Clinical Trial: Study of Long-term Safety, Efficacy Tolerability of BYM338 in Patients With Sporadic Inclusion Body Myositis
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: An Open-label, Long-term Study to Evaluate the Safety and Tolerability of BYM338 in Patients With Sporadic Inclusion Body Myositis
Brief Summary: This study is an open-label, long-term study for those patients who participated in the prior proof-of-concept protocol, in which the preliminary efficacy for BYM338 in patients with sIBM was demonstrated after a single 30 mg/kg i.v. dose of BYM338. This study is designed to confirm the efficacy, safety and tolerability of BYM338 in sIBM with long-term dosing.
Detailed Summary: This is a non-confirmatory, multicenter, open-label, non-randomized trial which will extend active treatment to those patients that participated in the preceding proof-of-concept study (CBYM338X2205) in order to collect long-term safety and tolerability data. Up to 14 patients with sIBM will be invited to enroll into the study to receive BYM338 open-label for a period of approximately 3 years or until BYM338 is commercially available, whichever comes first. The study consists of a maximum 28-day screening period, a 5-day baseline period, and a treatment period consisting of the site visits at 4-week intervals for treatment administration, safety and pharmacokinetic follow-up. All patients will be administered a medium level i.v. BYM338 dose regardless of their treatment allocation in the prior study.
Sponsor: Novartis Pharmaceuticals
Current Primary Outcome: Long-Term Safety & Tolerability [ Time Frame: Approx. 3 Years ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Changes in lean body mass from baseline [ Time Frame: Baseline, Week 8, 16, 24, 52, 76, 104, 132, 160 of Treatment ]To assess the effect of multiple doses of BYM338 on lean body mass as measured by DXA in terms of change from baseline.
- Collect pharmacokinetic data from multiple i.v. dosing [ Time Frame: Pre-dose, 30 mins & 4 hours post-dose on Day 1. Pre-dose only on each subsequent administration ]To obtain pharmacokinetic data from multiple i.v. dosing of BYM338 in this patient population.
- Changes in physical function reported by patients [ Time Frame: Baseline, Weeks 16, 24, 52, 76, 104 of Treatment ]- To assess the effect of BYM338 on physical function reported by patients as measured by the sIBM Functional Assessment (sIFA) in terms of change from baseline
- Changes in muscle strength from baseline. [ Time Frame: Baseline, Weeks 16, 24, 52, 76, 104, 132, 160 of Treatment ]- To assess the effect of BYM338 in muscle strength by Quantitative Muscle Testing (QMT) in patients with sIBM in terms of change from baseline.
- Changes in muscle function from baseline. [ Time Frame: Screening, Baseline, Weeks 16, 24, 52, 76, 104, 132, 160 of Treatment ]- To assess the effect of BYM338 on additional muscle function measures (hand-grip and pinch-grip dynamometry and 6 minute walking tests) in terms of change from baseline.
- Changes in thigh muscle volume from baseline [ Time Frame: Baseline, Weeks 8 & 16 of Treatment ]- To assess the effect of BYM338 on thigh muscle volume by MRI in terms of change from baseline.
Original Secondary Outcome: Same as current
Information By: Novartis
Dates:
Date Received: March 20, 2014
Date Started: March 2014
Date Completion:
Last Updated: October 6, 2016
Last Verified: October 2016