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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02268552 | An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) | PHASE1 | COMPLETED | 40 | — | — | Apr 2, 2015 | Dec 29, 2022 | Apr 9, 2025 | 14 | Belgium, Bulgaria +5 |
A DLT is defined as an adverse event or abnormal laboratory value assessed as unrelated to disease, disease progression, inter-current illness, or concomitant therapies that occurs within the first 14 days of treatment with LMI070 and meets any of the criteria for blood and lymphatic system disorders, gastrointestinal disorders, investigations and other toxicities considered clinically significant.
TEAEs are defined as adverse events starting on or after the first dose of study treatment that were absent pre-treatment, or events present prior to the first dose but increased in severity after the first dose. Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post last treatment.
| Arm | Type | Description |
|---|---|---|
| branaplam | EXPERIMENTAL | branaplam Treatment |
| Name | Type | Description |
|---|---|---|
| branaplam | DRUG | - |
Inclusion Criteria: Common for both Parts 1 and 2: * Type 1 SMA, diagnosed clinically, with symptom onset \<6 months of age and genetic confirmation of mutations in both alleles of the SMN1 gene, and with SMN2 copy number of 2. * Best supportive care in place and stable for at least 14 days before...