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)
- Conditions
- G12.2Motor neuron disease
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 65
•Disease duration of sporadic ALS is = 24 months after symptom onset and symptoms have not progressed to a permanent need of assisted ventilation of any kind (including non-invasive ventilation).
•Written consent to participate in the study
•Patients with the capacity to give informed consent
•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 75% of the predicted normal value for gender, height and age at the screening visit
•Recommended vaccination according to STIKO (incl. COVID-19 vaccination)
•Dysfunction (other than ALS) that could distort or obscure the diagnosis of ALS
•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
•Patients with HIV infections
•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
•History of a tuberculosis (TB) infection, signs/symptoms of TB or close contact with a person with an active TB infection
•Clinically significant infection involving intravenous administration of antibiotics and hospitalization in the 4 weeks prior to the screening visit
•Patients with a serious impairment of the immune system
•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)
•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
•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.
•The patient has used edaravone or sodium phenylbutyrate–taurursodiol in the first week before the baseline visit
•Hypersensitivity to the active substance, mouse proteins or sodium citrate, polysorbate 80
•Known cytokine release syndrome after infusions
•Hypersensitivity to the adjuvant medication (paracetamol and prednisolone)
•Pregnancy or lactation
Study & Design
- Study Type
- interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change in ALSFRS-R-SE (ALS Functional Rating Scale - revised - self-explanatory) Score from baseline to 79 weeks after first dose of study drug
- Secondary Outcome Measures
Name Time Method Body Mass Index, Slow Vital Capacity Score