NCT04371666

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

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)

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 98 (actual)
• Sponsor: Kyntra Bio · Industry
• Sex: Male
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

This is a global, Phase 3, randomized, double-blind trial of pamrevlumab or placebo in combination with systemic corticosteroids in participants with non-ambulatory Duchenne muscular dystrophy, aged 12 years and older. Approximately 90 male participants will be randomized at a 1:1 ratio to Arm A (pamrevlumab + systemic corticosteroid) or Arm B (placebo+ systemic corticosteroid), respectively. Participants 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. This trial has 3 study periods: * Screening period: Up to 4 weeks * Treatment period: 52 weeks * Safety Follow-up period/End of Study (EOS): A visit 28 days (+/- 3 Days) and a final safety follow-up phone call 60 days (+ 3 Days) after the last dose In the screening period, participants will be evaluated per the protocol inclusion/exclusion criteria to determine eligibility for participation in this trial. During the treatment period, each participant will receive pamrevlumab or placebo at 35 mg/kg every 2 weeks for up to 52 weeks. Participants who complete the 52-week study (either arm) may be eligible for rollover into an open-label extension treatment (OLE) with pamrevlumab + systemic corticosteroids. Participants who discontinue study treatment for any reason should be encouraged to return to the investigative site to complete final safety and efficacy assessments.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Pamrevlumab	Pamrevlumab per dose and schedule specified in the arm description
DRUG	Placebo	Matching placebo per schedule specified in the arm description
DRUG	Corticosteroids	Systemic deflazacort or equivalent potency of corticosteroids administered orally

Timeline

• Start date: 2020-08-10
• Primary completion: 2023-02-13
• Completion: 2023-08-17
• First posted: 2020-05-01
• Last updated: 2024-03-12
• Results posted: 2024-03-12

Locations

53 sites across 14 countries: United States, Australia, Austria, Belgium, Canada, China, Czechia, France, Israel, Italy, Netherlands, Spain, Switzerland, United Kingdom

Regulatory

• FDA-regulated drug study