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

Browse trials for

• Myositis 242
• Myositis, Inclusion Body 49

Keywords

• sporadic inclusion body myositis,
• myositis,
• muscle wasting,
• controlled clinical trial,
• randomized,
• body mass,
• muscle function,
• strength,
• performance,
• physical function

Myositis

242 studies on the registry are indexed under Myositis; 103 are open to participants now.

This study's enrollment of 251 is above the median of 30 across 159 interventional studies indexed under Myositis.

Lead sponsor

Novartis Pharmaceuticals is the lead sponsor of 2,673 studies on the registry; 228 are open to participants now.

Of its 576 completed or terminated interventional studies of FDA-regulated products, 431 (75%) have results posted.

Eligibility criteria

Study arms

• Experimental
  BYM338/bimagrumab 10 mg/kg

  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

• Experimental
  BYM338/bimagrumab 3 mg/kg

  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

• Experimental
  BYM338/bimagrumab 1 mg/kg

  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

• Placebo comparator
  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.

  Drug: Placebo

Interventions

• DrugBYM338/bimagrumab

  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.

• DrugPlacebo

  Matching placebo to BYM338 was provided in colorless glass vials with a rubber stopper and aluminum flip-off caps.

Primary outcomes

1. 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

Secondary outcomes

1. 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

2. 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

3. 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

4. Estimated Annual Number of Falls Per Patient Within Treatment Group

   Participants documented any fall occurrences in a paper diary during the study.

   Time frame: Week 52

5. 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

Baseline characteristics

Publications

• Hanna MG, Badrising UA, Benveniste O, Lloyd TE, Needham M, Chinoy H, Aoki M, Machado PM, Liang C, Reardon KA, de Visser M, Ascherman DP, Barohn RJ, Dimachkie MM, Miller JAL, Kissel JT, Oskarsson B, Joyce NC, Van den Bergh P, Baets J, De Bleecker JL, Karam C, David WS, Mirabella M, Nations SP, Jung HH, Pegoraro E, Maggi L, Rodolico C, Filosto M, Shaibani AI, Sivakumar K, Goyal NA, Mori-Yoshimura M, Yamashita S, Suzuki N, Katsuno M, Murata K, Nodera H, Nishino I, Romano CD, Williams VSL, Vissing J, Auberson LZ, Wu M, de Vera A, Papanicolaou DA, Amato AA; RESILIENT Study Group. Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial. Lancet Neurol. 2019 Sep;18(9):834-844. doi: 10.1016/S1474-4422(19)30200-5. PubMed 31397289 ↗