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Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD (LELANTOS-2)

Primary Purpose

Duchenne Muscular Dystrophy

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Pamrevlumab
Placebo
Corticosteroids
Sponsored by
FibroGen
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring 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

Eligibility Criteria

6 Years - 11 Years (Child)MaleDoes not accept healthy volunteers

Inclusion Criteria:

Age, and consent:

  1. Males at least 6 to <12 years of age at screening initiation
  2. Written consent by legal guardian as per regional/ country and/or IRB/IEC requirements

    DMD diagnosis:

  3. Medical history includes diagnosis of DMD and confirmed Duchenne mutation using a validated genetic test.

    Pulmonary criteria:

  4. Average (of screening and day 0) percent predicted FVC above 45%
  5. 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 (e.g. 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:

  6. Able to complete 6MWD test with a distance of at least 270M but no more than 450M on two occasions within 3 months prior to Randomization with ≤10% variation between these two tests.
  7. Able to rise (TTSTAND) from floor in <10 seconds (without aids/orthoses) at screening visit.
  8. Able to undergo MRI test for the lower extremities vastus lateralis muscle.

    Vaccination:

  9. Received pneumococcal vaccine (PPSV23) (or any other pneumococcal polysaccharide vaccine as per national recommendations) and is receiving annual influenza vaccinations.

    Laboratory criteria:

  10. Adequate renal function: cystatin C ≤1.4 mg/L
  11. Adequate hematology and electrolytes parameters:

    1. Platelets >100,000/mcL
    2. Hemoglobin >12 g/dL
    3. Absolute neutrophil count >1500 /μL
    4. Serum calcium (Ca), potassium (K), sodium (Na), magnesium (Mg) and phosphorus (P) levels are within a clinically accepted range
  12. Adequate hepatic function:

    1. No history or evidence of liver disease
    2. Gamma glutamyl transferase (GGT) ≤3x upper limit of normal (ULN)
    3. Total bilirubin ≤1.5xULN

Exclusion Criteria:

General Criteria:

  1. Concurrent illness other than DMD that can cause muscle weakness and/or impairment of motor function
  2. Severe intellectual impairment (eg, severe autism, severe cognitive impairment, severe behavioral disturbances) preventing the ability to perform study assessments in the Investigator's judgment
  3. Previous exposure to pamrevlumab
  4. BMI ≥40 kg/m2 or weight >117 kg
  5. History of allergic or anaphylactic reaction to human, humanized, chimeric or murine monoclonal antibodies
  6. Exposure to any investigational drug (for DMD or not), in the 30 days prior to screening initiation or use of approved DMD therapies (e.g., eteplirsen, ataluren, golodirsen) within 5 half-lives of screening, whichever is longer with the exception of the systemic corticosteroids, including deflazacort

    Pulmonary and Cardiac criteria:

  7. Requires ≥16 hours continuous ventilation
  8. 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
  9. Hospitalization due to respiratory failure within the 8 weeks prior to screening
  10. Severe uncontrolled heart failure (NYHA Classes III-IV), including any of the following:

    1. Need for intravenous diuretics or inotropic support within 8 weeks prior to screening
    2. Hospitalization for a heart failure exacerbation or arrhythmia within 8 weeks prior to screening
  11. Arrhythmia requiring anti-arrhythmic therapy
  12. Any other evidence of clinically significant structural or functional heart abnormality

    Clinical judgment:

