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Taldefgrobep Alfa for Spinal Muscular Atrophy (RESILIENT Trial)

Phase 3
Waitlist Available
Research Sponsored by Biohaven Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Ambulant or Non-Ambulant
Spinal Muscular Atrophy confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to week 48
Awards & highlights

RESILIENT Trial Summary

This trial will study if adding taldefgrobep alfa to current treatments for spinal muscular atrophy (SMA) is effective and safe.

Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
While the side effects of taldefgrobep alfa aren't specified here, similar drugs may cause injection site reactions, muscle pain, headaches, nausea, and potential allergic reactions.

RESILIENT Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I can walk with or without assistance.
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I have been genetically diagnosed with Spinal Muscular Atrophy.
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I am currently on a stable dose of SMA treatment and plan to stay on it.

RESILIENT Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to week 48
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to week 48 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Efficacy of taldefgrobep alfa compared to placebo in change in the 32 item Motor Function Measure (MFM-32) total score

RESILIENT Trial Design

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.

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Who is running the clinical trial?

Biohaven Pharmaceuticals, Inc.Lead Sponsor
47 Previous Clinical Trials
36,983 Total Patients Enrolled
Lindsey Lair, MDStudy DirectorBiohaven Pharmaceuticals, Inc.

Media Library

taldefgrobep alfa Clinical Trial Eligibility Overview. Trial Name: NCT05337553 — Phase 3
Spinal Muscular Atrophy Research Study Groups: taldefgrobep alfa, Placebo
Spinal Muscular Atrophy Clinical Trial 2023: taldefgrobep alfa Highlights & Side Effects. Trial Name: NCT05337553 — Phase 3
taldefgrobep alfa 2023 Treatment Timeline for Medical Study. Trial Name: NCT05337553 — Phase 3
~65 spots leftby Jan 2025
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