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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03710941
• Date of registration: 15/10/2018
• Prospective Registration: Yes
• Primary sponsor: Regeneron Pharmaceuticals
• Public title: Safety and Efficacy of REGN2477+REGN1033 in Patients With Sporadic Inclusion Body Myositis
• Scientific title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Intravenously Administered REGN2477+REGN1033 in Patients With Sporadic Inclusion Body Myositis
• Date of first enrolment: February 19, 2019
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT03710941
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Contacts

• Name: Clinical Trial Management
• Affiliation: Regeneron Pharmaceuticals

Key inclusion & 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



Age minimum: 45 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Sporadic Inclusion Body Myositis

Intervention(s)

Drug: REGN2477+REGN1033

Drug: Matching placebo

Primary Outcome(s)

Percent change in total lean mass as measured by dual-energy X-ray absorptiometry (DXA) [Time Frame: Up to Week 20]

Secondary Outcome(s)

Change in Clinician Global Impression of Change Items assessments (CGIS/CGIC) [Time Frame: Up to Week 30]

Change in hand-grip strength as measured by dynamometry [Time Frame: Up to Week 26]

Change in Patient Global Impression of Severity assessments (PGIS/PGIC) [Time Frame: Up to Week 30]

Change in Thigh muscle volume as measured by MRI (sub-study) [Time Frame: Up to Week 30]

Absolute change in total and regional body composition (including lean mass and fat mass) as measured by DXA [Time Frame: Up to Week 30]

Frequency of falls and near-falls as measured by the Hopkins Falls Grading Scale [Time Frame: Up to Week 30]

Immunogenicity of REGN2477+REGN1033 [Time Frame: Up to Week 30]

Change in Clinician Global Impression of Severity assessments (CGIS/CGIC) [Time Frame: Up to Week 30]

Change in the 1-repetition maximum (1-RM) chest press strength [Time Frame: Up to Week 26]

Change in the Sporadic inclusion body myositis (sIBM) Physical Functioning Assessment (sIFA) [Time Frame: Up to Week 26]

Percent change in total and regional body composition (including lean mass and fat mass) as measured by DXA [Time Frame: Up to Week 30]

Pharmacokinetics (PK) profile of REGN1033 [Time Frame: Up to Week 30]

Pharmacokinetics (PK) profile of REGN2477 [Time Frame: Up to Week 30]

Change in distance walked in the 6-minute walk test (6MWT) [Time Frame: Up to Week 26]

Change in instrumented stair climb power [Time Frame: Up to Week 26]

Change in the Inclusion Body Myositis-Functional Rating Scale (IBM-FRS) [Time Frame: Up to Week 26]

Incidence and severity of treatment-emergent adverse events (TEAEs) [Time Frame: Up to Week 30]

Change in time to complete the 10-meter walk test (10MWT) [Time Frame: Up to Week 26]

Change in fear of falling as measured via the Falls Efficacy Scale-International (FES-I) [Time Frame: Up to Week 30]

Change in instrumented, sensor-based sit-to-stand test time [Time Frame: Up to Week 26]

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 [Time Frame: Up to Week 30]

Change in Patient Global Impression of Change Items assessments (PGIS/PGIC) [Time Frame: Up to Week 30]

Secondary ID(s)

2018-002853-31

R2477-1033-sIBM-1828

Source(s) of Monetary Support

Please refer to primary and secondary sponsors