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).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
251
BYM338, a 150 mg/mL concentrate for solution for i.v. infusion, was provided in colorless glass vials with a rubber stopper and aluminum flip-off caps.
Matching placebo to BYM338 was provided in colorless glass vials with a rubber stopper and aluminum flip-off caps.
Change From Baseline in 6 Minute Walking Distance (6MWD) Test at 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.
Time frame: Baseline, Week 52
Estimated Within Treatment Group Lean Body Mass (LBM) Ratio at 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.
Time frame: Baseline, Week 52
Change From Baseline in Quadriceps Quantitative Muscle Testing (QMT) on the Right Side at Week 52
Quadriceps muscle strength was measured by portable fixed dynamometry (PFD) on the right side. A negative change from baseline indicates deterioration.
Time frame: Baseline, Week 52
Change From Baseline in Sporadic Inclusion Body Myositis (sIBM) Functional Assessment (sIFA) Score at 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.
Time frame: Baseline, Week 52
Estimated Annual Number of Falls Per Patient Within Treatment Group
Participants documented any fall occurrences in a paper diary during the study.
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Novartis Investigative Site
Phoenix, Arizona, United States
Novartis Investigative Site
Orange, California, United States
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Sacramento, California, United States
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Miami, Florida, United States
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Kansas City, Kansas, United States
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Baltimore, Maryland, United States
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Boston, Massachusetts, United States
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Boston, Massachusetts, United States
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Columbus, Ohio, United States
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Portland, Oregon, United States
...and 28 more locations
Time frame: Week 52
Change From Baseline in Short Physical Performance Battery (SPPB) Score at 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.
Time frame: Baseline, Week 52