Muscular Dystrophy, DuchennePhase 3NCT02090959
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
A Phase 3 study testing Ataluren in people with Muscular Dystrophy, Duchenne and Muscular Dystrophies, run by PTC Therapeutics at 58 sites in 19 countries.
Trial summary
The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren [PTC124-GD-020-DMD {NCT01826487}]) to augment the overall safety database. Read more about this study
- Who is it for?
- Ages 7 to 15 years, 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?
- No, everyone receives an active treatment
- How many people
- 219 participants
- Where
- 58 sitesin 19 countries
- Study dates
- 20 Mar 2014to 12 Jun 2018
- 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 7 to 15 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Completion of study treatment in the previous Phase 3, double-blind study (PTC124-GD-020-DMD).
- Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial. Note: If the study candidate is considered a child under local regulation, a parent or legal guardian must provide written consent prior to initiation of study screening procedures and the study candidate may be required to provide written assent. The rules of the responsible Institutional Review Board/Independent Ethic Committee (IRB/IEC) regarding whether 1 or both parents must provide consent and the appropriate ages for obtaining consent and assent from the participant should be followed.
- In participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the period of study drug administration and 6-week follow-up period.
- Willingness and ability to comply with scheduled visits, ataluren administration plan, study procedures, laboratory tests, and study restrictions.
Who cannot join
- Known hypersensitivity to any of the ingredients or excipients of the study drug (Litesse® UltraTM [refined polydextrose], polyethylene glycol 3350, Lutrol® micro F127 [poloxamer 407], mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, Cab-O-Sil® M5P [colloidal silica], and magnesium stearate).
- Ongoing participation in any other therapeutic clinical trial.
- Prior or ongoing medical condition (for example, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings, electrocardiogram (ECG) findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
Trial locations and contacts
58 sites in 19 countries.
58 sites in 19 countries
United States21 sites
Children's Hospital Colorado - Center for Cancer and Blood Disorders
Aurora, Colorado, 80045
Children's Hospital Boston
Boston, Massachusetts, 02115
Rush University Medical Center
Chicago, Illinois, 60612
Children's Hospital Cincinnati
Cincinnati, Ohio, 45229
Nationwide Children's Hospital
Columbus, Ohio, 43209
Childrens Medical Center Dallas, Texas
Dallas, Texas, 75207
Child Neurology Center of Northwest Florida
Gulf Breeze, Florida, 32561
Texas Children's Hospital
Houston, Texas, 77030
University of Iowa
Iowa City, Iowa, 52242
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of California, Los Angeles
Los Angeles, California, 90095
University of Minnesota
Minneapolis, Minnesota, 55455
Columbia University College of Physicians & Surgeons
New York, New York, 10032
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Oregon Health & Science University
Portland, Oregon, 97239
UC Davis Medical Center
Sacramento, California, 95817
University of Utah
Salt Lake City, Utah, 84112
Seattle Children's Hospital - Childhood Cancer and Blood Disorders
Seattle, Washington, 98105
Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research
St Louis, Missouri, 63110
Stanford University Medical Center
Stanford, California, 94305
France4 sites
Hospital de la Timone
Marseille, 13385
CHU de Nantes
Nantes, 44093
Hopital Necker - Enfants Malades
Paris, 75015
Groupe Hospitalier Pitie-Salpetriere
Paris, 75651
Italy4 sites
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
Milan, 20122
Bambino Gesu Hospital
Rome, 00165
U.O. Complessa di Neuropsichiatria Infantile
Rome, 00168
Policlinico Universitario G. Martino
Sicily, 98125
Canada3 sites
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8
Children's Hospital of Western Ontario
London, Ontario, N6C 2RC
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3V4
Germany3 sites
Universitaetsklinikum Essen
Essen, 45122
University Medical Center Freiburg
Freiburg im Breisgau, 79106
Universitat Munchen - von Haunersche Kinder Clinic
München, 80337
United Kingdom3 sites
University College London Institute of Child Health
London, WC1N 3JH
Royal Manchester Children's Hospital
Manchester, M13 9WL
