A Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD)

  • STATUS
    Recruiting
  • End date
    Sep 30, 2022
  • participants needed
    90
  • sponsor
    FibroGen
Updated on 4 September 2021
corticosteroids
calcium
total bilirubin
absolute neutrophil count
ejection fraction
prednisone
electrolytes
aldosterone
potassium
cardiomyopathy
prednisolone
pneumococcal polysaccharide vaccine
drug test
muscular dystrophy
pneumococcal vaccine
deflazacort
gamma glutamyl transferase
pamrevlumab
duchenne muscular dystrophy
serum electrolyte

Summary

To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in subjects with non-ambulatory Duchenne muscular dystrophy (age 12 years and older).

Description

This is a global, randomized, double-blind trial of pamrevlumab or placebo in combination with systemic corticosteroids in subjects with non-ambulatory Duchenne muscular dystrophy, aged 12 years and older. Approximately 90 male subjects will be randomized at a 1:1 ratio to Arm A (pamrevlumab + systemic corticosteroid) or Arm B (placebo+ systemic corticosteroid), respectively.

Subjects must be fully informed of the potential benefits of approved products and make an informed decision that they prefer to participate in a clinical trial in which they could be randomized to placebo.

Subjects will be randomized in a 1:1 ratio to one of the two study treatment arms; pamrevlumab or placebo in combination with systemic steroids.

This trial has three study periods:

  • Screening period: Up to 4 weeks
  • Treatment period: 52 weeks
  • Safety Follow-up period/final assessment: 4 weeks (Week 56 (+/-3 days))

In the screening period, subjects will be evaluated per the protocol inclusion/exclusion criteria to determine eligibility for participation in this trial.

During the treatment period, each subject will receive pamrevlumab or placebo at 35 mg/kg every 2 weeks for up to 52 weeks.

Subjects who complete the 52-week study (either arm) may be eligible for rollover into an open-label extension treatment (OLE) with pamrevlumab + systemic corticosteroids.

Subjects who discontinue study treatment for any reason should be encouraged to return to the investigative site to complete final safety and efficacy assessments.

Details
Condition Muscular Dystrophy, DUCHENNE MUSCULAR DYSTROPHY
Treatment Placebo, Pamrevlumab
Clinical Study IdentifierNCT04371666
SponsorFibroGen
Last Modified on4 September 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Males at least 12 years of age, non-ambulatory at screening initiation
Written consent by patient and/or legal guardian as per regional/ country and/or IRB/IEC requirements
Male subjects with partners of childbearing potential must use contraception during the conduct of the study, and for 3 months after the last dose of study drug
Medical history includes diagnosis of DMD and confirmed Duchenne mutation using a validated genetic test
Brooke Score for Arms and Shoulders 5
Able to undergo MRI test for the upper arm extremities (Biceps Brachii muscle) and cardiac muscle
Able to perform spirometry
Average (of Screening and Day 0) percent predicted FVC between 45 and 85, inclusive
Left ventricular ejection fraction 50% as determined by cardiac MRI at screening or within 3 months prior to randomization (Day 0)
Prior diagnosis of cardiomyopathy, subjects must be on a stable regimen dose for cardiomyopathy/ heart failure medications (e.g., angiotensin converting enzyme inhibitors, aldosterone receptors blockers, angiotensin-receptor blockers, and betablockers) for at least 1 month prior to screening
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
Received pneumococcal vaccine (PPSV23) (or any other pneumococcal polysaccharide vaccine as per national recommendations) and is receiving annual influenza vaccinations
Adequate renal function: cystatin C 1.4 mg/L
Adequate hematology and electrolytes parameters
Platelets >100,000/mcL
Hemoglobin >12 g/dL
Absolute neutrophil count >1500 /L
Serum calcium (Ca), potassium (K), sodium (Na), magnesium (Mg) and phosphorus (P) levels are within a clinically accepted range
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

Previous exposure to pamrevlumab
BMI 40 kg/m2 or weight >117 kg
History of allergic or anaphylactic reaction to human, humanized, chimeric or murine monoclonal antibodies
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
Severe uncontrolled heart failure (NYHA Classes III-IV), 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
Arrhythmia requiring anti-arrhythmic therapy
Requires 16 hours continuous ventilation
Hospitalization due to respiratory failure within the 8 weeks prior to screening
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
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 or psychiatric conditions
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