- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03710941
Safety and Efficacy of REGN2477+REGN1033 in Patients With Sporadic Inclusion Body Myositis
April 25, 2019 updated by: Regeneron Pharmaceuticals
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
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
Study Overview
Status
Withdrawn
Conditions
Intervention / Treatment
Study Type
Interventional
Phase
- Phase 2
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
43 years to 73 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: REGN2477+REGN1033
Single, sequential, repeat-dose IV or matching placebo
|
Single, sequential, repeat-dose Intravenous (IV) or matching placebo
|
Experimental: Placebo
Single, sequential, repeat-dose IV
|
Single, sequential, repeat-dose Intravenous (IV)
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Percent change in total lean mass as measured by dual-energy X-ray absorptiometry (DXA)
Time Frame: Up to Week 20
|
Up to Week 20
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in the Inclusion Body Myositis-Functional Rating Scale (IBM-FRS)
Time Frame: Up to Week 26
|
Up to Week 26
|
|
Change in the Sporadic inclusion body myositis (sIBM) Physical Functioning Assessment (sIFA)
Time Frame: Up to Week 26
|
Up to Week 26
|
|
Incidence and severity of treatment-emergent adverse events (TEAEs)
Time Frame: Up to Week 30
|
Up to Week 30
|
|
Percent change in total and regional body composition (including lean mass and fat mass) as measured by DXA
Time Frame: Up to Week 30
|
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
|
Up to Week 30
|
|
Change in the 1-repetition maximum (1-RM) chest press strength
Time Frame: Up to Week 26
|
Up to Week 26
|
|
Change in hand-grip strength as measured by dynamometry
Time Frame: Up to Week 26
|
Up to Week 26
|
|
Change in distance walked in the 6-minute walk test (6MWT)
Time Frame: Up to Week 26
|
Up to Week 26
|
|
Change in time to complete the 10-meter walk test (10MWT)
Time Frame: Up to Week 26
|
Up to Week 26
|
|
Change in instrumented stair climb power
Time Frame: Up to Week 26
|
Up to Week 26
|
|
Change in instrumented, sensor-based sit-to-stand test time
Time Frame: Up to Week 26
|
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
|
Up to Week 30
|
|
Change in Patient Global Impression of Severity assessments (PGIS/PGIC)
Time Frame: Up to Week 30
|
Up to Week 30
|
|
Change in Patient Global Impression of Change Items assessments (PGIS/PGIC)
Time Frame: Up to Week 30
|
Up to Week 30
|
|
Change in Clinician Global Impression of Severity assessments (CGIS/CGIC)
Time Frame: Up to Week 30
|
Up to Week 30
|
|
Change in Clinician Global Impression of Change Items assessments (CGIS/CGIC)
Time Frame: Up to Week 30
|
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
Time Frame: Up to Week 30
|
Up to Week 30
|
|
Change in Thigh muscle volume as measured by MRI (sub-study)
Time Frame: Up to Week 30
|
Up to Week 30
|
|
Frequency of falls and near-falls as measured by the Hopkins Falls Grading Scale
Time Frame: Up to Week 30
|
Up to Week 30
|
|
Pharmacokinetics (PK) profile of REGN2477
Time Frame: Up to Week 30
|
Assessed via serum concentration of REGN2477 over time
|
Up to Week 30
|
Pharmacokinetics (PK) profile of REGN1033
Time Frame: Up to Week 30
|
Assessed via serum concentration of REGN1033 over time
|
Up to Week 30
|
Immunogenicity of REGN2477+REGN1033
Time Frame: Up to Week 30
|
As determined by the presence or absence of anti-drug antibodies (ADA)
|
Up to Week 30
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Anticipated)
February 19, 2019
Primary Completion (Anticipated)
August 27, 2020
Study Completion (Anticipated)
November 5, 2020
Study Registration Dates
First Submitted
October 15, 2018
First Submitted That Met QC Criteria
October 15, 2018
First Posted (Actual)
October 18, 2018
Study Record Updates
Last Update Posted (Actual)
April 29, 2019
Last Update Submitted That Met QC Criteria
April 25, 2019
Last Verified
April 1, 2019
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- R2477-1033-sIBM-1828
- 2018-002853-31 (EudraCT Number)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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