Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05337553 | A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy | PHASE3 | ACTIVE NOT_RECRUITING | 269 | — | — | Jul 6, 2022 | Dec 1, 2027 | Jun 1, 2026 | 53 | United States, Belgium +7 |
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.
| Arm | Type | Description |
|---|---|---|
| taldefgrobep alfa | EXPERIMENTAL | 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. |
| Placebo | PLACEBO_COMPARATOR | 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. |
| Name | Type | Description |
|---|---|---|
| taldefgrobep alfa | DRUG | DB Phase: 35 mg/50 mg weekly subcutaneous injection |
| Placebo | DRUG | DB Phase: matching placebo 35 mg/50 mg weekly subcutaneous injection |
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 * Treated with an SMA disease-modifying therapy and anticipated to remain on that same treatment regimen and dose throughout the tria...