Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy

Novartis

Status and phase: Completed; Phase 2

Conditions: Spinal and Bulbar Muscular Atrophy

Treatments: Drug: Placebo; Drug: BVS857

Study type: Interventional

Funder types: Industry

Identifiers

NCT02024932

CBVS857X2202

Details and patient eligibility

About

The purpose of this study was to determine if BVS857 is safe, tolerable and increases thigh muscle thickness in patients with spinal bulbar and muscular atrophy (SBMA).

Enrollment: 37 patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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37 participants in 6 patient groups, including a placebo group

BVS857 Part A Open label (Cohort 1)

Experimental group

Description:

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.

Treatment:

Drug: BVS857

BVS857 Part A double blind (Cohort 2)

Experimental group

Description:

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.)

Treatment:

Drug: BVS857

Placebo Part A double blind (Cohort 2)

Placebo Comparator group

Description:

Participants received single doses of matching placebo i.v. on day 1 and matching placebo s.c. on days 15, 29, 43 and 57.

Treatment:

Drug: Placebo

BVS857 Part B open-label (Cohort 4)

Experimental group

Description:

Participants received 0.1 mg/kg BVS857 i.v. weekly for 12 weeks.

Treatment:

Drug: BVS857

BVS857 Part B double blind (Cohort 5)

Experimental group

Description:

Participants received 0.06 mg/kg (maximum 6 mg) BVS857 i.v. weekly for 12 weeks.

Treatment:

Drug: BVS857

Placebo Part B double blind (Cohort 5)

Placebo Comparator group

Description:

Participants received matching placebo i.v. to BVS857 weekly for 12 weeks.

Treatment:

Drug: Placebo