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

Regeneron Pharmaceuticals

Status and phase: Withdrawn; Phase 2

Conditions: Sporadic Inclusion Body Myositis

Treatments: Drug: Matching placebo; Drug: REGN2477+REGN1033

Study type: Interventional

Funder types: Industry

Identifiers

NCT03710941

R2477-1033-sIBM-1828

2018-002853-31 (EudraCT Number)

Details and patient eligibility

About

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

Sex: All

Ages: 45 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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0 participants in 2 patient groups

REGN2477+REGN1033

Experimental group

Description:

Single, sequential, repeat-dose IV or matching placebo

Treatment:

Drug: REGN2477+REGN1033

Placebo

Experimental group

Description:

Single, sequential, repeat-dose IV

Treatment:

Drug: Matching placebo