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 ]

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. Safety assessments will be performed at each site visit and will include physical examinations, ECGs, vital signs, standard clinical laboratory evaluations (hematology, blood chemistry, urinalysis) adverse event and serious adverse event monitoring of 14 patients will be evaluated.


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