A Phase 3 interventional study of taldefgrobep alfa and Placebo in Spinal Muscular Atrophy, Neuromuscular Diseases and SMA, sponsored by Biohaven Pharmaceuticals, Inc.. Active, not recruiting at 53 sites in 9 countries. Open to participants aged 4 Years to 21 Years. Per ClinicalTrials.gov, last updated 2026-06-01.
Sponsored by Biohaven Pharmaceuticals, Inc. · Phase 3, Interventional, and Treatment
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 and/or risdiplam and/or have a history of onasemnogene abeparvovec, compared to placebo.
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.
282 studies on the registry are indexed under Muscular Atrophy, Spinal; 73 are open to participants now.
This study's enrollment of 269 is above the median of 29 across 156 interventional studies indexed under Muscular Atrophy, Spinal.
Browse Muscular Atrophy, Spinal studies →Biohaven Pharmaceuticals, Inc. is the lead sponsor of 15 studies on the registry; 1 is open to participants now.
Of its 10 completed or terminated interventional studies of FDA-regulated products, 6 (60%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
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 Open label Extension (OLE) phase.
Drug: taldefgrobep alfa
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 OLE phase.
Drug: Placebo · Drug: taldefgrobep alfa
DB Phase: 35 mg/50 mg weekly subcutaneous injection
Also known as: BHV-2000, BMS-986089
DB Phase: matching placebo 35 mg/50 mg weekly subcutaneous injection
Extension Phase: 35 mg/50 mg weekly subcutaneous injection
Also known as: BHV-2000, BMS-986089
Efficacy of taldefgrobep alfa compared to placebo in change in the 32 item Motor Function Measure (MFM-32) total score
Change in MFM-32 total score from baseline to Week 48. Scores range from 0-3 on each item. The scores from the 32 items are summed and transformed to a 0-100 scale, with higher scores reflecting higher levels of functional abilities.
Time frame: Baseline to Week 48
Efficacy of taldefgrobep alfa compared to placebo in change in the Revised Upper Limb Module (RULM) score
The RULM includes 20 items, graded from 0 to 2, with a maximum score of 37. Higher scores indicate better function.
Time frame: Baseline to Week 48
Efficacy of taldefgrobep alfa compared to placebo in change in the Revised Hammersmith Scale (RHS)
The RHS has a maximum score of 69 points (33 items are scored 0-2, and 3 items scored 0-1). Higher scores indicate better function.
Time frame: Baseline to Week 48
Change from Baseline in lean body mass
Body mass will be measured as change in kg (greater change meaning more improvement)
Time frame: Baseline, Week 48
Change from Baseline in bone mineral density
Bone mineral density will be measured by Z-score change (higher score indicates improvement)
Time frame: Baseline, Week 48
Change from baseline in Tanner staging
Time frame: Baseline, Week 48
Injection acceptability assessments
If local injection site reactions (such as redness, itching, swelling, hardening or bruising) are experienced, the acceptability is scored on a scale of 1-5 with 1 being totally acceptable and 5 being not at all acceptable If pain is experienced at the injection site, pain will be scored on a scale of 1-5 with 1 being totally acceptable and 5 being not at all acceptable
Time frame: Week 48
Number of Participants with new or worsening lab abnormalities, Adverse Events (AEs), Serious AEs (SAEs) and AEs Leading to Study Drug Discontinuation in the Double-blind Phase
Time frame: Up to 48 Weeks
Trough plasma concentration
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48
Plan to share: No
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This study is active, not recruiting, as verified in May 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Taldefgrobep alfa
Biohaven Pharmaceuticals, Inc.