Duchenne Muscular DystrophyPhase 3LELANTOS-2NCT04632940
Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD
A Phase 3 study testing Pamrevlumab and Corticosteroids in people with Duchenne Muscular Dystrophy, run by Kyntra Bio at 52 sites in 11 countries.
Trial summary
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every 2 weeks in ambulatory participants with Duchenne muscular dystrophy (DMD) (age 6 to <12 years). Read more about this study
- Who is it for?
- Ages 6 to 11 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Pamrevlumab and Corticosteroids
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 73 participants
- Where
- 52 sitesin 11 countries
- Study dates
- 3 Mar 2021to 14 Dec 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 6 to 11 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Age, and consent:
- Males at least 6 to <12 years of age at screening initiation
- Written consent by participant and/or legal guardian as per regional/ country and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements
- DMD diagnosis:
- Medical history includes diagnosis of DMD and confirmed Duchenne mutation, including status of exon 44 using a validated genetic test.
- Pulmonary criteria:
Show all 25Show fewer
- Average (of screening and Day 0) percent predicted forced vital capacity (FVC) above 45%
- On a stable dose of systemic corticosteroids for a minimum of 6 months, with no substantial change in dosage for a minimum of 3 months (except for adjustments for changes in body weight) prior to screening. Corticosteroid dosage should be in compliance with the DMD Care Considerations Working Group recommendations (for example, prednisone or prednisolone 0.75 mg/kg per day or deflazacort 0.9 mg/kg per day) or stable dose. A reasonable expectation is that dosage and dosing regimen would not change significantly for the duration of the study.
- Performance criteria:
- Able to complete 6-minute walking distance (6MWD) test with a distance of at least 270 meters but no more than 450 meters on two occasions within 3 months prior to randomization with ≤10% variation between these two tests.
- Able to rise (TTSTAND) from floor in <10 seconds (without aids/orthoses) at screening visit.
- Able to undergo magnetic resonance imaging (MRI) test for the lower extremities vastus lateralis muscle.
- Vaccination:
- Agreement to receive annual influenza vaccinations during the conduct of the study.
- Laboratory criteria:
- Adequate renal function: cystatin C ≤1.4 mg/liter (L)
- Adequate hematology and electrolytes parameters:
- Platelets >100,000/microliter (μL)
- Hemoglobin >12 grams (g)/deciliter (dL)
- Absolute neutrophil count >1500/μL
- Serum calcium (Ca), potassium (K), sodium (Na), magnesium (Mg) and phosphorus (P) levels are within a clinically accepted range for DMD participants
- Adequate hepatic function:
- No history or evidence of liver disease
- Gamma glutamyl transferase (GGT) ≤3x upper limit of normal (ULN)
- Total bilirubin ≤1.5xULN
Who cannot join
- General Criteria:
- Concurrent illness other than DMD that can cause muscle weakness and/or impairment of motor function
- Severe intellectual impairment (for example, severe autism, severe cognitive impairment, severe behavioral disturbances) preventing the ability to perform study assessments in the Investigator's judgment
- Previous exposure to pamrevlumab
- Body mass index (BMI) ≥40 kg/square meter (m^2) or weight >117 kg
- History of
Show all 21Show fewer
- allergic or anaphylactic reaction to human, humanized, chimeric or murine monoclonal antibodies
- hypersensitivity to study drug or any component of study drug
- Exposure to any investigational drug (for DMD or not), in the 30 days prior to screening initiation or use of approved DMD therapies (for example, eteplirsen, ataluren, golodirsen, casimersen) within 5 half-lives of screening, whichever is longer with the exception of the systemic corticosteroids, including deflazacort
- Pulmonary and Cardiac criteria:
- Requires ≥16 hours continuous ventilation
- Poorly controlled asthma or underlying lung disease such as bronchitis, bronchiectasis, emphysema, recurrent pneumonia that in the opinion of the investigator might impact respiratory function
- Hospitalization due to respiratory failure within the 8 weeks prior to screening
- Severe uncontrolled heart failure (New York Heart Association [NYHA] Classes III-IV) or renal dysfunction, including any of the following:
- Need for intravenous diuretics or inotropic support within 8 weeks prior to screening
- Hospitalization for a heart failure exacerbation or arrhythmia within 8 weeks prior to screening
- Participants with glomerular filtration rate (GFR) of less than 30 mL/minute (min)/1.73 m^2 or with other evidence of acute kidney injury as determined by investigator
- Arrhythmia requiring anti-arrhythmic therapy
- Any other evidence of clinically significant structural or functional heart abnormality
- Clinical judgment:
- The Investigator judges that the participant will be unable to fully participate in the study and complete it for any reason, including inability to comply with study procedures and treatment, or any other relevant medical, surgical or psychiatric conditions
Trial locations and contacts
52 sites in 11 countries.
