Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy
Spinal and Bulbar Muscular Atrophy
About this trial
This is an interventional treatment trial for Spinal and Bulbar Muscular Atrophy
Eligibility Criteria
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
- Treatment with systemic steroids > 10 mg/day (or equivalent dose); androgens or androgen reducing agents; systemic beta agonists; or other muscle anabolic drugs within the previous 3 months
- History of cancer, other than non-melanomatous skin cancer
- Retinopathy
- Papilledema Other protocol defined inclusion/exclusion criteria may apply
Sites / Locations
- Novartis Investigative Site
- National Institutes of Health
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm 6
Experimental
Experimental
Placebo Comparator
Experimental
Experimental
Placebo Comparator
BVS857 Part A Open label (Cohort 1)
BVS857 Part A double blind (Cohort 2)
Placebo Part A double blind (Cohort 2)
BVS857 Part B open-label (Cohort 4)
BVS857 Part B double blind (Cohort 5)
Placebo Part B double blind (Cohort 5)
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.
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.)
Participants received single doses of matching placebo i.v. on day 1 and matching placebo s.c. on days 15, 29, 43 and 57.
Participants received 0.1 mg/kg BVS857 i.v. weekly for 12 weeks.
Participants received 0.06 mg/kg (maximum 6 mg) BVS857 i.v. weekly for 12 weeks.
Participants received matching placebo i.v. to BVS857 weekly for 12 weeks.