RESILIENT: A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
Study Details
Study Description
Brief Summary
This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of nusinersen or risdiplam or have a history of onasemnogene abeparvovec-xioi, compared to placebo.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
Detailed Description
Myostatin is a negative regulator of muscle growth. Blocking myostatin activity has been shown to increase muscle size and function. Taldefgrobep alfa directly blocks myostatin activity and was well tolerated in other clinical studies. In combination with medications that increase the amount of SMN protein in the body, taldefgrobep alfa has the potential to further improve motor function and clinical measures for people living with SMA.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: taldefgrobep alfa taldefgrobep alfa - Double-blind (DB) Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week DB phase. taldefgrobep alfa/taldefgrobep alfa - Extension Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week Open label Extension (OLE) phase. |
Drug: tadefgrobep alfa
DB Phase: 35 mg/50 mg weekly subcutaneous injection
Other Names:
Drug: tadefgrobep alfa
Extension Phase: 35 mg/50 mg weekly subcutaneous injection
Other Names:
|
| Placebo Comparator: Placebo Placebo - Double-blind (DB) Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week DB phase. Placebo/taldefgrobep alfa - Extension Phase: Participants who receive placebo during DB phase, receive weight based 35 mg/50 mg weekly subcutaneous taldefgrobep alfa injection for 48-week OLE phase. |
Drug: Placebo
DB Phase: matching placebo 35 mg/50 mg weekly subcutaneous injection
Drug: tadefgrobep alfa
Extension Phase: 35 mg/50 mg weekly subcutaneous injection
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Efficacy of taldefgrobep alfa compared to placebo in change in the 32 item Motor Function Measure (MFM-32) total score [Baseline to Week 48]
Change in MFM-32 total score from baseline to Week 48. Scores range from 0-3 on each item. The higher the score, indicates higher functioning.
Eligibility Criteria
Criteria
Key Inclusion Criteria:
-
Spinal Muscular Atrophy confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number
-
Ambulant or Non-Ambulant
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Treated with an SMA disease-modifying therapy and anticipated to remain on that same treatment regimen and dose throughout the trial including nusinersen, risdiplam, and/or a history of onasemnogene abeparvovec
Key Exclusion Criteria:
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Cannot have previously taken anti-myostatin therapies
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Must weigh at least 15kg
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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)
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History of Spinal Fusion within 6 months of Screening. MAGEC rod nonsurgical adjustments are allowed during the study
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Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Rare Disease Research | Atlanta | Georgia | United States | 30329 |
Sponsors and Collaborators
- Biohaven Pharmaceuticals, Inc.
Investigators
- Study Director: Lindsey Lair, MD, Biohaven Pharmaceuticals, Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- BHV2000-301