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

NCT03710941 | Withdrawn Phase 2 DMC FDA Drug

• 2 other identifiers: R2477-1033-sIBM-18282018-002853-31
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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

Conditions

Sporadic Inclusion Body Myositis

Outcome Measures

Primary Outcomes (1)

• Percent change in total lean mass as measured by dual-energy X-ray absorptiometry (DXA)

  Up to Week 20

Secondary Outcomes (22)

• 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

• Incidence and severity of treatment-emergent adverse events (TEAEs)

  Up to Week 30

• 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

Study Arms (2)

REGN2477+REGN1033

EXPERIMENTAL

Single, sequential, repeat-dose IV or matching placebo

Drug: REGN2477+REGN1033

Placebo

EXPERIMENTAL

Single, sequential, repeat-dose IV

Drug: Matching placebo

Interventions

REGN2477+REGN1033 DRUG

Single, sequential, repeat-dose Intravenous (IV) or matching placebo

REGN2477+REGN1033

Matching placebo DRUG

Single, sequential, repeat-dose Intravenous (IV)

Placebo

Eligibility Criteria

• Age: 45 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Regeneron Pharmaceuticals: lead

MeSH Terms

Conditions

Myositis, Inclusion Body

Condition Hierarchy (Ancestors)

Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases

Study Officials

• Clinical Trial Management

  Regeneron Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 15, 2018
• First Posted: October 18, 2018
• Study Start: February 19, 2019
• Primary Completion: August 27, 2020
• Study Completion: November 5, 2020
• Last Updated: April 29, 2019

Record last verified: 2019-04