2023-507666-32-00招募中2 期
BIGTIM: Blocking Interferon-γ by ruxolitinib for Treating Inclusion body Myositis: a phase IIb trial.
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 80
- 试验地点
- 14
- 主要终点
- Improvement of 60 meters for the distance walked during 6-minute (6WMD) from baseline to M12 (6MWD).
研究概览
简要总结
to evaluate whether ruxolitinib may improve the overall locomotor performance of IBM patients after a 1 year duration treatment period
研究设计
- 分配方式
- Randomized
- 主要目的
- Bigtim
- 盲法
- Double (Subject, Investigator)
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 否
入选标准
- •Age ≥ 45 years
- •Effective contraception for the duration of the clinical trial for fertile women of childbearing age. The participant agrees to follow the contraceptive requirements detailed in the protocol.
- •Defined diagnosis of IBM according to data-derived criteria (Llyod et al, 2014): Patient must fulfill the three following criteria for being diagnosed as IBM: (1) finger flexor or quadriceps weakness; and (2) muscle biopsy showing endomysial inflammation; and (3) muscle biopsy showing invasion of nonnecrotic muscle fibers or rimmed vacuoles
- •To be able to walk 6 min without assistance from another person (external assist devices permitted [e.g., canes, walkers, or rollators])
- •Patient informed and having signed the consent for participation, possibly assisted by a trusted person
排除标准
- •Pregnancy or breastfeeding
- •Any medical condition which limits the ability of participant to participate in study
- •Necessity to use a drug incompatible with ruxolitinib (see 7.4)
- •Hypersensitivity to the IMP’s active substance (ruxolitinib) or to any of the excipients (Cellulose microcrystalline, magnesium stearate, silica colloidal anhydrous, sodium carboxymethyl starch (Type A), povidone K30, hydroxypropylcellulose 300 to 600 cps, lactose monohydrate)
- •Non-affiliation to a social security scheme or to another social protection scheme, patient on AME (state medical aid)
- •Foreseeable inability, according to the investigator, to participate in all the visits, treatments and measures provided for in the protocol
- •Concomitant participation in another clinical trial on medical product for human use, to a clinical investigation on a medical device, to interventional study involving human participants or in the exclusion period at the end of a previous clinical trial on medical product for human use, a clinical investigation on a medical device, or study involving human participants. Participation in non-interventional research is permitted.
- •Patient under guardianship, curatorship, safeguard of justice or deprived of liberty
- •Patient with cognitive disorders or unable, according to the investigator, to understand the study and/or to give informed consent According to the appreciation of the investigator, non-French speaking patients may be included if a close one is able to translate the information provided. The patient must be able to benefit from the accompaniment of a relative for all of their visits (in teleconsultation and in hospital).
- •Quadriceps weakness (manual muscle testing, MRC) below or equal 1
- •Forced vital capacity (FVC) or forced expiratory volume (FEV) < 50% of predicted value
- •Concomitant use of immunomodulatory drugs including previous treatment with JAK inhibitor, or medications acting on muscle anabolism or catabolism
- •Live vaccine within the 4 weeks before starting ruxolitinib therapy
- •Comorbidity or active chronic disease which contraindicate ruxolitinib: • If the results of the biological assessment including blood count, blood formula and biochemistry and dating back less than three months are available, see the non-inclusion criteria below. For patients whose results of the biological assessment carried out on the day of the inclusion and randomization visit are not yet available, these criteria constitute secondary exclusion criteria to be checked upon receipt of the results and before the randomization. Lipid parameters abnormalities/elevations (in lack of cardiovascular risk factors, normal values with or without lipid-lowering treatment are: CTtotal cholesterol < 2 g/L; LDL-C < 1.6 g/L; HDL-C > 0.4 g/L; TGtriglycerides < 1.5 g/L) Severe renal impairment (stage 4) and end-stage renal disease (stage 5): GFR < 30mL/min/1.73m2 Hepatic impairment: AST/ALT > 3 ULN and bilirubin > 1.5 ULN Cytopenia (polymorphonuclear neutrophilsPNN ≤ 1.5 Giga/L or platelets ≤ 75 Giga/L or hemoglobin ≤ 10 g/dL)
- •Active SARS-CoV-2 infection (patient can be included once infection resolved)
结局指标
主要结局
Improvement of 60 meters for the distance walked during 6-minute (6WMD) from baseline to M12 (6MWD).
Improvement of 60 meters for the distance walked during 6-minute (6WMD) from baseline to M12 (6MWD).
次要结局
- 1. Safety and tolerance of ruxolitinib in IBM patients: Adverse events according to the MedDRA classification.
- Therapeutic muscular efficacy of ruxolitinib through change from baseline to M12 of the parameters depending on skeletal muscle involvement: (i) Muscle strengt (ii) Overall muscle status, (iii) Respiratory ability, (iv) Swallowing, (v) Lower limb Quantification of fat replacement of muscle tissue, residual muscle tissue and markers of disease activity using MRI
- 3. Improvement or stability between baseline and M12 in quality of life assessed by the scoring through Health Assessment Questionnaire without Disability Index (HAQ-DI), Duke health profile.
- 4. Quality of the blinding assessed by the New Blinding Index (Bang, 2004).
研究者
Pr François Jérôme AUTHIER
Scientific
Assistance Publique Hopitaux De Paris
研究点 (14)
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