Safety and Efficacy of REGN2477+REGN1033 in Patients With Sporadic Inclusion Body Myositis
- Conditions
- Sporadic Inclusion Body Myositis
- Interventions
- Drug: REGN2477+REGN1033Drug: Matching placebo
- Registration Number
- NCT03710941
- Lead Sponsor
- Regeneron Pharmaceuticals
- Brief 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
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- WITHDRAWN
- Sex
- All
- Target Recruitment
- Not specified
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description REGN2477+REGN1033 REGN2477+REGN1033 Single, sequential, repeat-dose IV or matching placebo Placebo Matching placebo Single, sequential, repeat-dose IV
- Primary Outcome Measures
Name Time Method Percent change in total lean mass as measured by dual-energy X-ray absorptiometry (DXA) Up to Week 20
- Secondary Outcome Measures
Name Time Method Change in the Inclusion Body Myositis-Functional Rating Scale (IBM-FRS) Up to Week 26 Change in the Sporadic inclusion body myositis (sIBM) Physical Functioning Assessment (sIFA) Up to Week 26 Percent change in total and regional body composition (including lean mass and fat mass) as measured by DXA Up to Week 30 Absolute change in total and regional body composition (including lean mass and fat mass) as measured by DXA Up to Week 30 Change in the 1-repetition maximum (1-RM) chest press strength Up to Week 26 Change in hand-grip strength as measured by dynamometry Up to Week 26 Change in distance walked in the 6-minute walk test (6MWT) Up to Week 26 Change in time to complete the 10-meter walk test (10MWT) Up to Week 26 Incidence and severity of treatment-emergent adverse events (TEAEs) Up to Week 30 Change in instrumented stair climb power Up to Week 26 Change in instrumented, sensor-based sit-to-stand test time Up to Week 26 Change in fear of falling as measured via the Falls Efficacy Scale-International (FES-I) Up to Week 30 Change in Patient Global Impression of Severity assessments (PGIS/PGIC) Up to Week 30 Change in Patient Global Impression of Change Items assessments (PGIS/PGIC) Up to Week 30 Change in Clinician Global Impression of Severity assessments (CGIS/CGIC) Up to Week 30 Change in Clinician Global Impression of Change Items assessments (CGIS/CGIC) Up to Week 30 Change in 36-item Short Form Health Survey (SF-36), including the Physical Function Items (PF-10) and the Vitality Scale of the SF-36 Up to Week 30 Change in Thigh muscle volume as measured by MRI (sub-study) Up to Week 30 Frequency of falls and near-falls as measured by the Hopkins Falls Grading Scale Up to Week 30 Pharmacokinetics (PK) profile of REGN2477 Up to Week 30 Assessed via serum concentration of REGN2477 over time
Pharmacokinetics (PK) profile of REGN1033 Up to Week 30 Assessed via serum concentration of REGN1033 over time
Immunogenicity of REGN2477+REGN1033 Up to Week 30 As determined by the presence or absence of anti-drug antibodies (ADA)