Clinical Trial: Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Two-part Placebo-controlled Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)
Brief Summary: The purpose of this study is to determine if BVS857 is safe, tolerable and increases thigh muscle thickness in patients with spinal bulbar and muscular atrophy (SBMA).
Detailed Summary:
Sponsor: Novartis Pharmaceuticals
Current Primary Outcome:
- Number of patients with adverse events as a measure of safety and tolerability [ Time Frame: After 78 days in Part A and after 85 days in Part B. ]
- Number of mild, moderate and severe adverse events as a measure of safety and tolerability [ Time Frame: After 78 days in Part A and after 85 days in Part B. ]
- Change in thigh muscle volume [ Time Frame: Baseline and Day 85 in Part B. ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Change in score on the adult myopathy assessment tool. [ Time Frame: Baseline and Day 85 in Part B. ]
- Change in lean body mass. [ Time Frame: Baseline and Day 85 in Part B. ]
- Plasma Pharmacokinetics (PK) of BVS857: Observed maximum concentration following drug administration (Cmax) [ Time Frame: Part A: days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose. ]
- Plasma Pharmacokinetics (PK) of BVS857: Time to reach the maximum concentration after drug administration (Tmax) [ Time Frame: Part A: days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose. ]
- 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) [ Time Frame: Part A: days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose. ]
- 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) [ Time Frame: Part A: days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose. ]
- Plasma Pharmacokinetics (PK) of BVS857: The area under the serum concentration-time curve from time zero to infinity (AUCinf) [ Time Frame: Part A: days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose. ]
- Plasma Pharmacokinetics (PK) of BVS857: The terminal elimination half-life (T1/2) [ Time Frame: Part A: days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose. ]
- Compare dose normalized log-transformed AUCinf following IV and SC administrations. [ Time Frame: In Part A: days 1 and 15, pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. ]
Original Secondary Outcome:
- Change in score on the adult myopathy assessment tool. [ Time Frame: Baseline and Day 85 in Part B. ]
- Change in lean body mass. [ Time Frame: Baseline and Day 85 in Part B. ]
- Plasma Pharmacokinetics (PK) of BVS857: Observed maximum concentration following drug administration (Cmax) [ Time Frame: Part A: days 1, 15, 29, 43, pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. On day 57, pre-dose. 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36, pre-dose, 4, 24, 48 hours post-dose. On day 78, pre-dose, 4, 24, 48, 168 hours post-dose. ]
- Plasma Pharmacokinetics (PK) of BVS857: Time to reach the maximum concentration after drug administration (Tmax) [ Time Frame: Part A: days 1, 15, 29, 43, pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. On day 57, pre-dose. 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36, pre-dose, 4, 24, 48 hours post-dose. On day 78, pre-dose, 4, 24, 48, 168 hours post-dose. ]
- 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) [ Time Frame: Part A: days 1, 15, 29, 43, pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. On day 57, pre-dose. 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36, pre-dose, 4, 24, 48 hours post-dose. On day 78, pre-dose, 4, 24, 48, 168 hours post-dose. ]
- 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) [ Time Frame: Part A: days 1, 15, 29, 43, pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. On day 57, pre-dose. 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36, pre-dose, 4, 24, 48 hours post-dose. On day 78, pre-dose, 4, 24, 48, 168 hours post-dose. ]
- Plasma Pharmacokinetics (PK) of BVS857: The area under the serum concentration-time curve from time zero to infinity (AUCinf) [ Time Frame: Part A: days 1, 15, 29, 43, pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. On day 57, pre-dose. 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36, pre-dose, 4, 24, 48 hours post-dose. On day 78, pre-dose, 4, 24, 48, 168 hours post-dose. ]
- Plasma Pharmacokinetics (PK) of BVS857: The terminal elimination half-life (T1/2) [ Time Frame: Part A: days 1, 15, 29, 43, pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. On day 57, pre-dose. 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36, pre-dose, 4, 24, 48 hours post-dose. On day 78, pre-dose, 4, 24, 48, 168 hours post-dose. ]
- Compare dose normalized log-transformed AUCinf following IV and SC administrations. [ Time Frame: In Part A: days 1 and 15, pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. ]
Information By: Novartis
Dates:
Date Received: December 29, 2013
Date Started: February 2014
Date Completion:
Last Updated: January 17, 2017
Last Verified: January 2017