Phase 2
N=37
Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy
Spinal and Bulbar Muscular Atrophy
Bottom Line
View on ClinicalTrials.gov: NCT02024932 ↗Enrolled (actual)
37
Serious AEs
0.0%
Results posted
Aug 2017
Primary outcome: Primary: Number of Patients With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths as a Measure of Safety and Tolerability — 2; 4; 2; 1 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- BVS857 (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- Male
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Apr 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths as a Measure of Safety and Tolerability |
2; 4; 2; 1; 17; 8 | — |
| PRIMARY Number of Mild, Moderate and Severe Adverse Events as a Measure of Safety and Tolerability |
1; 2; 2; 0; 5; 4 | — |
| PRIMARY Mean Percent Change From Baseline in Thigh Muscle Volume in Part B, Cohort 5 |
0.0; -3.4 | 0.0164 sig |
| SECONDARY Mean Change From Baseline in Score on the Adult Myopathy Assessment Tool (AMAT) in Part B, Cohort 5 |
1.0; 2.3 | — |
| SECONDARY Mean Change From Baseline in Total Lean Body Mass (LBM) in Part B, Cohort 5 |
0.77; 0.16 | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part A, Cohort 1 |
184; 34.6; 83.1; 74.2 | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part A, Cohort 2 |
393; 77.3; 113; 191; 232 | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857: Time to Reach the Maximum Concentration After Drug Administration (Tmax) in Part A, Cohort 1 |
4.04; 12.1; 18.1; 36.0 | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857: Time to Reach the Maximum Concentration After Drug Administration (Tmax) in Part A, Cohort 2 |
2.53; 24.1; 24.0; 24.0; 48.0 | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) in Part A, Cohort 1 |
4630; 1060; 2720; 5310 | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) in Part A, Cohort 2 |
9850; 6480; 7340; 14400; 28400 | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Plasma Concentration-time Curve From Zero to 48 Hours (AUC0_48h) in Part A, Cohort 1 |
4620; 2120; 2720; 2210 | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Plasma Concentration-time Curve From Zero to 48 Hours (AUC0_48h) in Part A, Cohort 2 |
7980; 2640; 3880; 6390; 7360 | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part B, Cohort 4 |
2490 | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part B, Cohort 5 |
854; 790; 712 | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857: Time to Reach the Maximum Concentration After Drug Administration (Tmax) in Part B, Cohort 4 |
1.08 | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857: Time to Reach the Maximum Concentration After Drug Administration (Tmax) in Part B, Cohort 5 |
2.39; 1.73; 1.49 | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) in Part B, Cohort 5 |
13100; 28000 | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857:The Area Under the Plasma Concentration-time Curve From Zero to 48 Hours (AUC0_48h) in Part B, Cohort 4 |
40600 | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857:The Area Under the Plasma Concentration-time Curve From Zero to 48 Hours (AUC0_48h) in Part B, Cohort 5 |
19400; 19600; 18100 | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to the End of the Dosing Interval Tau (AUCtau) |
— | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857: The Terminal Elimination Half-life (T1/2) |
— | — |
| SECONDARY Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to Infinity (AUCinf) |
— | — |
| SECONDARY Compare Dose Normalized Log-transformed AUCinf Following IV and SC Administrations |
— | — |
Summary
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).
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
Data sourced from ClinicalTrials.gov (NCT02024932). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.