Efficacy and Safety of Bimagrumab in Sporadic Inclusion Body Myositis: Long-term Extension of RESILIENT

Anthony A. Amato, Michael G. Hanna, Pedro M. Machado, Umesh A. Badrising, Hector Chinoy, Olivier Benveniste, Ananda Krishna Karanam, Min Wu, László B. Tankó, Agnes Annette Schubert-Tennigkeit, Dimitris A. Papanicolaou, Thomas E. Lloyd, Merrilee Needham, Christina Liang, Katrina A. Reardon, Marianne de Visser, Dana P. Ascherman, Richard J. Barohn, Mazen M. Dimachkie, James A.L. MillerJohn T. Kissel, Björn Oskarsson, Nanette C. Joyce, Peter Van den Bergh, Jonathan Baets, Jan L. De Bleecker, Chafic Karam, William S. David, Massimiliano Mirabella, Sharon P. Nations, Hans H. Jung, Elena Pegoraro, Lorenzo Maggi, Carmelo Rodolico, Massimiliano Filosto, Aziz I. Shaibani, Kumaraswamy Sivakumar, Namita A. Goyal, Madoka Mori-Yoshimura, Satoshi Yamashita, Naoki Suzuki, Masashi Aoki, Masahisa Katsuno, Hirokazu Morihata, Kenya Murata, Hiroyuki Nodera, Ichizo Nishino, Carla D. Romano, Valerie S.L. Williams, John Vissing, RESILIENT Study Extension Group

24 Citationer (Scopus)

Abstract

OBJECTIVE: To assess long-term (2 years) effects of bimagrumab in participants with sporadic inclusion body myositis (sIBM). METHODS: Participants (aged 36-85 years) who completed the core study (RESILIENT [Efficacy and Safety of Bimagrumab/BYM338 at 52 Weeks on Physical Function, Muscle Strength, Mobility in sIBM Patients]) were invited to join an extension study. Individuals continued on the same treatment as in the core study (10 mg/kg, 3 mg/kg, 1 mg/kg bimagrumab or matching placebo administered as IV infusions every 4 weeks). The co-primary outcome measures were 6-minute walk distance (6MWD) and safety. RESULTS: Between November 2015 and February 2017, 211 participants entered double-blind placebo-controlled period of the extension study. Mean change in 6MWD from baseline was highly variable across treatment groups, but indicated progressive deterioration from weeks 24-104 in all treatment groups. Overall, 91.0% (n = 142) of participants in the pooled bimagrumab group and 89.1% (n = 49) in the placebo group had ≥1 treatment-emergent adverse event (AE). Falls were slightly higher in the bimagrumab 3 mg/kg group vs 10 mg/kg, 1 mg/kg, and placebo groups (69.2% [n = 36 of 52] vs 56.6% [n = 30 of 53], 58.8% [n = 30 of 51], and 61.8% [n = 34 of 55], respectively). The most frequently reported AEs in the pooled bimagrumab group were diarrhea 14.7% (n = 23), involuntary muscle contractions 9.6% (n = 15), and rash 5.1% (n = 8). Incidence of serious AEs was comparable between the pooled bimagrumab and the placebo group (18.6% [n = 29] vs 14.5% [n = 8], respectively). CONCLUSION: Extended treatment with bimagrumab up to 2 years produced a good safety profile and was well-tolerated, but did not provide clinical benefits in terms of improvement in mobility. The extension study was terminated early due to core study not meeting its primary endpoint. CLINICAL TRIAL REGISTRATION: Clinicaltrials.gov identifier NCT02573467. CLASSIFICATION OF EVIDENCE: This study provides Class IV evidence that for patients with sIBM, long-term treatment with bimagrumab was safe, well-tolerated, and did not provide meaningful functional benefit. The study is rated Class IV because of the open-label design of extension treatment period 2.

OriginalsprogEngelsk
TidsskriftNeurology
Vol/bind96
Udgave nummer12
Sider (fra-til)e1595-e1607
ISSN0028-3878
DOI
StatusUdgivet - 23 mar. 2021

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