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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT04632940
Registration number
NCT04632940
Ethics application status
Date submitted
12/11/2020
Date registered
17/11/2020
Date last updated
3/06/2024
Titles & IDs
Public title
Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD
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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)
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Secondary ID [1]
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2020-000699-39
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Secondary ID [2]
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FGCL-3019-094
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Universal Trial Number (UTN)
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Trial acronym
LELANTOS-2
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy
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Condition category
Condition code
Musculoskeletal
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Other muscular and skeletal disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Neurological
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Other neurological disorders
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Intervention/exposure
Study type
Interventional
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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
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52
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Assessment method [1]
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Timepoint [1]
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Baseline, Week 52
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Secondary outcome [1]
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Change From Baseline in 4-Stair Climb Velocity (4SCV) Assessment at Week 52
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Assessment method [1]
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Timepoint [1]
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Baseline, Week 52
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Secondary outcome [2]
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Change From Baseline in the 10-Meter Walk/Run Test at Week 52
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Assessment method [2]
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Timepoint [2]
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Baseline, Week 52
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Secondary outcome [3]
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Change From Baseline in Time to Stand (TTSTAND) at Week 52
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Assessment method [3]
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Timepoint [3]
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Baseline, Week 52
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Secondary outcome [4]
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Time to Loss of Ambulation (LoA) From Baseline to Week 52
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Assessment method [4]
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Timepoint [4]
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Baseline to Week 52
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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
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Minimum age
6
Years
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Maximum age
11
Years
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Sex
Males
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Can healthy volunteers participate?
No
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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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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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
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Terminated
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
11/12/2020
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
12/12/2023
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Sample size
Target
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Accrual to date
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Final
73
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Murdoch Children's Research Institute - Parkville
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Recruitment postcode(s) [1]
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3052 - Parkville
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Recruitment outside Australia
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United States of America
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Arkansas
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California
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Colorado
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Nantes
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
FibroGen
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Address
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Ethics approval
Ethics application status
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Summary
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).
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Trial website
https://clinicaltrials.gov/ct2/show/NCT04632940
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Public notes
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Contacts
Principal investigator
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT04632940
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