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

Study Overview
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Brief Summary
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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).

Official Title
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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 Weeks on Physical Function, Muscle Strength, and Mobility and Additional Long Term Safety up to 2 Years in Patients With Sporadic Inclusion Body Myositis

Conditions
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Sporadic Inclusion Body Myositis

Intervention / Treatment
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* Drug: BYM338/bimagrumab
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Diagnosed with sporadic inclusion body myositis; Must be able to walk (assistive aids allowed, including intermittent use of wheelchair); Key Exclusion Criteria: Must not have other conditions that significantly limit ability to move around; Must not be using corticosteroids. Must not have used systemic corticosteroid (at daily dose >=10mg prednisone) for the past 3 months; Must meet cardiovascular requirements; Must not be pregnant or nursing; Must not have a chronic active infection (e.g., HIV, hepatitis B or C, tuberculosis, etc.);

Ages Eligible for Study
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Minimum Age: 36 Years
Maximum Age: 85 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BYM338/bimagrumab 10 mg/kg<br>Participants received study medication with BYM338 at 10 mg/kg from Day 1 to Week 52 and up to Week 104, administered by intravenous (i.v.) infusion every 4 weeks. | Drug: BYM338/bimagrumab<br>* 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.<br>|
| Experimental: BYM338/bimagrumab 3 mg/kg<br>Participants received study medication with BYM338 at 3 mg/kg from Day 1 to Week 52 and up to Week 104, administered by i.v. infusion every 4 weeks. | Drug: BYM338/bimagrumab<br>* 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.<br>|
| Experimental: BYM338/bimagrumab 1 mg/kg<br>Participants received study medication with BYM338 at 1 mg/kg from Day 1 to Week 52 and up to Week 104, administered by i.v. infusion every 4 weeks. | Drug: BYM338/bimagrumab<br>* 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.<br>|
| Placebo Comparator: Placebo<br>Participants received matching placebo to BYM338 from Day 1 to Week 52 and up to Week 104, administered by i.v. infusion every 4 weeks. | Drug: Placebo<br>* Matching placebo to BYM338 was provided in colorless glass vials with a rubber stopper and aluminum flip-off caps.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | Baseline, Week 52 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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. | 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. | 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. | 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. | Baseline, Week 52 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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sporadic inclusion body myositis,, myositis,, muscle wasting,, controlled clinical trial,, randomized,, body mass,, muscle function,, strength,, performance,, physical function