: Change From Baseline in 6MWD at Week 72 - Intent-to-Treat (ITT) Population
Time frame: Baseline, Week 72
The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Participants with confirmed loss of ambulation at a particular visit were assigned a 6MWD result of 0. Baseline was defined as the maximum measurement of valid Day 1 and Day 2 6MWD values. LS mean and SE was calculated using the MMRM.
DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - mITT Population
Time frame: Baseline, Week 72
The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Participants with confirmed loss of ambulation at a particular visit were assigned a 6MWD result of 0. Baseline was defined as the maximum measurement of valid Day 1 and Day 2 6MWD values. LS mean and SE was calculated using the MMRM.
DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - ITT Population
Time frame: Baseline, Week 72
The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Participants with confirmed loss of ambulation at a particular visit were assigned a 6MWD result of 0. Baseline was defined as the maximum measurement of valid Day 1 and Day 2 6MWD values. LS mean and SE was calculated using the MMRM.
35 secondary outcomes
DB Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 72 - mITT Population
Time frame: Baseline, Week 72
DB Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 72 - ITT Population
Time frame: Baseline, Week 72
DB Period: Change From Baseline in Time to Climb 4 Stairs at Week 72 - mITT Population
Time frame: Baseline, Week 72
DB Period: Change From Baseline in Time to Climb 4 Stairs at Week 72 - ITT Population
Time frame: Baseline, Week 72
DB Period: Change From Baseline in Time to Descend 4 Stairs at Week 72 - mITT Population
Time frame: Baseline, Week 72
DB Period: Change From Baseline in Time to Descend 4 Stairs at Week 72 - ITT Population
Time frame: Baseline, Week 72
DB Period: Composite of Average Change From Baseline in TFTs at Week 72 - mITT Population
Time frame: Baseline, Week 72
DB Period: Composite of Average Change From Baseline in TFTs at Week 72 - ITT Population
Time frame: Baseline, Week 72
DB Period: Composite of Average Rate of Change From Baseline in TFTs at Week 72 - mITT Population
Time frame: Baseline, Week 72
DB Period: Composite of Average Rate of Change From Baseline in TFTs at Week 72 - ITT Population
Time frame: Baseline, Week 72
DB Period: Time to Persistent 10% Worsening in 6MWD at Week 72 - mITT Population
Time frame: Baseline to Week 72
DB Period: Time to Persistent 10% Worsening in 6MWD - ITT Population
Time frame: Baseline to approximately Week 72 (Due to COVID, many participants attended the protocol-defined Week 72 visit late)
DB Period: Change From Baseline in North Start Ambulatory Assessment (NSAA) Total Score at Week 72 - mITT Population
Time frame: Baseline, Week 72
DB Period: Change From Baseline in NSAA Total Score at Week 72 - ITT Population
Time frame: Baseline, Week 72
DB Period: Time to Loss of Ambulation - mITT Population
Time frame: Baseline up to approximately Week 72 (Due to COVID, many participants attended the protocol-defined Week 72 visit late)
DB Period: Time to Loss of Ambulation Over 72 Weeks - ITT Population
Time frame: Baseline up to Week 72
DB Period: Time to Loss of Stair-Climbing - mITT Population
Time frame: Baseline up to approximately Week 72 (Due to COVID, many participants attended the protocol-defined Week 72 visit late)
DB Period: Time to Loss of Stair-Climbing - ITT Population
Time frame: Baseline up to approximately Week 72 (Due to COVID, many participants attended the protocol-defined Week 72 visit late)
DB Period: Time to Loss of Stair-Descending - mITT Population
Time frame: Baseline up to approximately Week 72 (Due to COVID, many participants attended the protocol-defined Week 72 visit late)
DB Period: Time to Loss of Stair-Descending - ITT Population
Time frame: Baseline up to approximately Week 72 (Due to COVID, many participants attended the protocol-defined Week 72 visit late)
DB Period: Number of Participants With Function Loss of NSAA Items at Week 72 - mITT Population
Time frame: Week 72
DB Period: Number of Participants With Function Loss of NSAA Items at Week 72 - ITT Population
Time frame: Week 72
DB Period: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: Baseline up to Week 76
Overall Treatment Period: Change From Baseline in 6MWD at Week 144
Time frame: Baseline, Week 144
Overall Treatment Period: Composite of Average Change From Baseline in TFTs at Week 144
Time frame: Baseline, Week 144
Overall Treatment Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 144
Time frame: Baseline, Week 144
Overall Treatment Period: Change From Baseline in Time to Climb 4 Stairs at Week 144
Time frame: Baseline, Week 144
Overall Treatment Period: Change From Baseline in Time to Descend 4 Stairs at Week 144
Time frame: Baseline, Week 144
Overall Treatment Period: Change From Baseline in NSAA Total Score at Week 144
Time frame: Baseline, Week 144
Overall Treatment Period: Time to Loss of Ambulation Over 144 Weeks
Time frame: Baseline up to Week 144
Overall Treatment Period: Time to Loss of Stair-Climbing Over 144 Weeks
Time frame: Baseline up to Week 144
Overall Treatment Period: Time to Loss of Stair- Descending Over 144 Weeks
Time frame: Baseline up to Week 144
Overall Treatment Period: Number of Participants With Function Loss of NSAA Items at Week 144
Time frame: Week 144
Overall Treatment Period: Number of Participants With TEAEs
Time frame: Baseline up to Week 148
OL Period: Plasma Pharmacokinetic (PK) Concentration of Ataluren
Time frame: Predose and 2 hours postdose at Week 144
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41879641Wu S, Vlodavets D, de Queiroz Campos Araujo AP, Castle J. Ataluren for the treatment of people living with nonsense mutation Duchenne muscular dystrophy: a plain language summary of Study 041. J Comp Eff Res. 2026 Apr;15(4):e250184. doi: 10.57264/cer-2025-0184. Epub 2026 Mar 25.
- ReferencesLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Vinay Penematsa, MD study director
PTC Therapeutics, Inc.
64 sites in 18 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ataluren in people with Muscular Dystrophy, Duchenne and Muscular Dystrophies, run by PTC Therapeutics at 64 sites in 18 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 64 sites in United States, India, China, Australia, Brazil and 13 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population (time frame: Baseline, Week 72). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03179631, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Mar 2026.