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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02024932 | Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy | PHASE2 | COMPLETED | 37 | — | — | Feb 4, 2014 | Apr 13, 2016 | Jan 5, 2021 | 6 | United States, Denmark +2 |
Safety was monitored throughout the study.
Safety was monitored throughout the study.
Thigh muscle volume was assessed by magnetic resonance imaging (MRI). Change from baseline was calculated from the ratio of the post-baseline mean value to the baseline mean value: \[(Day 85/baseline) - 1)\] x 100. A positive change from baseline indicates improvement.
| Arm | Type | Description |
|---|---|---|
| BVS857 Part A Open label (Cohort 1) | EXPERIMENTAL | Participants received single doses of 0.01 mg/kg BVS857 intravenously (i.v.) on day 1, 0.01 mg/kg BVS857 subcutaneously (s.c.) on day 15, 0.03 mg/kg BVS857 s.c. on day 29, 0.06 mg/kg BVS857 s.c. on day 43 and 0.10 mg/kg BVS857 s.c. on day 57. |
| BVS857 Part A double blind (Cohort 2) | EXPERIMENTAL | Participants received single doses of 0.03 mg/kg BVS857 i.v on day 1, 0.03 mg/kg BVS857 s.c. on day 15, 0.06 mg/kg BVS857 s.c. on day 29, 0.10 mg/kg BVS857 s.c. on day 43 and 0.10 mg/kg BVS857 s.c. on day 57. (BVS857 concentrations differed on days 43 and 57.) |
| Placebo Part A double blind (Cohort 2) | PLACEBO_COMPARATOR | Participants received single doses of matching placebo i.v. on day 1 and matching placebo s.c. on days 15, 29, 43 and 57. |
| BVS857 Part B open-label (Cohort 4) | EXPERIMENTAL | Participants received 0.1 mg/kg BVS857 i.v. weekly for 12 weeks. |
| BVS857 Part B double blind (Cohort 5) | EXPERIMENTAL | Participants received 0.06 mg/kg (maximum 6 mg) BVS857 i.v. weekly for 12 weeks. |
| Placebo Part B double blind (Cohort 5) | PLACEBO_COMPARATOR | Participants received matching placebo i.v. to BVS857 weekly for 12 weeks. |
| Name | Type | Description |
|---|---|---|
| BVS857 | DRUG | BVS857 lyophilisate in vial; the lyophilisate was reconstituted with sterile water for injection, diluted as appropriate, and administered either i.v. or s.c.. |
| Placebo | DRUG | Placebo lyophilisate in vial; the lyophilisate was reconstituted with sterile water for injection, diluted as appropriate, and administered either i.v. or s.c.. |
Key Inclusion Criteria: * Genetic diagnosis of SBMA with symptomatic muscle weakness * Able to complete 2 minute timed walk * Serum IGF-1 level less than or equal to 170 ng/mL Key Exclusion Criteria: * Medically treated diabetes mellitus or known history of hypoglycemia * History of Bell's palsy ...