The Newcastle upon Tyne Hospitals, NHS Foundation Trust
Newcastle upon Tyne, NE1 3BZ
Australia2 sites
The Royal Children's Hospital
Parkville, Victoria, 3052
The Children's Hospital at Westmead
Westmead, New South Wales, 2145
Brazil2 sites
Universidade Federal do Rio de Janeiro - Instituto de Puericultura e Pediatria Martagao Gesteira
Rio de Janeiro, 21.941-912
Sao Paulo University -HC/FMUSP
São Paulo, 05403-900
Chile2 sites
Hospital Luis Calvo Mackenna
Santiago, Santiago Metropolitan
Hospital Clinico Universidad Catolica
Santiago, 8330073
Czechia2 sites
University Hospital Brno
Brno, 635 00
Motol University Hospital
Prague, 150 06
Spain2 sites
Hospital Sant Joan de Deu
Barcelona, 08950
Hospital Universitari i Politecnic la Fe
Valencia, 46026
Sweden2 sites
Queen Silvia Children's Hospital
Gothenburg, SE-416 85
Astrid Lindgren Childrens Hospital
Stockholm, 17176
Turkey (Türkiye)2 sites
Hacettepe Childrens Hospital
Ankara, 06100
Erciyes University Faculty of Medicine
Talas/Kayseri, 38039
About this study
Official title: A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy
The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren [PTC124-GD-020-DMD {NCT01826487}]) to augment the overall safety database. Screening and baseline procedures are structured to avoid a gap in treatment between the double-blind study (PTC124-GD-020-DMD) and this extension study.
This study may be further extended by amendment until either ataluren becomes commercially available or the clinical development of ataluren in duchenne muscular dystrophy (DMD) is discontinued.
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases
- Being tested
- Ataluren
- 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
Ataluren will be administered per the dose and schedule specified in the arm.
| Study group | Role | What they receive |
|---|---|---|
| Ataluren | Experimental | Participants will receive ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks. Receives: Drug: Ataluren |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
An adverse event (AE): any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of AEs: graded per Common Terminology Criteria for AEs (CTCAE), Version 3.0 as Grade 1 (mild), 2 (moderate), 3 (severe), 4 (life-threatening), 5 (death). Drug-related AEs: AEs with possible, probable, unlikely relationship, or unrelated to study drug. Serious AEs (SAEs): death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: an AE that occurred or worsened in the period extending from first dose of study drug in this study to 6 weeks after last dose of study drug in this study. A summary of other non-serious AEs and all SAEs, regardless of causality is located in Reported AE section.
Measured: Baseline (Day 1) up to 6 weeks post-treatment (Week 150)
Number of Participants With Abnormalities in Clinical Laboratory Parameters
Abnormalities in laboratory variables as pre-defined in protocol for safety-monitoring were: Hepatic (Serum alanine aminotransferase [ALT]: increase of greater than [>] 150 units/liter [U/L] with stable or decrease of creatinine kinese [CK]; Serum glutamyl amino transferase [GGT] [U/L]: Grade 2 [>2.5 - 5.0 * upper limit of normal {ULN}]), renal (Serum cystatin C miiligrams/liter [mg/L] >1.33 - 2.00 mg/L; Serum blood urea nitrogen [UREAN] [millimoles/liter {mmol/L}] greater than or equal to [≥]1.5 - 3.0 * ULN; Urine occult blood: 2+ [Small], 3+ [Moderate], 4+ [Large]), and electrolytes (Serum sodium: low [mmol/L], Grade 3-4 [less than {<}130 mmol/L]; serum potassium: high [mmol/L], Grade 3-4 [>6.0 mmol/L]; and Serum bicarbonate [mmol/L]: Grade 2 [<16 - 11 mmol/L]).
Measured: Baseline (Day 1) up to 6 weeks post-treatment (Week 150)
Secondary outcomes (17)
Change From Baseline in 6MWD at Week 144
The 6MWD test was performed in a 30 meters long flat corridor, where the participant was 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 by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test.
Measured: Baseline, Week 144
Change From Baseline in Time to Stand From Supine Position at Week 144
If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used.
Measured: Baseline, Week 144
Change From Baseline in Time to Walk/Run 10 Meters at Week 144
If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used.
Measured: Baseline, Week 144
Change From Baseline in Time to Climb 4 Stairs at Week 144
If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used.
Measured: Baseline, Week 144
Change From Baseline in Time to Descend 4 Stairs at Week 144
If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used.
