Safety and Efficacy of REGN2477+REGN1033 in Patients With Sporadic Inclusion Body Myositis

• Status: Withdrawn
• Trial end date: 2020-11-05
• Target enrollment: 0
• Participant gender: All

Summary

The primary objective of the study is to evaluate the efficacy of REGN2477+REGN1033 in combination on total lean mass, as measured by Dual-energy X-ray absorptiometry (DXA) in patients with sporadic inclusion body myositis (sIBM).

The secondary objectives of the study are:

• To evaluate the efficacy of REGN2477+REGN1033 on the IBM-Functional Rating Scale (IBM-FRS)

• To evaluate the efficacy of REGN2477+REGN1033 on the sIBM Physical Functioning Assessment (sIFA)

• To evaluate the safety and tolerability of REGN2477+REGN1033

• To evaluate the effects of REGN2477+REGN1033 on body composition by DXA, including appendicular lean mass and total fat mass

• To evaluate the efficacy of REGN2477+REGN1033 on measures of muscle performance and physical function

• To evaluate the efficacy of REGN2477+REGN1033 on patient reported outcome measures including the fear of falling, falls and near falls, and health-related quality of life

• To evaluate the pharmacokinetic(s) (PK) profile of REGN2477+REGN1033, including functional REGN2477 and functional REGN1033 concentrations in serum over time

• To evaluate the immunogenicity of REGN2477+REGN1033

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Regeneron Pharmaceuticals

Criteria

Key Inclusion Criteria:

• Men and postmenopausal* women

• Diagnosed with sIBM based on the European Neuromuscular Centre (ENMC) IBM Research Diagnostic Criteria

• Ability to walk ≥150 meters, with or without a walking aid such as cane or walker, in 6 minutes.

• Ability to climb 4 steps of stairs unassisted (may use handrails)

• Willing and able to comply with clinic visits and study-related procedures

Key Exclusion Criteria:

• Other neurological conditions (eg, hemiplegia post stroke, Parkinson's) or musculo-skeletal conditions (eg, severe osteoarthritis) causing mobility impairment

• Mini-Mental State Examination (MMSE) score <24

• Ongoing, chronic, high-dose (>20 mg prednisone equivalent per day), systemic corticosteroid therapy within 6 weeks prior to screening.

• Any condition that precludes adequate intake of energy and protein; malnutrition; presence of an eating disorder.

• Unintentional weight loss of ≥10% in the past 6 months (patient-reported)

• Hospitalization for heart failure in last year or New York Heart Association Class 4

• History of hypertrophic cardiomyopathy

• Any drugs known to influence muscle mass and performance such as anabolic steroids or growth hormone within 6 weeks prior to screening

• Unable to fit on the site's DXA scanner table, within borders for scanning of total lean mass

Note: Other protocol Inclusion/Exclusion criteria apply