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269 Participants Needed

Taldefgrobep Alfa for Spinal Muscular Atrophy
(RESILIENT Trial)

Recruiting at 66 trial locations

Overseen By

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: Biohaven Pharmaceuticals, Inc.

Must be taking: Nusinersen, Risdiplam, Onasemnogene

Must not be taking: Anti-myostatin therapies

Disqualifiers: Respiratory insufficiency, Spinal fusion, others

Stay on Your Current MedsYou can continue your current medications while participating

Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval

Prior Safety DataThis treatment has passed at least one previous human trial

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.

Will I have to stop taking my current medications?

No, you will not have to stop taking your current medications. The trial requires that you continue your existing treatment with nusinersen, risdiplam, or have a history of onasemnogene abeparvovec.

How does the drug Taldefgrobep Alfa differ from other treatments for spinal muscular atrophy?

Taldefgrobep Alfa is unique because it targets myostatin, a protein that limits muscle growth, to potentially increase muscle mass and strength in patients with spinal muscular atrophy. This approach is different from existing treatments that focus on increasing SMN protein levels.¹ ² ³ ⁴ ⁵

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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Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Double-blind Treatment

Participants receive weight-based 35 mg/50 mg weekly subcutaneous injection of taldefgrobep alfa or placebo for 48 weeks

48 weeks

Open-label Extension

Participants receive weight-based 35 mg/50 mg weekly subcutaneous injection of taldefgrobep alfa for 48 weeks

48 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

Taldefgrobep Alfa

Trial Overview The 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.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Biohaven Pharmaceuticals, Inc.

Lead Sponsor

Trials

Recruited

30,100+

Findings from Research

In a study of 21 infants with spinal muscular atrophy (SMA) treated with onasemnogene abeparvovec, 76% achieved at least one World Health Organization motor milestone, indicating significant efficacy in improving motor function.

All children experienced transient side effects, with vomiting being universal and moderate to severe transaminitis occurring more frequently in infants weighing 8 kg or more, highlighting the importance of monitoring and individualized management for safety.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy.D'Silva, AM., Holland, S., Kariyawasam, D., et al.[2022]

In a phase 3 trial involving 22 infants with spinal muscular atrophy type 1, 59% achieved independent sitting for 30 seconds or longer by 18 months, compared to 0% in an untreated cohort, demonstrating significant efficacy of the gene therapy onasemnogene abeparvovec.

91% of treated patients survived without the need for permanent ventilation by 14 months, highlighting the therapy's potential to improve survival outcomes compared to only 26% in the untreated group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial.Day, JW., Finkel, RS., Chiriboga, CA., et al.[2021]