Fecal Microbiota Transplantation Effect on Amyotrophic Lateral Sclerosis Patients (FETR-ALS)
Amyotrophic Lateral Sclerosis
About this trial
This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring microbiota, fecal transplantation, adaptive immunity, T regulatory lymphocytes
Eligibility Criteria
Inclusion Criteria:
- Patients diagnosed with a laboratory supported, clinically "probable" or "definite" amyotrophic lateral sclerosis according to the Revised El Escorial criteria (Brooks, 2000)
- Sporadic or familial ALS
- Female or male patients aged between 18 and 70 years old
- Disease duration from symptoms onset no longer than 18 months at the screening visit
- Patients treated with a stable dose of Riluzole (100 mg/day) for at least 30 days prior to screening
- Patients with a weight > 50 kg and a BMI ≥18
- Patients with a FVC (Forced Vital Capacity) equal or more than 70% predicted normal value for gender, height, and age at the screening visit
- Patients able and willing to comply with study procedures as per protocol
- Patients able to understand, and capable of providing informed consent at screening visit prior to any protocol-specific procedures
- Use of effective contraception both for males and females
Exclusion Criteria:
- Known organic gastrointestinal disease
- History of gastrointestinal malignancy; ongoing malignancies
- Use of immunosuppressive or chemotherapy within the past 2 years
- Celiac disease and/or food (e.g.lactose) intolerance
- Previous gastrointestinal surgery
- Any condition that would make endoscopic procedures contraindicated
- Acute infections requiring antibiotics
- Antimicrobial treatment or probiotics 4 weeks prior to screening
- Severe comorbidities (heart, renal, liver failure); severe renal (eGFR< 30ml/min/1.73m2), or liver failure or liver aminotransferase (ALT/AST > 2x Upper limit of normal),
- Autoimmune diseases, inflammatory disorders (SLE, Rheumatoid arthritis, connective tissue disorder) or chronic infections (HIV, hepatitis B or C infection)
- Abuse of alcohol or drugs
- HIV, tuberculosis, hepatitis
- Participation in clinical trials <30 days before screening
- Existing blood dyscrasia (e.g., myelodysplasia)
- White blood cells<4,000/mm³, platelets count<100,000/mm³, hematocrit<30%
- Patients who underwent non-invasive ventilation, tracheotomy and /or gastrostomy
- Women who are pregnant or breastfeeding
Sites / Locations
- Clinica Neurologica, Ospedale Clinicizzato "SS Annunziata"
- Azienda Ospedaliero Universitaria di Modena
- UO Neurofisiopatologia, Azienda Ospedaliera dì Perugia
- Catholic University of Sacred Heart - Fondazione Policlinico "A. Gemelli"
- NEuroMuscular Omnicentre Centre (NeMO), Fondazione Serena Onlus-Fondazione Policlinico A. Gemelli
Arms of the Study
Arm 1
Arm 2
Active Comparator
Placebo Comparator
Fecal microbiota transplantation (FMT)
Placebo
Fecal microbiota transplantation will be performed during two endoscopic procedures (at baseline and at 6 months) by allogenic infusion of collected feces in the duodenum-jejunum. Fecal microbiota will be diluted in saline solution 200 ml and infused at 30 ml/minute speed. Every endoscopic procedure will be performed with sedation of the patient. Feces for FMT will be obtained by known healthy donors for C. difficile infection according to standard selection procedures.
ALS patients will undergo upper GI endoscopy with small-intestine biopsies at baseline and after 6 months. Patients in the placebo arm will not receive any treatment during these procedures, but will undergo intestinal biopsy. Every endoscopic procedure will be performed with sedation of the patient.