A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-Label Extension (RESILIENT)

Phase 3

Recruiting

Conditions: RESILIENT
10029299
10028302

Registration Number: NL-OMON53477

Lead Sponsor: Biohaven Pharmaceuticals, Inc

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 8

Inclusion Criteria

* Spinal Muscular Atrophy confirmed by genetic diagnosis of 5qautosomal
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.

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

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Change from baseline in the MFM-32 at Week 48</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Change from baseline in the RULM at Week 48;<br /><br>Change from baseline in the RHS at Week 48;<br /><br>Safety and tolerability assessments including change in lean body mass and bone<br /><br>mineral density on DXA scan from baseline at Week 48 and Tanner staging for<br /><br>puberty monitoring, monitoring of injection acceptability assessments, and<br /><br>frequency of unique subjects with: new or worsening lab abnormalities,<br /><br>treatment related adverse events, serious adverse events, and adverse events<br /><br>leading to discontinuation;<br /><br>Trough plasma concentrations of taldefgrobep alfa and pharmacokinetic<br /><br>parameters estimated with population PK modeling.</p><br>