Measured: Baseline, Week 144
Change From Baseline in Physical Function Total Score as Measured by NSAA at Week 144
Physical function was assessed via the NSAA, a functional scale specifically designed for ambulant Duchenne muscular dystrophy (DMD) participants. The assessment comprised tests for 17 abilities of a participant, such as ability to stand, rise from the floor, get from lying to sitting, get from sitting to standing, raise one's head, stand on one's heels, hop, jump, and run. For each activity, a score of 0, 1, or 2 was recorded, with 0 = "unable to achieve independently," 1 = "modified method but achieves goal independent of physical assistance from another," or 2 = "normal- achieves goal without any assistance." The sum of these scores (except for 'raise one's head' activity score) was reported as the ordinal total score, which was transformed to a linear total score ranging from 0 (worst) to 100 (best). Participants with confirmed loss of ambulation at a particular visit were assigned a score of 0.
Measured: Baseline, Week 144
Change From Baseline in PUL Total Score at Week 144
The PUL was used to assess motor performance of the upper limb. The PUL scale includes 22 items; an entry item defining the starting functional level, and 21 items subdivided into shoulder level (4 items), elbow level (9 items), and distal level (8 items) dimensions. Scoring options per item may not be uniform and may vary from 0-1 and 0-6, according to the performance, with higher values corresponding to better performance. Each dimension was scored separately with a maximum score of 16 for shoulder level, 34 for elbow level, and 24 for distal level. Total score was calculated by adding the 3 level scores, with a maximum global score of 74 (total score range = 0-74). Higher score = better outcome.
Measured: Baseline, Week 144
Change From Baseline in Percent Predicted FVC as Measured by Spirometry at Week 144
FVC is a standard pulmonary function test. FVC was defined as the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Percent predicted FVC (in %) = [(observed FVC)/(predicted FVC)]*100.
Measured: Baseline, Week 144
Change From Baseline in Percent Predicted FEV1 as Measured by Spirometry at Week 144
FEV1 is a standard pulmonary function test. FEV1 was defined as the volume of air that can forcibly be blown out in 1 second, after full inspiration in the upright position, measured in liters. Percent predicted FEV1 (in %) = [(observed FEV1)/(predicted FEV1)]*100.
Measured: Baseline, Week 144
Change From Baseline in PEF as Measured by Spirometry at Week 144
PEF was defined as the maximum airflow during a forced expiration beginning with the lungs fully inflated.
Measured: Baseline, Week 144
Change From Baseline in PCF as Measured by Spirometry at Week 144
PCF measures an individual's maximum speed of expiration during cough.
Measured: Baseline, Week 144
Change From Baseline in PODCI Transfers/Basic Mobility Score at Week 144
Changes in health-related quality of life (HRQL) were measured via the PODCI questionnaire that has been shown to correlate with disease progression and clinical outcome measures in DMD. PODCI includes a Global Functioning Scale and 5 core scales: Upper Extremity and Physical Function,Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort,and Happiness. The following PODCI domain was prespecified in the protocol for analysis:Transfers/Basic Mobility domain assesses difficulty experienced in performing routine motor activities in daily life. Each domain was scored from 0 to 100, with 0 representing a poor outcome/worse health, while 100 representing the highest level of functioning and least pain.
Measured: Baseline, Week 144
Number of Participants With Change From Baseline in Activities of Daily Living and Disease Status at Week 144, as Assessed by a Standardized Survey Administered by Site Personnel
Changes in activities of daily living and disease symptoms were captured via a DMD-specific survey administered by Site personnel. At screening or baseline, the participant and/or parent/caregiver were asked to identify any activities of daily living (for example, ambulation, balance, personal hygiene/grooming, dressing and undressing, self-feeding, using the bathroom, handwriting, school performance, behavior or energy level) or symptoms that were affected by the participant's DMD. At post-baseline visit (Week 144), the same participant and/or parent/caregiver was asked to describe any changes from baseline in those activities of daily living/symptoms, within the following categories: physical functioning; general energy level; cognition/school function; emotional/social functioning; and sleep. Changes from baseline were reported on a 5-point Likert scale: 1 (much better), 2 (slightly better), 3 (unchanged), 4 (slightly worse), or 5 (much worse).
