Spinal Muscular Atrophy
Conditions
Brief summary
Change from baseline in the MFM-32 at Week 48
Detailed description
Change from baseline in the RULM at Week 48, Change from baseline in the RHS at Week 48, Safety and tolerability assessments including change in lean body mass and bone mineral density on DXA scan from baseline at Week 48 and Tanner staging for puberty monitoring, monitoring of injection acceptability assessments, and frequency of unique subjects with: new or worsening lab abnormalities, treatment related adverse events, serious adverse events, and adverse events leading to discontinuation., Trough plasma concentrations of taldefgrobep alfa and pharmacokinetic parameters estimated with population PK modeling
Interventions
DRUGTaldefgrobep
DRUGPlacebo Solution for injection in pre-filled syringe
Sponsors
Biohaven Pharmaceuticals Inc., Biohaven Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in the MFM-32 at Week 48 | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in the RULM at Week 48, Change from baseline in the RHS at Week 48, Safety and tolerability assessments including change in lean body mass and bone mineral density on DXA scan from baseline at Week 48 and Tanner staging for puberty monitoring, monitoring of injection acceptability assessments, and frequency of unique subjects with: new or worsening lab abnormalities, treatment related adverse events, serious adverse events, and adverse events leading to discontinuation., Trough plasma concentrations of taldefgrobep alfa and pharmacokinetic parameters estimated with population PK modeling | — |
Countries
Belgium, Czechia, Germany, Italy, Netherlands, Poland, Spain
Outcome results
None listed