A study to test Taldefgropeb Alfa in patients with Spinal Muscular Atrophy.

Phase 1

• Conditions: Spinal Muscular Atrophy; MedDRA version: 20.1Level: PTClassification code: 10041582Term: Spinal muscular atrophy Class: 100000004850; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: CTIS2024-511852-42-00
• Lead Sponsor: Biohaven Pharmaceuticals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-06-17
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 169

Inclusion Criteria

Spinal Muscular Atrophy confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number, Ambulant or Non-ambulant, Treated with an SMA disease-modifying therapy and anticipated to remain on that same treatment regimen dose throughout the trial including nusinersen, risdiplam and/or history of onasemnogene abeparvovec

Exclusion Criteria

Cannot have previously taken anti-myostatin therapies, Must weigh at least 15 kg, Respiratory insufficiency, defined by the medical necessity for invasive or non-invasive ventilation for daytime treatment while awake (use overnight or during daytime naps is acceptable)., History of Spinal Fusion within 6 months of Screening. MAGEC rod non-surgical adjustments are allowed during the study, Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified