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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07444450 | A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec | PHASE3 | NOT YET_RECRUITING | 42 | — | — | Sep 4, 2026 | Jul 26, 2033 | Jun 5, 2026 | - | — |
| NCT07221669 | A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA) | PHASE3 | RECRUITING | 30 | — | — | Apr 28, 2026 | May 29, 2032 | May 6, 2026 | 2 | United States |
The WHO motor milestones will include six key developmental milestones: sitting without support, standing with assistance, hands-and-knees crawling, walking with assistance, standing alone, and walking alone.
| Arm | Type | Description |
|---|---|---|
| Salanersen | EXPERIMENTAL | Participants will receive a single dose of salanersen, 80 milligrams (mg) administered intrathecally on Day 1 in Part A. In Part B, participants will continue to receive four additional intrathecal doses of salanersen, 80 mg administered on Days 365, 730, 1095, and 1460. |
| Sham Procedure | SHAM_COMPARATOR | Participants will undergo a sham procedure on Day 1 in Part A. In Part B, participants will receive four intrathecal doses of salanersen, 80 mg administered on Days 365, 730, 1095, and 1460. |
| Name | Type | Description |
|---|---|---|
| Salanersen | DRUG | Administered intrathecally |
| Sham Procedure | PROCEDURE | A sham lumbar puncture is a skin-only needle prick at the usual lumbar puncture site. The needle does not enter the spinal canal. |
Key Inclusion Criteria: * Genetic documentation of 5q spinal muscular atrophy (SMA) homozygous gene deletion or mutation or compound heterozygous mutation. * 2 copies of the survival motor neuron 2 (SMN2) gene. * Onasemnogene Abeparvovec (OA) dose given at ≤ 42 days of age and screening initiated l...