  13. The Investigator judges that the subject 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

Sites / Locations

  • Arkansas Children's Hospital
  • Children's Hospital Los Angeles
  • University of California Davis Children's Hospital
  • University of California San Diego Health
  • Children's Hospital Colorado
  • University of Florida Health Shands Hospital
  • Rare Disease Research - Tampa
  • Rare Disease Research Center
  • Ann & Robert H. Lurie Children's Hospital of Chicago
  • University of Iowa Hospitals and Clinics
  • University of Kansas Medical Center Research Institute
  • Kennedy Krieger Institute
  • University of Massachusetts Memorial Center
  • C.S. Mott Children's Hospital
  • Spectrum Health Hospitals Helen DeVos Children's Hospital
  • Washington University School of Medicine in St. Louis
  • Cincinnati Children's Hospital Medical Center
  • Shriners Hospital for Children
  • Penn State Health Milton S. Hershey Medical Center
  • Children's Hospital of Philadelphia
  • Vanderbilt University Medical Center
  • Texas Children's Hospital
  • University of Utah Health
  • University of Virginia Children's Hospital
  • Children's Hospital of The King's Daughters
  • Seattle Children's Hospital
  • Children's Wisconsin Corporate Center
  • Murdoch Children's Research Institute
  • Klinik Favoriten
  • Universitair Ziekenhuis Leuven - Campus Gasthuisberg
  • Centre Hospitalier Régional de la Citadelle
  • Universitair Ziekenhuis Gent
  • London Health Sciences Centre
  • Children's Hospital of Chongqing Medical University
  • The 1st Affiliated Hospital, Sun Yat-sen University
  • Xiangya Hospital Central South University
  • West China Second University Hospital, Sichuan University
  • Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
  • Hôpital Hautepierre
  • Centre Hospitalier Universitaire Nantes - Hôtel Dieu
  • Association Institut de Myologie
  • IRRCS Ospedale San Raffaele
  • Istituto di Ricovero e Cura a Carattere Scientifico Eugenio Medea - Lombardia
  • Centro Clinico NeMO
  • Fondazione Policlinico Universitario Agostino Gemelli
  • Ospedale Pediatrico Bambino Gesù - Roma - Gianicolo
  • Leiden Universitair Medisch Centrum
  • Radboud Universitair Medisch Centrum
  • Hospital Universitari Vall d'Hebrón
  • Hospital Universitari i Politecnic La Fe de Valencia
  • Leeds Teaching Hospitals NHS Trust
  • Oxford University Hospitals NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Pamrevlumab

Placebo

Arm Description

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

Matching placebo IV every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks

Outcomes

Primary Outcome Measures

Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52

Secondary Outcome Measures

Change From Baseline in 4-Stair Climb Velocity (4SCV) Assessment at Week 52
Change From Baseline in the 10-Meter Walk/Run Test at Week 52
Change From Baseline in Time to Stand (TTSTAND) at Week 52
Change From Baseline in Time to Loss of Ambulation (LoA) at Week 52

Full Information

First Posted
November 12, 2020
Last Updated
January 25, 2023
Sponsor
FibroGen
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1. Study Identification

Unique Protocol Identification Number
NCT04632940
Brief Title
Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD
Acronym
LELANTOS-2
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)
Study Type
Interventional

2. Study Status

Record Verification Date
January 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
December 11, 2020 (Actual)
Primary Completion Date
August 31, 2023 (Anticipated)
Study Completion Date
May 31, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
FibroGen

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief 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).
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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, Genetic Diseases, Inborn, Genetic Diseases, X-Linked

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
70 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Pamrevlumab
Arm Type
Experimental
Arm Description
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
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Matching placebo IV every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks
Intervention Type
Drug
Intervention Name(s)
Pamrevlumab
Other Intervention Name(s)
FG-3019
Intervention Description
Pamrevlumab will be administered per dose and schedule specified in the arm description.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo will be administered per schedule specified in the arm description.
Intervention Type
Drug
Intervention Name(s)
Corticosteroids
Intervention Description
Systemic deflazacort or equivalent potency of corticosteroids administered orally
Primary Outcome Measure Information:
Title
Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52
Time Frame
Baseline, Week 52
Secondary Outcome Measure Information:
Title
Change From Baseline in 4-Stair Climb Velocity (4SCV) Assessment at Week 52
Time Frame
Baseline, Week 52
Title
Change From Baseline in the 10-Meter Walk/Run Test at Week 52
Time Frame
Baseline, Week 52
Title
Change From Baseline in Time to Stand (TTSTAND) at Week 52
Time Frame
Baseline, Week 52
Title
Change From Baseline in Time to Loss of Ambulation (LoA) at Week 52
Time Frame
Baseline, Week 52

