2022-502743-35-00招募中2 期
B-cell depletion for the treatment of patients with amyotrophic lateral sclerosis (ALS) - A Randomized, Double-blind, Placebo controlled Pilot Study in Patients with Amyotrophic Lateral Sclerosis for the Evaluation of Efficacy and Safety of B-Cell Depletion with Rituximab (ABCD)
Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V.2 个研究点 分布在 1 个国家目标入组 52 人开始时间: 2023年5月15日最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 52
- 试验地点
- 2
- 主要终点
- ALS Functional Rating Scale - Revised – self-explenatory (ALSFRS-R-SE) change from baseline (first dose of study drug) to 79 weeks after administration of the first dose compared to standard therapy riluzole alone
研究概览
简要总结
The primary objective of the trial is to investigate if Rituximab as add-on treatment can reduce symptom progression in patients with ALS in comparison to standard therapy alone.
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Disease duration of sporadic ALS is ≤ 36 months after symptom onset and symptoms have not progressed to a permanent need of assisted ventilation of any kind (including non-invasive ventilation).
- •Age ≥ 18 years
- •Written consent to participate in the study
- •The patient is capable to attend study visits
- •Medication with riluzole at a stable dose of 50 mg BID for ≥ 30 days prior to the screening visit and if possible, throughout the study.
- •Slow vital capacity (VC) equal to or more than 60% of the predicted normal value for gender, height and age at the screening visit
- •Review of vaccination status and individual counseling according to STIKO guidelines. In case of missing vaccination, it is recommended to perform the vaccination parallel to the trial. Between vaccinations and Rituximab infusion, there should be an interval of four weeks (dose 1 at least four weeks before the first infusion, dose 2 four weeks before the third infusion). Decline of recommended vaccinations is acceptable only if participants have undergone a thorough informed consent process.
- •Patients with the capacity to give informed consent
排除标准
- •Dysfunction (other than ALS) that could distort or obscure the diagnosis of ALS.
- •Participation in any other investigational drug study or exposure to an investigational drug within 5 half-lives of the study drug at baseline
- •Patients with a history of recurrent or chronic infections or with underlying diseases which may further predispose patients to serious infection
- •Patients with a tracheostomy or ongoing treatment (more than 7 consecutive days in the 4 weeks prior to the screening visit) requiring noninvasive positive pressure ventilation of any kind
- •Hypersensitivity to the active substance, mouse proteins or sodium citrate, polysorbate 80
- •Known cytokine release syndrome after infusions
- •Hypersensitivity to the adjuvant medication (paracetamol, methylprednisolone and dimetinden maleate)
- •Pregnancy or lactation
- •The patient has used edaravone or sodium phenylbutyrate–taurursodiol in the first week before the baseline visit
- •Sexually active male and female patients of reproductive potential (female patients/ female partners of patients less than 12 months postmenopausal) who do not use highly effective contraception methods (pearl index <1) during and up to 12 months after treatment
- •Patients with HIV infections
- •Patients with a serious impairment of the immune system
- •Active severe infections (e.g. tuberculosis, sepsis and opportunistic infections)
- •History of chronically active hepatitis including active or chronic hepatitis B, acute or chronic hepatitis C
- •Clinically significant infection involving intravenous administration of antibiotics and hospitalization in the 4 weeks prior to the screening visit
- •Severe heart failure (New York Heart Association Class IV) or severe, uncontrolled heart disease
- •Patients having severe disease in the renal, cardiovascular or hematological system
- •Patients with neutrophils < 1000 cells per µl and/or platelet counts 75.000 cells per µl
- •Any medical condition that, in the opinion of the Investigator, might interfere with the patient’s participation in the trial or poses any added risk for the patient
- •Patients with other causes of neuromuscular weakness
- •Patients with severe active psychiatric illness
- •Patients with a diagnosis of another neurodegenerative disease (e.g. Parkinson disease, Alzheimer’s disease)
结局指标
主要结局
ALS Functional Rating Scale - Revised – self-explenatory (ALSFRS-R-SE) change from baseline (first dose of study drug) to 79 weeks after administration of the first dose compared to standard therapy riluzole alone
ALS Functional Rating Scale - Revised – self-explenatory (ALSFRS-R-SE) change from baseline (first dose of study drug) to 79 weeks after administration of the first dose compared to standard therapy riluzole alone
次要结局
- ALSFRS-R-SE change from baseline to 3, 27, 53, 105 and 131 weeks
- Change in the slow vital capacity score (pulmonary fuction test) from baseline to 79 weeks
- Change of BMI from baseline to 79 weeks
- Tracheostomy-free survival at 79 weeks
- Overall survival in Rituximab and standard therapy group
- Laboratory parameters evaluating the safety of treatment with Rituximab
- Serum and cerebrospinal fluid analyses with cell count, cytology, protein, glucose, lactate and infection markers
- B cell counts
- Neuropsychological tests (ECAS)
- Quality of Life questionnaire
研究者
Project Leader (Harald Prüß)
Scientific
Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V.
研究点 (2)
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