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A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy

NCT05337553 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 269

Last updated 2026-02-04

No results posted yet for this study

Summary

This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of nusinersen and/or risdiplam and/or have a history of onasemnogene abeparvovec, compared to placebo.

Conditions

Interventions

DRUG

taldefgrobep alfa

DB Phase: 35 mg/50 mg weekly subcutaneous injection

DRUG

Placebo

DB Phase: matching placebo 35 mg/50 mg weekly subcutaneous injection

DRUG

taldefgrobep alfa

Extension Phase: 35 mg/50 mg weekly subcutaneous injection

Sponsors & Collaborators

  • Biohaven Pharmaceuticals, Inc.

    lead INDUSTRY

Principal Investigators

  • Lindsey Lair, MD · Biohaven Pharmaceuticals, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
4 Years
Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-07-06
Primary Completion
2024-09-25
Completion
2026-06-30
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Czechia
  • Germany
  • Italy
  • Netherlands
  • Poland
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05337553 on ClinicalTrials.gov