Safety and Efficacy of REGN2477+REGN1033 in Patients With Sporadic Inclusion Body Myositis (NCT03710941) | Clinical Trial Compass
WithdrawnPhase 2
Safety and Efficacy of REGN2477+REGN1033 in Patients With Sporadic Inclusion Body Myositis
Stopped: The study was withdrawn due to an internal decision
0Started 2019-02-19
Plain-language 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
Who can participate
Age range
45 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Percent change in total lean mass as measured by dual-energy X-ray absorptiometry (DXA)