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

Status

Indicates the current recruitment status or the expanded access status.
Withdrawn

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

41 years to 71 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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: REGN2477+REGN1033
Single, sequential, repeat-dose IV or matching placebo
Drug: REGN2477+REGN1033
Single, sequential, repeat-dose Intravenous (IV) or matching placebo
Experimental: Placebo
Single, sequential, repeat-dose IV
Drug: Matching placebo
Single, sequential, repeat-dose Intravenous (IV)

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Regeneron Pharmaceuticals

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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
February 19, 2019

Primary Completion (Anticipated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 27, 2020

Study Completion (Anticipated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 5, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 15, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 15, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 18, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 29, 2019

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 25, 2019

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2019

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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