A Phase 3 Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)

  • STATUS
    Recruiting
  • End date
    Mar 31, 2023
  • participants needed
    70
  • sponsor
    FibroGen
Updated on 8 September 2021
corticosteroids
calcium
prednisolone
duchenne muscular dystrophy
serum electrolyte

Summary

To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every two weeks in ambulatory subjects with Duchenne muscular dystrophy (age 6 to <12 years).

Description

This is a global, randomized, double-blind, trial of pamrevlumab or placebo in combination with systemic corticosteroids in subjects with Duchenne muscular dystrophy, aged 6 to <12 years (ambulatory subjects only). Approximately 70 subjects 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.

Subjects 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.

Subjects will be randomized in a 1:1 ratio to one of the two study treatment arms; pamrevlumab or placebo. Randomization will be stratified by exon 44 deletion.

The main study has three 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 subject will receive pamrevlumab or placebo at 35 mg/kg every 2 weeks for up to 52 weeks. Subjects who complete 52 weeks of treatment may be eligible for an open-label extension (OLE), offering extended treatment with pamrevlumab.

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 IdentifierNCT04632940
SponsorFibroGen
Last Modified on8 September 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Age, and consent
Males at least 6 to <12 years of age at screening initiation
Written consent by legal guardian as per regional/ country and/or IRB/IEC requirements
DMD diagnosis
\. Medical history includes diagnosis of DMD and confirmed Duchenne mutation
using a validated genetic test
Pulmonary criteria
\. Average (of screening and day 0) percent predicted 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 (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
\. 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
\. Able to rise (TTSTAND) from floor in <10 seconds (without aids/orthoses)
at screening visit
\. Able to undergo MRI test for the lower extremities vastus lateralis
muscle
Vaccination
\. Received pneumococcal vaccine (PPSV23) (or any other pneumococcal polysaccharide vaccine as per national recommendations) and is receiving annual influenza vaccinations
Laboratory criteria
\. 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

General Criteria
Concurrent illness other than DMD that can cause muscle weakness and/or impairment of motor function
Severe intellectual impairment (eg, severe autism, severe cognitive impairment, severe behavioral disturbances) preventing the ability to perform study assessments in the Investigator's judgment
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
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 (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
Any other evidence of clinically significant structural or functional heart abnormality
Clinical judgment
\. 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
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