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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04632940




Registration number
NCT04632940
Ethics application status
Date submitted
12/11/2020
Date registered
17/11/2020

Titles & IDs
Public title
Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD
Scientific 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)
Secondary ID [1] 0 0
2020-000699-39
Secondary ID [2] 0 0
FGCL-3019-094
Universal Trial Number (UTN)
Trial acronym
LELANTOS-2
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Pamrevlumab
Treatment: Drugs - Placebo
Treatment: Drugs - Corticosteroids

Experimental: Pamrevlumab - 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

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


Treatment: Drugs: Pamrevlumab
Pamrevlumab will be administered per dose and schedule specified in the arm description.

Treatment: Drugs: Placebo
Placebo will be administered per schedule specified in the arm description.

Treatment: Drugs: Corticosteroids
Systemic deflazacort or equivalent potency of corticosteroids administered orally
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52
Timepoint [1] 0 0
Baseline, Week 52
Secondary outcome [1] 0 0
Change From Baseline in 4-Stair Climb Velocity (4SCV) Assessment at Week 52
Timepoint [1] 0 0
Baseline, Week 52
Secondary outcome [2] 0 0
Change From Baseline in the 10-Meter Walk/Run Test at Week 52
Timepoint [2] 0 0
Baseline, Week 52
Secondary outcome [3] 0 0
Change From Baseline in Time to Stand (TTSTAND) at Week 52
Timepoint [3] 0 0
Baseline, Week 52
Secondary outcome [4] 0 0
Time to Loss of Ambulation (LoA) From Baseline to Week 52
Timepoint [4] 0 0
Baseline to Week 52

Eligibility
Key inclusion criteria
Age, and consent:

1. Males at least 6 to <12 years of age at screening initiation
2. 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:
3. Medical history includes diagnosis of DMD and confirmed Duchenne mutation, including status of exon 44 using a validated genetic test.

Pulmonary criteria:
4. Average (of screening and Day 0) percent predicted forced vital capacity (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 (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:
6. 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.
7. Able to rise (TTSTAND) from floor in <10 seconds (without aids/orthoses) at screening visit.
8. Able to undergo magnetic resonance imaging (MRI) test for the lower extremities vastus lateralis muscle.

Vaccination:
9. Agreement to receive annual influenza vaccinations during the conduct of the study.

Laboratory criteria:
10. Adequate renal function: cystatin C =1.4 mg/liter (L)
11. Adequate hematology and electrolytes parameters:

1. Platelets >100,000/microliter (µL)
2. Hemoglobin >12 grams (g)/deciliter (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 for DMD participants
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
Minimum age
6 Years
Maximum age
11 Years
Sex
Males
Can healthy volunteers participate?
No
Key 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 (for example, 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. Body mass index (BMI) =40 kg/square meter (m^2) or weight >117 kg
5. History of

1. allergic or anaphylactic reaction to human, humanized, chimeric or murine monoclonal antibodies
2. hypersensitivity to study drug or any component of study drug
6. 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:
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 (New York Heart Association [NYHA] Classes III-IV) or renal dysfunction, 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
3. 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
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 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
3/03/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
14/12/2023
Sample size
Target
Accrual to date
Final
73
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Murdoch Children's Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Iowa
Country [8] 0 0
United States of America
State/province [8] 0 0
Kansas
Country [9] 0 0
United States of America
State/province [9] 0 0
Maryland
Country [10] 0 0
United States of America
State/province [10] 0 0
Massachusetts
Country [11] 0 0
United States of America
State/province [11] 0 0
Michigan
Country [12] 0 0
United States of America
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Missouri
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United States of America
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Ohio
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United States of America
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Oregon
Country [15] 0 0
United States of America
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Pennsylvania
Country [16] 0 0
United States of America
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Tennessee
Country [17] 0 0
United States of America
State/province [17] 0 0
Texas
Country [18] 0 0
United States of America
State/province [18] 0 0
Utah
Country [19] 0 0
United States of America
State/province [19] 0 0
Virginia
Country [20] 0 0
United States of America
State/province [20] 0 0
Washington
Country [21] 0 0
United States of America
State/province [21] 0 0
Wisconsin
Country [22] 0 0
Austria
State/province [22] 0 0
Vienna
Country [23] 0 0
Belgium
State/province [23] 0 0
Flemish Brabant
Country [24] 0 0
Belgium
State/province [24] 0 0
Liege
Country [25] 0 0
Belgium
State/province [25] 0 0
Oost-Vlaanderen
Country [26] 0 0
Canada
State/province [26] 0 0
Ontario
Country [27] 0 0
China
State/province [27] 0 0
Chongqing
Country [28] 0 0
China
State/province [28] 0 0
Guangdong
Country [29] 0 0
China
State/province [29] 0 0
Hunan
Country [30] 0 0
China
State/province [30] 0 0
Sichuan
Country [31] 0 0
China
State/province [31] 0 0
Beijing
Country [32] 0 0
France
State/province [32] 0 0
Bas-Rhin
Country [33] 0 0
France
State/province [33] 0 0
Nantes
Country [34] 0 0
France
State/province [34] 0 0
Paris
Country [35] 0 0
Italy
State/province [35] 0 0
Milan
Country [36] 0 0
Italy
State/province [36] 0 0
Bosisio ParIni
Country [37] 0 0
Italy
State/province [37] 0 0
Milano
Country [38] 0 0
Italy
State/province [38] 0 0
Roma
Country [39] 0 0
Netherlands
State/province [39] 0 0
Leiden
Country [40] 0 0
Netherlands
State/province [40] 0 0
Nijmegen
Country [41] 0 0
Spain
State/province [41] 0 0
Barcelona
Country [42] 0 0
Spain
State/province [42] 0 0
Valencia
Country [43] 0 0
United Kingdom
State/province [43] 0 0
England

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
FibroGen
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT04632940