Measured: Baseline, Week 144
Ataluren Plasma Concentration
Pre-dose ataluren plasma concentrations prior to morning ataluren administration at each clinic visit was assessed using a validated high performance liquid chromatography with tandem mass spectrometry (HPLC/MS-MS) method.
Measured: Pre-dose at Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, and 144
Change From Baseline in Systolic and Diastolic Blood Pressure at Week 144
Blood pressure determination was performed with the participant in a sitting position after a 5-minute rest.
Measured: Baseline, Week 144
Change From Baseline in Pulse Rate at Week 144
Pulse rate determination was performed with the participant in a sitting position after a 5-minute rest.
Measured: Baseline, Week 144
Change From Baseline in Body Temperature at Week 144
Measured: Baseline, Week 144
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2014 (estimated)
- Study start20 Mar 2014
- Primary completion12 Jun 2018
- Study completion12 Jun 2018
Why it stopped: Sponsor decision due to commercial availability of ataluren.
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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 219 participants (actual)
Sponsor and investigators
- Lead sponsor
- PTC Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Francesco Bibbiani, M.D., Study director, PTC Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02090959
- Sponsor's protocol ID
- PTC124-GD-020e-DMD
- Status last verified
- Aug 2020
- First submitted
- 17 Mar 2014
- Last updated
- 11 Aug 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Thangarajh M, Elfring GL, Trifillis P, McIntosh J, Peltz SW; Ataluren Phase 2b Study Group. The relationship between deficit in digit span and genotype in nonsense mutation Duchenne muscular dystrophy. Neurology. 2018 Sep 25;91(13):e1215-e1219. doi: 10.1212/WNL.0000000000006245. Epub 2018 Aug 22.
PubMed 30135256
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy
- ClinicalTrials.gov ID
- NCT02090959
- Acronym / sponsor ID
- PTC124-GD-020e-DMD
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, 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
- TerminatedReason stopped: Sponsor decision due to commercial availability of ataluren.
- Enrolment
- 219 participants (actual)
- Lead sponsor
- PTC TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 20 Mar 2014 (actual)
- Primary completion
- 12 Jun 2018 (actual)
- Study completion
- 12 Jun 2018 (actual)
- First posted
- 19 Mar 2014 (estimated)
- Results
- Posted 11 Aug 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren [PTC124-GD-020-DMD {NCT01826487}]) to augment the overall safety database. Screening and baseline procedures are structured to avoid a gap in treatment between the double-blind study (PTC124-GD-020-DMD) and this extension study.
This study may be further extended by amendment until either ataluren becomes commercially available or the clinical development of ataluren in duchenne muscular dystrophy (DMD) is discontinued.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ataluren | Experimental | Drug: AtalurenParticipants will receive ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks. |
Who may qualify
- Ages
- 7 to 15 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Completion of study treatment in the previous Phase 3, double-blind study (PTC124-GD-020-DMD).
- Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial. Note: If the study candidate is considered a child under local regulation, a parent or legal guardian must provide written consent prior to initiation of study screening procedures and the study candidate may be required to provide written assent. The rules of the responsible Institutional Review Board/Independent Ethic Committee (IRB/IEC) regarding whether 1 or both parents must provide consent and the appropriate ages for obtaining consent and assent from the participant should be followed.
- In participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the period of study drug administration and 6-week follow-up period.
- Willingness and ability to comply with scheduled visits, ataluren administration plan, study procedures, laboratory tests, and study restrictions.
Key exclusion criteria (3 listed)
- Known hypersensitivity to any of the ingredients or excipients of the study drug (Litesse® UltraTM [refined polydextrose], polyethylene glycol 3350, Lutrol® micro F127 [poloxamer 407], mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, Cab-O-Sil® M5P [colloidal silica], and magnesium stearate).
- Ongoing participation in any other therapeutic clinical trial.
- Prior or ongoing medical condition (for example, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings, electrocardiogram (ECG) findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
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: PTC124Ataluren will be administered per the dose and schedule specified in the arm. | Drug | Ataluren |
Primary outcome
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: Baseline (Day 1) up to 6 weeks post-treatment (Week 150)
An adverse event (AE): any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of AEs: graded per Common Terminology Criteria for AEs (CTCAE), Version 3.0 as Grade 1 (mild), 2 (moderate), 3 (severe), 4 (life-threatening), 5 (death). Drug-related AEs: AEs with possible, probable, unlikely relationship, or unrelated to study drug. Serious AEs (SAEs): death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: an AE that occurred or worsened in the period extending from first dose of study drug in this study to 6 weeks after last dose of study drug in this study. A summary of other non-serious AEs and all SAEs, regardless of causality is located in Reported AE section.
