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

(LELANTOS-1 Trial)

Recruiting at55 trial locations

Age: Any Age

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: FibroGen

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This trial tests pamrevlumab with steroids in patients aged 12+ who have severe Duchenne muscular dystrophy and can't walk. The drug aims to reduce muscle damage by blocking a harmful protein.

Research Team

Eligibility Criteria

Inclusion Criteria

Written consent by participant and/or legal guardian as per regional/country and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements

Male participants 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

Left ventricular ejection fraction ≥50% as determined by local cardiac MRI read at screening or within 3 months prior to randomization (Day 0)

Exclusion Criteria

You have taken pamrevlumab before.

You have had a severe allergic reaction to certain types of antibodies used in treatments.

BMI ≥40 kg/square meter (m^2) or weight >117 kg

Treatment Details

Interventions

• Corticosteroids (Corticosteroid)
• Pamrevlumab (Monoclonal Antibody)
• Placebo (Other)

Participant Groups

Group I: Pamrevlumab Experimental Treatment 2 Interventions

Pamrevlumab 35 milligrams (mg)/kilogram (kg) intravenously (IV) every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks

Group II: Placebo Placebo Group 2 Interventions

Matching placebo IV every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks

Who Is Running the Clinical Trial?

FibroGen

Lead Sponsor

Trials

60

Recruited

15,400+

Thane Wettig

FibroGen

Chief Executive Officer since 2023

Bachelor's degree in Mechanical Engineering from Case Western Reserve University, MBA from Duke University

Dr. Mark Eisner

FibroGen

Chief Medical Officer since 2020

MD from Harvard Medical School