NCT03710941撤回2 期
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
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 主要终点
- Percent change in total lean mass as measured by dual-energy X-ray absorptiometry (DXA)
研究概览
简要总结
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
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 45 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
REGN2477+REGN1033
Experimental
Single, sequential, repeat-dose IV or matching placebo
干预措施: REGN2477+REGN1033 (Drug)
Placebo
Experimental
Single, sequential, repeat-dose IV
干预措施: Matching placebo (Drug)
结局指标
主要结局
Percent change in total lean mass as measured by dual-energy X-ray absorptiometry (DXA)
时间窗: Up to Week 20
次要结局
- Change in distance walked in the 6-minute walk test (6MWT)(Up to Week 26)
- Change in time to complete the 10-meter walk test (10MWT)(Up to Week 26)
- Change in the Inclusion Body Myositis-Functional Rating Scale (IBM-FRS)(Up to Week 26)
- Change in the Sporadic inclusion body myositis (sIBM) Physical Functioning Assessment (sIFA)(Up to Week 26)
- Percent change in total and regional body composition (including lean mass and fat mass) as measured by DXA(Up to Week 30)
- Absolute change in total and regional body composition (including lean mass and fat mass) as measured by DXA(Up to Week 30)
- Change in the 1-repetition maximum (1-RM) chest press strength(Up to Week 26)
- Change in hand-grip strength as measured by dynamometry(Up to Week 26)
- Incidence and severity of treatment-emergent adverse events (TEAEs)(Up to Week 30)
- Change in instrumented stair climb power(Up to Week 26)
- Change in instrumented, sensor-based sit-to-stand test time(Up to Week 26)
- Change in fear of falling as measured via the Falls Efficacy Scale-International (FES-I)(Up to Week 30)
- Change in Patient Global Impression of Severity assessments (PGIS/PGIC)(Up to Week 30)
- Change in Patient Global Impression of Change Items assessments (PGIS/PGIC)(Up to Week 30)
- Change in Clinician Global Impression of Severity assessments (CGIS/CGIC)(Up to Week 30)
- Change in Clinician Global Impression of Change Items assessments (CGIS/CGIC)(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(Up to Week 30)
- Change in Thigh muscle volume as measured by MRI (sub-study)(Up to Week 30)
- Frequency of falls and near-falls as measured by the Hopkins Falls Grading Scale(Up to Week 30)
- Pharmacokinetics (PK) profile of REGN2477(Up to Week 30)
- Pharmacokinetics (PK) profile of REGN1033(Up to Week 30)
- Immunogenicity of REGN2477+REGN1033(Up to Week 30)
研究者
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