Number of Participants With Abnormalities in Clinical Laboratory Parameters
Time frame: Baseline (Day 1) up to 6 weeks post-treatment (Week 150)
Abnormalities in laboratory variables as pre-defined in protocol for safety-monitoring were: Hepatic (Serum alanine aminotransferase [ALT]: increase of greater than [>] 150 units/liter [U/L] with stable or decrease of creatinine kinese [CK]; Serum glutamyl amino transferase [GGT] [U/L]: Grade 2 [>2.5 - 5.0 * upper limit of normal {ULN}]), renal (Serum cystatin C miiligrams/liter [mg/L] >1.33 - 2.00 mg/L; Serum blood urea nitrogen [UREAN] [millimoles/liter {mmol/L}] greater than or equal to [≥]1.5 - 3.0 * ULN; Urine occult blood: 2+ [Small], 3+ [Moderate], 4+ [Large]), and electrolytes (Serum sodium: low [mmol/L], Grade 3-4 [less than {<}130 mmol/L]; serum potassium: high [mmol/L], Grade 3-4 [>6.0 mmol/L]; and Serum bicarbonate [mmol/L]: Grade 2 [<16 - 11 mmol/L]).
17 secondary outcomes
Change From Baseline in 6MWD at Week 144
Time frame: Baseline, Week 144
Change From Baseline in Time to Stand From Supine Position at Week 144
Time frame: Baseline, Week 144
Change From Baseline in Time to Walk/Run 10 Meters at Week 144
Time frame: Baseline, Week 144
Change From Baseline in Time to Climb 4 Stairs at Week 144
Time frame: Baseline, Week 144
Change From Baseline in Time to Descend 4 Stairs at Week 144
Time frame: Baseline, Week 144
Change From Baseline in Physical Function Total Score as Measured by NSAA at Week 144
Time frame: Baseline, Week 144
Change From Baseline in PUL Total Score at Week 144
Time frame: Baseline, Week 144
Change From Baseline in Percent Predicted FVC as Measured by Spirometry at Week 144
Time frame: Baseline, Week 144
Change From Baseline in Percent Predicted FEV1 as Measured by Spirometry at Week 144
Time frame: Baseline, Week 144
Change From Baseline in PEF as Measured by Spirometry at Week 144
Time frame: Baseline, Week 144
Change From Baseline in PCF as Measured by Spirometry at Week 144
Time frame: Baseline, Week 144
Change From Baseline in PODCI Transfers/Basic Mobility Score at Week 144
Time frame: Baseline, Week 144
Number of Participants With Change From Baseline in Activities of Daily Living and Disease Status at Week 144, as Assessed by a Standardized Survey Administered by Site Personnel
Time frame: Baseline, Week 144
Ataluren Plasma Concentration
Time frame: Pre-dose at Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, and 144
Change From Baseline in Systolic and Diastolic Blood Pressure at Week 144
Time frame: Baseline, Week 144
Change From Baseline in Pulse Rate at Week 144
Time frame: Baseline, Week 144
Change From Baseline in Body Temperature at Week 144
Time frame: Baseline, Week 144
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 30135256Thangarajh M, Elfring GL, Trifillis P, McIntosh J, Peltz SW; Ataluren Phase 2b Study Group. The relationship between deficit in digit span and genotype in nonsense mutation Duchenne muscular dystrophy. Neurology. 2018 Sep 25;91(13):e1215-e1219. doi: 10.1212/WNL.0000000000006245. Epub 2018 Aug 22.
- Related InfoLink 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.
Francesco Bibbiani, M.D. study director
PTC Therapeutics
58 sites in 19 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 58 sites in 19 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 4 inclusion and 3 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: stopped early, as of the sponsor's last update on 11 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 58 sites in United States, France, Italy, Canada, Germany and 14 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. 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 11 Aug 2020). 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?
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) (time frame: Baseline (Day 1) up to 6 weeks post-treatment (Week 150)). The study lists 2 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 NCT02090959, 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 11 Aug 2020.