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

Primary Purpose

Sporadic Inclusion Body Myositis

Status

Withdrawn

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

REGN2477+REGN1033

Matching placebo

About this trial

This is an interventional treatment trial for Sporadic Inclusion Body Myositis

Eligibility Criteria

45 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

REGN2477+REGN1033

Placebo

Arm Description

Single, sequential, repeat-dose IV or matching placebo

Single, sequential, repeat-dose IV

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Change in the Inclusion Body Myositis-Functional Rating Scale (IBM-FRS)

Change in the Sporadic inclusion body myositis (sIBM) Physical Functioning Assessment (sIFA)

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

Percent change in total and regional body composition (including lean mass and fat mass) as measured by DXA

Absolute change in total and regional body composition (including lean mass and fat mass) as measured by DXA

Change in the 1-repetition maximum (1-RM) chest press strength

Change in hand-grip strength as measured by dynamometry

Change in distance walked in the 6-minute walk test (6MWT)

Change in time to complete the 10-meter walk test (10MWT)

Change in instrumented stair climb power

Change in instrumented, sensor-based sit-to-stand test time

Change in fear of falling as measured via the Falls Efficacy Scale-International (FES-I)

Change in Patient Global Impression of Severity assessments (PGIS/PGIC)

Change in Patient Global Impression of Change Items assessments (PGIS/PGIC)

Change in Clinician Global Impression of Severity assessments (CGIS/CGIC)

Change in Clinician Global Impression of Change Items assessments (CGIS/CGIC)

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

Change in Thigh muscle volume as measured by MRI (sub-study)

Frequency of falls and near-falls as measured by the Hopkins Falls Grading Scale

Pharmacokinetics (PK) profile of REGN2477

Assessed via serum concentration of REGN2477 over time

Pharmacokinetics (PK) profile of REGN1033

Assessed via serum concentration of REGN1033 over time

Immunogenicity of REGN2477+REGN1033

As determined by the presence or absence of anti-drug antibodies (ADA)

Full Information

NCT ID

NCT03710941

First Posted

October 15, 2018

Last Updated

April 25, 2019

Sponsor

Regeneron Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT03710941

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2019

Overall Recruitment Status

Withdrawn

Why Stopped

The study was withdrawn due to an internal decision

Study Start Date

February 19, 2019 (Anticipated)

Primary Completion Date

August 27, 2020 (Anticipated)

Study Completion Date

November 5, 2020 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Regeneron Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Sporadic Inclusion Body Myositis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

REGN2477+REGN1033

Arm Type

Experimental

Arm Description

Single, sequential, repeat-dose IV or matching placebo

Arm Title

Placebo

Arm Type

Experimental

Arm Description

Single, sequential, repeat-dose IV

Intervention Type

Drug

Intervention Name(s)

REGN2477+REGN1033

Intervention Description

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

Intervention Type

Drug

Intervention Name(s)

Matching placebo

Intervention Description

Single, sequential, repeat-dose Intravenous (IV)

Primary Outcome Measure Information:

Title

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

Time Frame

Up to Week 20

Secondary Outcome Measure Information:

Title

Change in the Inclusion Body Myositis-Functional Rating Scale (IBM-FRS)

Time Frame

Up to Week 26

Title

Change in the Sporadic inclusion body myositis (sIBM) Physical Functioning Assessment (sIFA)

Time Frame

Up to Week 26

Title

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

Time Frame

Up to Week 30

Title

Percent change in total and regional body composition (including lean mass and fat mass) as measured by DXA

Time Frame

Up to Week 30

Title

Absolute change in total and regional body composition (including lean mass and fat mass) as measured by DXA

Time Frame

Up to Week 30

Title

Change in the 1-repetition maximum (1-RM) chest press strength

Time Frame

Up to Week 26

Title

Change in hand-grip strength as measured by dynamometry

Time Frame

Up to Week 26

Title

Change in distance walked in the 6-minute walk test (6MWT)

Time Frame

Up to Week 26

Title

Change in time to complete the 10-meter walk test (10MWT)

Time Frame

Up to Week 26

Title

Change in instrumented stair climb power

Time Frame

Up to Week 26

Title

Change in instrumented, sensor-based sit-to-stand test time

Time Frame

Up to Week 26

Title

Change in fear of falling as measured via the Falls Efficacy Scale-International (FES-I)

Time Frame

Up to Week 30

Title

Change in Patient Global Impression of Severity assessments (PGIS/PGIC)

Time Frame

Up to Week 30

Title

Change in Patient Global Impression of Change Items assessments (PGIS/PGIC)

Time Frame

Up to Week 30

Title

Change in Clinician Global Impression of Severity assessments (CGIS/CGIC)

Time Frame

Up to Week 30

Title

Change in Clinician Global Impression of Change Items assessments (CGIS/CGIC)

Time Frame

Up to Week 30

Title

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

Title

Change in Thigh muscle volume as measured by MRI (sub-study)

Time Frame

Up to Week 30

Title

Frequency of falls and near-falls as measured by the Hopkins Falls Grading Scale

Time Frame

Up to Week 30

Title

Pharmacokinetics (PK) profile of REGN2477

Description

Assessed via serum concentration of REGN2477 over time

Time Frame

Up to Week 30

Title

Pharmacokinetics (PK) profile of REGN1033

Description

Assessed via serum concentration of REGN1033 over time

Time Frame

Up to Week 30

Title

Immunogenicity of REGN2477+REGN1033

Description

As determined by the presence or absence of anti-drug antibodies (ADA)

Time Frame

Up to Week 30

10. Eligibility

Sex

All

Minimum Age & Unit of Time

45 Years

Maximum Age & Unit of Time

75 Years

Accepts Healthy Volunteers

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Clinical Trial Management

Organizational Affiliation

Regeneron Pharmaceuticals

Official's Role

Study Director

12. IPD Sharing Statement

Learn more about this trial

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

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