Fecal Microbiota Transplantation Effect on Amyotrophic Lateral Sclerosis Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 12 February 2024
• Main ID: NCT03766321
• Date of registration: 30/11/2018
• Prospective Registration: Yes
• Primary sponsor: Azienda Ospedaliero-Universitaria di Modena
• Public title: Fecal Microbiota Transplantation Effect on Amyotrophic Lateral Sclerosis Patients FETR-ALS
• Scientific title: Interplay Between Gut Microbiota and Adaptive Immunity in Amyotrophic Lateral Sclerosis: a Clinical Trial
• Date of first enrolment: July 1, 2020
• Target sample size: 42
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03766321
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: N/A

Countries of recruitment

Italy

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Biological: Fecal microbiota transplantation

Biological: Placebo

Primary Outcome(s)

Change in Tregs number [Time Frame: 6 months]

Secondary Outcome(s)

Forced vital capacity (FVC) [Time Frame: 12 months (at baseline and month 3, 6, 9, 12)]

Change in T cell subsets frequency in blood and gut tissue samples [Time Frame: 12 months (at time points: baseline, month 3 - 6 (both arms)- 9 -12 (both arms))]

Change in heavy neurofilaments levels in CSF [Time Frame: 6 months (at baseline and at month 6)]

Changes in microbiota profile [Time Frame: 12 months (at baseline and at month 6 and 12)]

Changes in levels of pro-inflammatory cytokines and cytokines linked to T cell proliferation and differentiation [Time Frame: 6 months (at baseline and at month 6)]

Tracheostomy free survival [Time Frame: 12 months]

Incidence of Adverse Events [Time Frame: 12 months ( at screening, baseline, month 1-3-6-7-9-12)]

quality of life: Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) [Time Frame: 12 months (at baseline and month 6 and 12)]

disease progression [Time Frame: 12 months (at baseline and month 3, 6, 9, 12)]

Secondary ID(s)

FeTr-ALS

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Campus Bio-Medico University

University of Florence

University of Modena and Reggio Emilia

Catholic University of the Sacred Heart

Azienda Ospedaliera di Perugia

Azienda Ospedaliero-Universitaria Careggi

University of Chieti