A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD)

Phase 3

Completed

• Conditions: Duchenne disease; Duchenne Muscular Dystrophy; 10028396

• Registration Number: NL-OMON54339
• Lead Sponsor: FibroGen, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 2024-04-09

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 2

Inclusion Criteria

Subjects must meet all of the following criteria in order to be eligible for
the study:

Age, Consent, and Contraception
1. Males at least 12 years of age, non-ambulatory at screening initiation
2. Written consent by patient and/or legal guardian as per regional/ country
and/or IRB/IEC requirements
3. Male subjects with partners of childbearing potential must use contraception
during the conduct of the study, and for 12 weeks after the last dose of study
drug.

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

Performance criteria:
5. Brooke Score for Arms and Shoulders <=5
6. Able to undergo MRI test for the upper arm extremities (Biceps Brachii
muscle) and cardiac muscle
7. Able to perform spirometry

Pulmonary and Cardiac criteria:
8. Average (of Screening and Day 0) percent predicted FVC between 45 and 85,
inclusive
9. Left ventricular ejection fraction >=50% as determined by local cardiac MRI
read at screening or within 3 months prior to randomization (Day 0)
10. If subjects have a history of cardiomyopathy, then subject must be on a
stable dose of cardiomyopathy/ heart failure medications (e.g., angiotensin
converting enzyme inhibitors, aldosterone receptors blockers,
angiotensin-receptor blockers, and beta- blockers) for at least 1 month prior
to screening. If subjects have no diagnosis of cardiomyopathy, then no dose of
cardiomyopathy/heart failure medication is required for eligibility.
11. 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.

Vaccination:
12. Agreement to receive annual influenza vaccinations during the course of the
study.

Laboratory criteria:
13. Adequate renal function: cystatin C <=1.4 mg/L
14. Adequate hematology and electrolytes parameters:
a. Platelets >100,000/mcL
b. Hemoglobin >12 g/dL
c. Absolute neutrophil count >1500 /µL
d. Serum calcium (Ca), potassium (K), sodium (Na), magnesium (Mg) and phosphorus
(P) levels are within a clinically accepted range for DMD patients./
15. Adequate hepatic function:
a. No history or evidence of liver disease
b. Gamma glutamyl transferase (GGT) <=3x upper limit of normal (ULN)
c. Total bilirubin <=1.5xULN

Exclusion Criteria

Subjects must not meet any of the following criteria in order to be eligible:

General criteria:
1. Previous exposure to pamrevlumab
2. BMI >=40 kg/m2 or weight >117 kg
3. History of
a. allergic or anaphylactic reaction to human, humanized, chimeric or murine
monoclonal antibodies
b. hypersensitivity to study drug or any component of study drug
c. hypersensitivity reaction to Gadolinium-based Constrast Agens (GBCA)
required for MRI acquisition
4. 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
(exondys 51), ataluren, golodirsen (vyondys 53), casimersen (amondys 45))
within 5 half-lives of screening, whichever is longer, with the exception of
the systemic corticosteroids, including deflazacort

Cardiac, Renal and Pulmonary assessments:
5. Severe uncontrolled heart failure (NYHA Classes III-IV), or renal
dysfunction, including any of the following:
a. Need for intravenous diuretics or inotropic support within 8 weeks prior to
screening
b. Hospitalization for a heart failure exacerbation or arrhythmia within 8
weeks prior to screening
c. Patients with glomerular filtration rate (GFR) of less than 30 mL/min/1.73m2
or with other evidence of acute kidney injury as determined by investigator
6. Arrhythmia requiring anti-arrhythmic therapy
7. Requires >=16 hours continuous ventilation
8. Hospitalization due to respiratory failure within the 8 weeks prior to
screening
9. 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

Clinical judgments:
10. 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

Study & Design

• Study Type: Interventional
• Study Design: Not specified