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

Sporadic Inclusion Body Myositis Clinical Trial

Official 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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT03710941
• Other study ID #: R2477-1033-sIBM-1828
• Secondary ID: 2018-002853-31
• Status: Withdrawn
• Phase: Phase 2
• Start date: February 19, 2019
• Est. completion date: November 5, 2020

Study information

• Verified date: April 2019
• Source: Regeneron Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: November 5, 2020
• Est. primary completion date: August 27, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 45 Years to 75 Years

Eligibility

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

Related Conditions & MeSH terms

• Myositis
• Myositis, Inclusion Body
• Sporadic Inclusion Body Myositis

Intervention

Drug:
• REGN2477+REGN1033
Single, sequential, repeat-dose Intravenous (IV) or matching placebo
• Matching placebo
Single, sequential, repeat-dose Intravenous (IV)

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Regeneron Pharmaceuticals

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent change in total lean mass as measured by dual-energy X-ray absorptiometry (DXA)		Up to Week 20
Secondary	Change in the Inclusion Body Myositis-Functional Rating Scale (IBM-FRS)		Up to Week 26
Secondary	Change in the Sporadic inclusion body myositis (sIBM) Physical Functioning Assessment (sIFA)		Up to Week 26
Secondary	Incidence and severity of treatment-emergent adverse events (TEAEs)		Up to Week 30
Secondary	Percent change in total and regional body composition (including lean mass and fat mass) as measured by DXA		Up to Week 30
Secondary	Absolute change in total and regional body composition (including lean mass and fat mass) as measured by DXA		Up to Week 30
Secondary	Change in the 1-repetition maximum (1-RM) chest press strength		Up to Week 26
Secondary	Change in hand-grip strength as measured by dynamometry		Up to Week 26
Secondary	Change in distance walked in the 6-minute walk test (6MWT)		Up to Week 26
Secondary	Change in time to complete the 10-meter walk test (10MWT)		Up to Week 26
Secondary	Change in instrumented stair climb power		Up to Week 26
Secondary	Change in instrumented, sensor-based sit-to-stand test time		Up to Week 26
Secondary	Change in fear of falling as measured via the Falls Efficacy Scale-International (FES-I)		Up to Week 30
Secondary	Change in Patient Global Impression of Severity assessments (PGIS/PGIC)		Up to Week 30
Secondary	Change in Patient Global Impression of Change Items assessments (PGIS/PGIC)		Up to Week 30
Secondary	Change in Clinician Global Impression of Severity assessments (CGIS/CGIC)		Up to Week 30
Secondary	Change in Clinician Global Impression of Change Items assessments (CGIS/CGIC)		Up to Week 30
Secondary	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
Secondary	Change in Thigh muscle volume as measured by MRI (sub-study)		Up to Week 30
Secondary	Frequency of falls and near-falls as measured by the Hopkins Falls Grading Scale		Up to Week 30
Secondary	Pharmacokinetics (PK) profile of REGN2477	Assessed via serum concentration of REGN2477 over time	Up to Week 30
Secondary	Pharmacokinetics (PK) profile of REGN1033	Assessed via serum concentration of REGN1033 over time	Up to Week 30
Secondary	Immunogenicity of REGN2477+REGN1033	As determined by the presence or absence of anti-drug antibodies (ADA)	Up to Week 30