Spinal Muscular Atrophy (SMA) MedDRA version: 20.1 Level: PT Classification code 10041582 Term: Spinal muscular atrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Biohaven Pharmaceuticals, Inc
Eligibility
Sex/Gender
All
Inclusion criteria
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: 153 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 27 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of taldefgrobep alfa in participants who are already taking a stable dose of nusinersen or risdiplam or have a history of onasemnogene abeparvovec-xioi, compared to placebo, measured by change in the 32 item Motor Function Measure (MFM-32) total score between Baseline and Week 48;Secondary Objective: - To compare the efficacy of taldefgrobep alfa to placebo using the Revised Upper Limb Module (RULM). -To compare the efficacy of taldefgrobep alfa to placebo using the Revised Hammersmith Scale (RHS). - To assess the safety and tolerability of taldefgrobep alfa as reflected byincluding change from baseline in lean body mass and bone mineral density on DXA scan at Week 48, Tanner staging for puberty monitoring, new or worsening lab abnormalities, injection acceptability assessments, treatment-related adverse events (AEs), serious AEs, and AEs leading to discontinuation. - To assess pharmacokinetic (PK) parameters of taldefgrobep as estimated with population PK modeling.;Primary end point(s): Change from baseline in the MFM-32 at Week 48 ;Timepoint(s) of evaluation of this end point: Double blind phase: Baseline, visit 5, visit 6, visit 7, visit 8 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - 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 ;Timepoint(s) of evaluation of this end point: Double blind phase: RULM: Baseline, visit 5, visit 6, visit 7, visit 8 RHS: Baseline, visit 5, visit 6, visit 7, visit 8 | — |
Countries
Belgium, Czech Republic, France, Germany, Italy, Poland, Spain
Contacts
Public ContactJackie Marin
Biohaven Pharmaceuticals, Inc
Outcome results
None listed