G12
Conditions
Interventions
Group 1: The to be tested IMP Taledefgrobep Alfa 35mg/50mg (depending on body weight) over 48 weeks with weekly subcutaneous injections
Group 2: Placebo over a time period of 48 weeks with weekly subc
Sponsors
Biohaven Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Age
4 Years to 21 Years
Inclusion criteria
Inclusion criteria: 1. Signed Written Informed Consent 2. Participants must agree to provide all requested demographic information (i.e. gender, race). Spinal Muscular Atrophy confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number (genetic diagnosis and SMN2 copy number confirmed with documentation either prior to enrollment from previous testing or confirmed during study)
Exclusion criteria
Exclusion criteria: 1. Participation in any other investigational clinical trial while participating in this clinical trial with the exception of registry and natural history studies that do not involve procedures that could impact blinding (such as drawing blood for biomarkers). Also see exclusion 4.j regarding investigational drug trials. 2. Receiving or have received previous administration of anti-myostatin therapies 3. Weight <15 kg 4. Medical History and Concurrent Diseases a. 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) b. Hospitalization for a pulmonary event within the last 2 months or planned hospitalization at the time of screening c. History of spinal fusion within 6 months prior to screening. Note: MAGEC rod nonsurgical adjustments are allowed during the study. ... 5. Any major illness within one month before the screening examination or any febrile illness within one week prior to screening and up to first dose administration. 6. All vaccines should not be given within 2 weeks of the first dose. 7. The participant has a history of intolerance to venipuncture and/or difficult venipuncture access. 8. ECG and laboratory test findings: Clinically significant abnormality identified on the medical or laboratory evaluation. A participant with a clinical abnormality or laboratory parameters outside the reference range may be included only if the investigator considers the finding not clinically significant, that it will not introduce additional risk factors, nor interfere with the studyprocedures. ...
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 (MFM32) total score between Baseline and Week 48. | — |
Secondary
| Measure | Time frame |
|---|---|
| • 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 by new or worsening lab abnormalities, treatment related adverse events (AE’s) and AE’s leading to discontinuation. | — |
Countries
Belgium, France, Italy, Netherlands, Poland, Spain, United Kingdom, United States
Contacts
Public Contactl. Donahue
Biohaven Pharmaceuticals, Inc.
Outcome results
None listed