Clinical Trial: Efficacy and Safety of Bimagrumab/BYM338 at 52 Weeks on Physical Function, Muscle Strength, Mobility in sIBM Patients

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Randomized, Double-blind, Placebo-controlled, Multicenter, Parallel Group, Dose-finding, Pivotal, Phase 2b/3 Study to Evaluate the Efficacy, Safety, and Tolerability of Intravenous BYM338 at 52 Week

Brief Summary: This study evaluated the efficacy, safety and tolerability of multiple doses of bimagrumab/BYM338 vs placebo, when administered intravenously (i.v.), on physical function, muscle strength, and mobility in patients with sporadic inclusion body myositis (sIBM).

Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: Change From Baseline in 6 Minute Walking Distance (6MWD) Test at Week 52 [ Time Frame: Baseline, Week 52 ]

The 6MWD test measured the distance (in meters) that a participant walked in a 6 minute timeframe. A positive change from baseline indicates improvement.


Original Primary Outcome: Change from Baseline in 6 Minute Walking Distance Test (6MWD) meters to Week 52 [ Time Frame: Baseline, Week 52 ]

Change from baseline to Week 52 in the distance a patient can walk in a set timeframe. 6 Minute Walking Distance Test measures the distance (in meters) that a patient can walk in a 6 minute timeframe.


Current Secondary Outcome:

  • Estimated Within Treatment Group Lean Body Mass (LBM) Ratio at Week 52 [ Time Frame: Baseline, Week 52 ]
    LBM was measured via dual energy x-ray absorptiometry (DXA) and calculated as (LBM at Week 52/LBM at baseline)*100 . A positive change from baseline indicates improvement.
  • Change From Baseline in Quadriceps Quantitative Muscle Testing (QMT) on the Right Side at Week 52 [ Time Frame: Baseline, Week 52 ]
    Quadriceps muscle strength was measured by portable fixed dynamometry (PFD) on the right side. A negative change from baseline indicates deterioration.
  • Change From Baseline in Sporadic Inclusion Body Myositis (sIBM) Functional Assessment (sIFA) Score at Week 52 [ Time Frame: Baseline, Week 52 ]
    Self-reported physical function was assessed by a newly developed patient reported outcome named sporadic inclusion body myositis (sIBM) functional assessment (sIFA). The sIFA consists of 11 items scored on an 11 point numerical rating scale from 0 (no difficulty) to 10 (unable to do) across 3 domains: upper body functioning, lower body functioning and general functioning. Participants completed the assessment where the recall period was the past week prior to completing the patient reported outcome (PRO). The total score on the sIFA scale ranges from 0 (minimum) to 110 (maximum). Higher values represent a worse outcome. A positive change from baseline indicates deterioration.
  • Estimated Annual Number of Falls Per Patient Within Treatment Group [ Time Frame: Week 52 ]
    Participants documented any fall occurrences in a paper diary during the study.
  • Change From Baseline in Short Physical Performance Battery (SPPB) Score at Week 52 [ Time Frame: Baseline, Week 52 ]
    The SPPB evaluated lower extremities function by testing gait speed, ability to keep standing balance and time to rise from a chair five times. The sub-score for each test ranged from 0 to 4. The summary score, which was a summation of scores from the 3 tests, ranged from 0 to 12. An increase in score indicates improvement in physical performance. A negative change from baseline indicates deterioration.


Original Secondary Outcome:

  • Change from Baseline in lean body mass (LBM) at Week 52 [ Time Frame: Baseline, Week 52 ]
    Change from baseline to Week 52 in the total and appendicular lean body mass
  • Change from Baseline in quadriceps Quantitative Muscle Testing (QMT) at Week 52 [ Time Frame: Baseline, Week 52 ]
    Change from baseline to Week 52 in quadriceps QMT.
  • Change from Baseline in Patient-Reported Physical Function at Week 52 [ Time Frame: Baseline, Week 52 ]
    Change from baseline to Week 52 in a patient reported outcome instrument.
  • Rate of Fall Events [ Time Frame: Baseline, Day 1, Weeks 2, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 60, 64, 68, 72, 76, 80, 84, 88, 92, 96, 100, 104, 108 ]
    Number of falls
  • Change from Baseline in Short Physical Performance Battery score at Week 52 [ Time Frame: Baseline, Week 52 ]
    Change from baseline to Week 52 in physical performance as measured by the Short Physical Performance Battery (SPPB).
  • Safety and Tolerability of different i.v. BYM338 doses [ Time Frame: Baseline, Day 1, Weeks 2, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 60, 64, 68, 72, 76, 80, 84, 88, 92, 96, 100, 104, 108 ]
    Safety and tolerability
  • Change from Baseline in 6MWD meters to Week 52 [ Time Frame: Baseline, Week 52 ]
    Dose-response relationship


Information By: Novartis

Dates:
Date Received: August 15, 2013
Date Started: September 26, 2013
Date Completion:
Last Updated: April 5, 2017
Last Verified: April 2017