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

Phase 1

• Conditions: Spinal Muscular Atrophy (SMA); MedDRA version: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2022-000193-25-PL
• Lead Sponsor: Biohaven Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2022-09-29
• Last Update Posted: 26 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 180

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 and dose throughout the trial, including the following:
i. a stable regimen of nusinersen for 6 months prior to Screening; and/or
ii. a stable regimen of risdiplam, for 6 months prior to Screening; and/or
iii. a single dose of onasemnogene abeparvovec, received at least 2 years prior to Screening.
Are the trial subjects under 18? yes
Number of subjects for this age range: 191
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 34
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

? Receiving or have received previous administration of anti-myostatin therapies

? Weight <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 or major surgeries within 6 months prior to screening or planned during the study. Non-surgical adjustments are allowed during the study (such as MAGEC rods).

? 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