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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: 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 Exclusion Criteria: 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 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
Facility Information:
Facility Name
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
University of California Davis Children's Hospital
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
University of California San Diego Health
City
San Diego
State/Province
California
ZIP/Postal Code
92161
Country
United States
Facility Name
Children's Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of Florida Health Shands Hospital
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
Rare Disease Research - Tampa
City
Tampa
State/Province
Florida
ZIP/Postal Code
33614
Country
United States
Facility Name
Rare Disease Research Center
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Facility Name
Ann & Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
University of Iowa Hospitals and Clinics
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
University of Kansas Medical Center Research Institute
City
Fairway
State/Province
Kansas
ZIP/Postal Code
66205
Country
United States
Facility Name
Kennedy Krieger Institute
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
University of Massachusetts Memorial Center
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01655
Country
United States
Facility Name
C.S. Mott Children's Hospital
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109-4234
Country
United States
Facility Name
Spectrum Health Hospitals Helen DeVos Children's Hospital
City
Grand Rapids
State/Province
Michigan
ZIP/Postal Code
49503
Country
United States
Facility Name
Washington University School of Medicine in St. Louis
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229-3026
Country
United States
Facility Name
Shriners Hospital for Children
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Penn State Health Milton S. Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Utah Health
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Facility Name
University of Virginia Children's Hospital
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22903
Country
United States
Facility Name
Children's Hospital of The King's Daughters
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23507
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
Children's Wisconsin Corporate Center
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
Murdoch Children's Research Institute
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
Klinik Favoriten
City
Wien
State/Province
Vienna
ZIP/Postal Code
1100
Country
Austria
Facility Name
Universitair Ziekenhuis Leuven - Campus Gasthuisberg
City
Leuven
State/Province
Flemish Brabant
ZIP/Postal Code
3000
Country
Belgium
Facility Name
Centre Hospitalier Régional de la Citadelle
City
Liège
State/Province
Liege
ZIP/Postal Code
4000
Country
Belgium
Facility Name
Universitair Ziekenhuis Gent
City
Gent
State/Province
Oost-Vlaanderen
ZIP/Postal Code
9000
Country
Belgium
Facility Name
London Health Sciences Centre
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5W9
Country
Canada
Facility Name
Children's Hospital of Chongqing Medical University
City
Chongqing
State/Province
Chongqing
ZIP/Postal Code
401122
Country
China
Facility Name
The 1st Affiliated Hospital, Sun Yat-sen University
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
510080
Country
China
Facility Name
Xiangya Hospital Central South University
City
Changsha
State/Province
Hunan
ZIP/Postal Code
410008
Country
China
Facility Name
West China Second University Hospital, Sichuan University
City
Chengdu
State/Province
Sichuan
ZIP/Postal Code
610041
Country
China
Facility Name
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
City
Beijing
ZIP/Postal Code
100730
Country
China
Facility Name
Hôpital Hautepierre
City
Strasbourg
State/Province
Bas-Rhin
ZIP/Postal Code
67200
Country
France
Facility Name
Centre Hospitalier Universitaire Nantes - Hôtel Dieu
City
Nantes
ZIP/Postal Code
44093
Country
France
Facility Name
Association Institut de Myologie
City
Paris
ZIP/Postal Code
75012
Country
France
Facility Name
IRRCS Ospedale San Raffaele
City
Milano
State/Province
Milan
ZIP/Postal Code
20132
Country
Italy
Facility Name
Istituto di Ricovero e Cura a Carattere Scientifico Eugenio Medea - Lombardia
City
Bosisio ParIni
ZIP/Postal Code
23842
Country
Italy
Facility Name
Centro Clinico NeMO
City
Milano
ZIP/Postal Code
20162
Country
Italy
Facility Name
Fondazione Policlinico Universitario Agostino Gemelli
City
Roma
ZIP/Postal Code
168
Country
Italy
Facility Name
Ospedale Pediatrico Bambino Gesù - Roma - Gianicolo
City
Roma
Country
Italy
Facility Name
Leiden Universitair Medisch Centrum
City
Leiden
Country
Netherlands
Facility Name
Radboud Universitair Medisch Centrum
City
Nijmegen
Country
Netherlands
Facility Name
Hospital Universitari Vall d'Hebrón
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
Hospital Universitari i Politecnic La Fe de Valencia
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Facility Name
Leeds Teaching Hospitals NHS Trust
City
Leeds
State/Province
England
ZIP/Postal Code
LS1 3EX
Country
United Kingdom
Facility Name
Oxford University Hospitals NHS Foundation Trust
City
Oxford
State/Province
England
ZIP/Postal Code
OX3 9DU
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Undecided

Learn more about this trial

Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD

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