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

Phase 2

Completed

• Conditions: Sporadic Inclusion Body Myositis
• Interventions: Drug: BYM338/bimagrumab; Drug: Placebo

• Registration Number: NCT01925209
• Lead Sponsor: Novartis Pharmaceuticals

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 Description

Not available

Study Timeline

• Study First Submitted: 2013-08-15
• Study First Submitted QC: 2013-08-15
• Study First Posted: 2013-08-19
• Study Start: 2013-09-26
• Primary Completion: 2016-01-06
• Study Completion: 2016-01-06
• Results First Submitted: 2017-01-05
• Results First Submitted QC: 2017-04-05
• Results First Posted: 2017-05-12
• Status Verified: 2017-08
• Last Update Submitted: 2017-08-07
• Last Update Posted: 2017-08-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 251

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.);

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
BYM338/bimagrumab 10 mg/kg	BYM338/bimagrumab	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.
BYM338/bimagrumab 3 mg/kg	BYM338/bimagrumab	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.
BYM338/bimagrumab 1 mg/kg	BYM338/bimagrumab	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.
Placebo	Placebo	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.

Primary Outcome Measures

Name	Time	Method
Change From Baseline in 6 Minute Walking Distance (6MWD) Test at Week 52	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.

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in Quadriceps Quantitative Muscle Testing (QMT) on the Right Side at Week 52	Baseline, Week 52	Quadriceps muscle strength was measured by portable fixed dynamometry (PFD) on the right side. A negative change from baseline indicates deterioration.
Estimated Within Treatment Group Lean Body Mass (LBM) Ratio at Week 52	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 Sporadic Inclusion Body Myositis (sIBM) Functional Assessment (sIFA) Score at Week 52	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	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	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.

Trial Locations

Locations (1)

Novartis Investigative Site; 🇬🇧 Newcastle upon Tyne, United Kingdom