Taldefgrobep Alfa for Spinal Muscular Atrophy
(RESILIENT Trial)

Recruiting in Palo Alto (17 mi)

+66 other locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Biohaven Pharmaceuticals, Inc.

Stay on Your Current Meds

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This trial will test if taldefgrobep alfa can help people with SMA who are already on other treatments. The drug works by blocking a protein that stops muscle growth, potentially making muscles stronger and improving movement. Taldefgrobep alfa has shown promise in increasing muscle mass and function in SMA patients.

Research Team

Lindsey Lair, MD

Principal Investigator

Biohaven Pharmaceuticals, Inc.

Eligibility Criteria

This trial is for people with Spinal Muscular Atrophy who are already on a stable dose of specific SMA treatments like nusinersen or risdiplam, or have had gene therapy. Participants must weigh at least 15kg and can be ambulant or non-ambulant. Those needing daytime ventilation, recent spinal fusion patients, and those with certain implants or previous anti-myostatin therapies cannot join.

Inclusion Criteria

I can walk with or without assistance.

I have been genetically diagnosed with Spinal Muscular Atrophy.

I am currently on a stable dose of SMA treatment and plan to stay on it.

Exclusion Criteria

I need a ventilator to breathe during the day.

I weigh at least 15kg.

I had spinal fusion surgery less than 6 months ago, but adjustments to MAGEC rods are okay.

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Treatment Details

Interventions

Taldefgrobep Alfa (Protein Replacement Therapy)

Trial OverviewThe study tests taldefgrobep alfa's effectiveness and safety as an additional treatment for Spinal Muscular Atrophy alongside existing medications compared to a placebo. It aims to see if adding taldefgrobep alfa improves patient outcomes.

Participant Groups

2Treatment groups

Experimental Treatment

Placebo Group

Group I: taldefgrobep alfaExperimental Treatment1 Intervention

taldefgrobep alfa - Double-blind (DB) Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week DB phase. taldefgrobep alfa/taldefgrobep alfa - Extension Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week Open label Extension (OLE) phase.

Group II: PlaceboPlacebo Group2 Interventions

Placebo - Double-blind (DB) Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week DB phase. Placebo/taldefgrobep alfa - Extension Phase: Participants who receive placebo during DB phase, receive weight based 35 mg/50 mg weekly subcutaneous taldefgrobep alfa injection for 48-week OLE phase.