52 sites in 11 countries
United States27 sites
C.S. Mott Children's Hospital
Ann Arbor, Michigan, 48109-4234
Rare Disease Research Center
Atlanta, Georgia, 30329
Children's Hospital Colorado
Aurora, Colorado, 80045
Kennedy Krieger Institute
Baltimore, Maryland, 21205
University of Virginia Children's Hospital
Charlottesville, Virginia, 22903
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229-3026
University of Kansas Medical Center Research Institute
Fairway, Kansas, 66205
University of Florida Health Shands Hospital
Gainesville, Florida, 32610
Spectrum Health Hospitals Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
Texas Children's Hospital
Houston, Texas, 77030
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Children's Hospital Los Angeles
Los Angeles, California, 90027
Children's Wisconsin Corporate Center
Milwaukee, Wisconsin, 53226
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Shriners Hospital for Children
Portland, Oregon, 97239
University of California Davis Children's Hospital
Sacramento, California, 95817
University of Utah Health
Salt Lake City, Utah, 84108
University of California San Diego Health
San Diego, California, 92161
Seattle Children's Hospital
Seattle, Washington, 98105
Washington University School of Medicine in St. Louis
St Louis, Missouri, 63110
Rare Disease Research - Tampa
Tampa, Florida, 33614
University of Massachusetts Memorial Center
Worcester, Massachusetts, 01655
China5 sites
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Beijing, 100730
Xiangya Hospital Central South University
Changsha, Hunan, 410008
West China Second University Hospital, Sichuan University
Chengdu, Sichuan, 610041
Children's Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality, 401122
The 1st Affiliated Hospital, Sun Yat-sen University
Guangzhou, Guangdong, 510080
Italy5 sites
Istituto di Ricovero e Cura a Carattere Scientifico Eugenio Medea - Lombardia
Bosisio Parini, 23842
IRRCS Ospedale San Raffaele
Milan, Milan, 20132
Centro Clinico NeMO
Milan, 20162
Fondazione Policlinico Universitario Agostino Gemelli
Roma, 168
Ospedale Pediatrico Bambino Gesù - Roma - Gianicolo
Roma
Belgium3 sites
Universitair Ziekenhuis Gent
Ghent, Oost-Vlaanderen, 9000
Universitair Ziekenhuis Leuven - Campus Gasthuisberg
Leuven, Flemish Brabant, 3000
Centre Hospitalier Régional de la Citadelle
Liège, Liege, 4000
France3 sites
Centre Hospitalier Universitaire Nantes - Hôtel Dieu
Nantes, 44093
Association Institut de Myologie
Paris, 75012
Hôpital Hautepierre
Strasbourg, Bas-Rhin, 67200
Netherlands2 sites
Leiden Universitair Medisch Centrum
Leiden
Radboud Universitair Medisch Centrum
Nijmegen
Spain2 sites
Hospital Universitari Vall d'Hebrón
Barcelona, 08035
Hospital Universitari i Politecnic La Fe de Valencia
Valencia, 46026
United Kingdom2 sites
Leeds Teaching Hospitals NHS Trust
Leeds, England, LS1 3EX
Oxford University Hospitals NHS Foundation Trust
Oxford, England, OX3 9DU
Australia1 site
Murdoch Children's Research Institute
Parkville, Victoria, 3052
About this study
Official title: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every 2 weeks in ambulatory participants with Duchenne muscular dystrophy (DMD) (age 6 to <12 years).
Read the detailed description
This is a global, randomized, double-blind, trial of pamrevlumab or placebo in combination with systemic corticosteroids in participants with DMD, aged 6 to <12 years (ambulatory participants only). Approximately 70 participants will be randomized at a 1:1 ratio to Arm A (pamrevlumab + systemic deflazacort or equivalent potency of corticosteroids administered orally) or Arm B (placebo+ systemic deflazacort or equivalent potency of corticosteroids administered orally), respectively. Randomization will be stratified by exon 44 deletion for analysis. Stratification has no impact upon treatment assignment nor dosage.
Participants must be fully informed of the potential benefits of approved products and make an informed decision when participating in a clinical trial in which they could be randomized to placebo.
The main study has 3 study periods:
- Screening period: Up to 4 weeks
- Treatment period: 52 weeks
- Safety Follow-up period/final assessment: A visit 28 days (+/- 3 Days) and a final safety follow-up phone call 60 days (+ 3 Days) after the last dose
Each participant will receive pamrevlumab or placebo at 35 mg/kg every 2 weeks for up to 52 weeks. Participants who complete 52 weeks of treatment may be eligible for an open-label extension (OLE), offering extended treatment with pamrevlumab.
Participants who discontinue study treatment for any reason should be encouraged to return to the investigative site to complete final safety and efficacy assessments.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Pamrevlumab, Placebo, Corticosteroids
- Keywords
- Duchenne Muscular Dystrophy, DMD, Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Genetic Diseases, X-Linked
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PamrevlumabDrug
Also called FG-3019
Pamrevlumab will be administered per dose and schedule specified in the arm description.
PlaceboDrug
Placebo will be administered per schedule specified in the arm description.
CorticosteroidsDrug
Systemic deflazacort or equivalent potency of corticosteroids administered orally
| Study group | Role | What they receive |
|---|---|---|
| Pamrevlumab | Experimental | Pamrevlumab 35 milligrams (mg)/kilogram (kg) intravenously (IV) every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks Receives: Drug: Pamrevlumab; Drug: Corticosteroids |
| Placebo | Placebo comparator | Matching placebo IV every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks Receives: Drug: Placebo; Drug: Corticosteroids |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52
The NSAA consisted of 17 activities, each scored as 0 (activity could not be performed), 1 (modified method but achieved goal without physical assistance from another), or 2 (normal, achieved goal without assistance). The sum of these 17 scores was used to form a total score ranging from 0 (worst) to 34 (fully independent function). If fewer than 15 of the 17 activities were performed, the total score was considered missing. If 15 to 16 activities were performed, the total score was calculated by multiplying the sum of the scores in the x activities that were performed by 17/x. If an activity could not be performed due to disease progression/loss of ambulation, a score of 0 was assigned. Higher scores indicated better functioning. Least square (LS) mean and standard error (SE) were analyzed using a mixed model for repeated measure (MMRM).
Measured: Baseline, Week 52
Secondary outcomes (4)
Change From Baseline in 4-Stair Climb Velocity (4SCV) Assessment at Week 52
The 4SCV (centimeters [cm]/second [sec]) was calculated as the ratio of the total height (cm) of stairs climbed divided by the number of seconds taken to complete the 4-stair climb.
Measured: Baseline, Week 52
Change From Baseline in the 10-Meter Walk/Run Test at Week 52
The time (in sec) required for a participant to run or walk a distance of 10 meters as quickly as possible was calculated as velocity (meters/sec).
Measured: Baseline, Week 52
Change From Baseline in Time to Stand (TTSTAND) at Week 52
The time (in sec) required for a participant to stand from supine position has been reported. A longer time taken reflected a worse outcome.
Measured: Baseline, Week 52
Time to Loss of Ambulation (LoA) From Baseline to Week 52
Time (days) to LoA was defined as the number of days from randomization to the date of LoA, or all-cause death based on observed data, whichever occurred earlier during the on-study period. Median time (days) to LoA was calculated using Kaplan Meier Survival Estimates.
Measured: Baseline to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Nov 2020
- Study start3 Mar 2021
- Primary completion12 Jun 2023
- Study completion14 Dec 2023
Why it stopped: Study did not meet its primary endpoint.
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.
- Other trials for Duchenne Muscular DystrophyStudies 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
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 73 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kyntra Bio
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04632940
- Sponsor's protocol ID
- FGCL-3019-094
- Other IDs
- 2020-000699-39
- Acronym
- LELANTOS-2
- Status last verified
- Jul 2024
- First submitted
- 12 Nov 2020
- Last updated
- 26 Aug 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)
- ClinicalTrials.gov ID
- NCT04632940
- Acronym / sponsor ID
- LELANTOS-2 / FGCL-3019-094
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, DMD, Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study did not meet its primary endpoint.
- Enrolment
- 73 participants (actual)
- Lead sponsor
- Kyntra BioIndustry (pharma, biotech, devices)
- Study start
- 3 Mar 2021 (actual)
- Primary completion
- 12 Jun 2023 (actual)
- Study completion
- 14 Dec 2023 (actual)
- First posted
- 17 Nov 2020 (actual)
- Results
- Posted 26 Aug 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every 2 weeks in ambulatory participants with Duchenne muscular dystrophy (DMD) (age 6 to <12 years).
Detailed description
This is a global, randomized, double-blind, trial of pamrevlumab or placebo in combination with systemic corticosteroids in participants with DMD, aged 6 to <12 years (ambulatory participants only). Approximately 70 participants will be randomized at a 1:1 ratio to Arm A (pamrevlumab + systemic deflazacort or equivalent potency of corticosteroids administered orally) or Arm B (placebo+ systemic deflazacort or equivalent potency of corticosteroids administered orally), respectively. Randomization will be stratified by exon 44 deletion for analysis. Stratification has no impact upon treatment assignment nor dosage.
