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)

FibroGen, Inc.0 sites70 target enrollmentJanuary 14, 2021

Overview

No summary available.

Registry

who.int

Start Date

January 14, 2021

End Date

TBD

Last Updated

2 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

Male

Sponsor

•Double\-blind phase:
•Age and Consent
•1\. Males at least 6 to \< 12 years of age at screening initiation;
•2\. Written consent by legal guardian as per regional/ country and/or IRB/IEC requirements;
•DMD Diagnosis:
•3\. Medical history includes diagnosis of DMD and confirmed Duchenne mutation using a validated genetic test.
•Pulmonary Criteria:
•4\. Average (of Screening and Day 0\) percent predicted 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 (e.g. prednisone or prednisolone 0\.75 mg/kg per day or deflazacort 0\.9 mg/kg per day) orstable dose. A reasonable expectation is that dosage and dosing regimen would not change significantly for the duration of the study.
•Performance Criteria:

•Both phases:
•General criteria:
•1\. Concurrent illness other than DMD that can cause muscle weakness and/or impairment of motor function.
•2\. Severe intellectual impairment (eg. severe autism, sever cognitive impairment, severe behavioral disturbance) preventing the ability to perform study assessments in the investigator´s judgement.
•3\. Previous exposure to pamrevlumab.
•4\. BMI \=40 kg/m2 or weight \>117 kg.
•5\. History of allergic or anaphylactic reaction to human, humanized, chimeric or murine monoclonal antibodies.
•6\. 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) within 5 half\-lives of screening, whichever is longer, with the exception of the systemic corticosteroids, including deflazacort.
•Cardiac and Pulmonary Criteria:
•7\. Requires \>\= 16 hours continuous ventilation.