Participants must be fully informed of the potential benefits of approved products and make an informed decision when participating in a clinical trial in which they could be randomized to placebo.
The main study has 3 study periods:
- Screening period: Up to 4 weeks
- Treatment period: 52 weeks
- Safety Follow-up period/final assessment: A visit 28 days (+/- 3 Days) and a final safety follow-up phone call 60 days (+ 3 Days) after the last dose
Each participant will receive pamrevlumab or placebo at 35 mg/kg every 2 weeks for up to 52 weeks. Participants who complete 52 weeks of treatment may be eligible for an open-label extension (OLE), offering extended treatment with pamrevlumab.
Participants who discontinue study treatment for any reason should be encouraged to return to the investigative site to complete final safety and efficacy assessments.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Pamrevlumab | Experimental | Drug: Pamrevlumab, Drug: CorticosteroidsPamrevlumab 35 milligrams (mg)/kilogram (kg) intravenously (IV) every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks |
| Placebo | Placebo comparator | Drug: Placebo, Drug: CorticosteroidsMatching placebo IV every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks |
Who may qualify
- Ages
- 6 to 11 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (25 listed)
- Males at least 6 to <12 years of age at screening initiation
- Written consent by participant and/or legal guardian as per regional/ country and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements
- Medical history includes diagnosis of DMD and confirmed Duchenne mutation, including status of exon 44 using a validated genetic test.
- Average (of screening and Day 0) percent predicted forced vital capacity (FVC) above 45%
- On a stable dose of systemic corticosteroids for a minimum of 6 months, with no substantial change in dosage for a minimum of 3 months (except for adjustments for changes in body weight) prior to screening. Corticosteroid dosage should be in compliance with the DMD Care Considerations Working Group recommendations (for example, prednisone or prednisolone 0.75 mg/kg per day or deflazacort 0.9 mg/kg per day) or stable dose. A reasonable expectation is that dosage and dosing regimen would not change significantly for the duration of the study.
Key exclusion criteria (21 listed)
- Concurrent illness other than DMD that can cause muscle weakness and/or impairment of motor function
- Severe intellectual impairment (for example, severe autism, severe cognitive impairment, severe behavioral disturbances) preventing the ability to perform study assessments in the Investigator's judgment
- Previous exposure to pamrevlumab
- Body mass index (BMI) ≥40 kg/square meter (m^2) or weight >117 kg
- History of
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 |
|---|---|---|
| PamrevlumabAlso: FG-3019Pamrevlumab will be administered per dose and schedule specified in the arm description. | Drug | Pamrevlumab |
| PlaceboPlacebo will be administered per schedule specified in the arm description. | Drug | Placebo |
| CorticosteroidsSystemic deflazacort or equivalent potency of corticosteroids administered orally | Drug | Pamrevlumab, Placebo |
Primary outcome
Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52
Time frame: Baseline, Week 52
The NSAA consisted of 17 activities, each scored as 0 (activity could not be performed), 1 (modified method but achieved goal without physical assistance from another), or 2 (normal, achieved goal without assistance). The sum of these 17 scores was used to form a total score ranging from 0 (worst) to 34 (fully independent function). If fewer than 15 of the 17 activities were performed, the total score was considered missing. If 15 to 16 activities were performed, the total score was calculated by multiplying the sum of the scores in the x activities that were performed by 17/x. If an activity could not be performed due to disease progression/loss of ambulation, a score of 0 was assigned. Higher scores indicated better functioning. Least square (LS) mean and standard error (SE) were analyzed using a mixed model for repeated measure (MMRM).
4 secondary outcomes
Change From Baseline in 4-Stair Climb Velocity (4SCV) Assessment at Week 52
Time frame: Baseline, Week 52
Change From Baseline in the 10-Meter Walk/Run Test at Week 52
Time frame: Baseline, Week 52
Change From Baseline in Time to Stand (TTSTAND) at Week 52
Time frame: Baseline, Week 52
Time to Loss of Ambulation (LoA) From Baseline to Week 52
Time frame: Baseline to Week 52
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Undecided
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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.
The record lists no contact. Use the official record to reach the sponsor.
52 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Pamrevlumab and Corticosteroids in people with Duchenne Muscular Dystrophy, run by Kyntra Bio at 52 sites in 11 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 25 inclusion and 21 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 26 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 52 sites in United States, China, Italy, Belgium, France and 6 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 26 Aug 2024). 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?
Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 (time frame: Baseline, Week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 26 Aug 2024.