Safety and Gut Microbiota Analysis of an Oral Microbiotherapy in Patients With Amyotrophic Lateral Sclerosis

NCT05889572 | Completed Phase 1 DMC

• 1 other identifier: MPNS01
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 2

Brief Summary

The purpose of this pilot study is to assess the safety and tolerability of multiple doses of MaaT033 in ALS patients and to analyze the gut microbiota composition and evolution before considering a larger randomized controlled efficacy study.

Conditions

Amyotrophic Lateral Sclerosis; ALS

Keywords

Microbiotherapy; Amyotrophic Lateral Sclerosis; Neurodegenerative disease; ALS

Outcome Measures

Primary Outcomes (1)

• Safety and tolerability: Incidence of Treatment Emergent Adverse Events (TEAE) grade >3, according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0

  To assess the safety and tolerability of MaaT033 treatment

  Day 84

Secondary Outcomes (3)

• Changes in gut microbiota profile

  From Day -5 to Day 84 (at Day -5, Day 10, Day 28, Day 56 and Day 84)

• Changes in levels of biomarkers in blood

  From Day -5 to Day 84

• Changes in levels of fecal calprotectin

  From Day -5 to Day 84

Study Arms (1)

MaaT033

EXPERIMENTAL

Route of administration: oral (capsule) Between D-5 to D-1: Bowel preparation with Macrogol and Rifamixin Between D1 to D28: MaaT033 treatment period 1 Between D28 to D56: MaaT033 treatment period 2

Drug: MaaT033

Interventions

MaaT033 DRUG

MaaT033 is a Microbiome Ecosystem Therapy (MET), composed of allogeneic, full-ecosystem pooled biotherapeutic gut microbiota.

MaaT033

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female subjects aged between18 and 80 years
• ALS meeting the revised El Escorial criteria for possible, probable, laboratory-supported probable, or definite ALS (familial or sporadic)
• Time since first motor deficit at screening: at least 6 months, up to 24 months
• Slope of progression of ALS Functional Rating Scale - revised (ALSFRS-R) from date of symptom onset to date of screening test (ΔFS/number of months) between \[0.4 and 1.1\]
• Able to swallow study treatments (including capsules without opening or chewing them) as per the investigator's assessment
• SVC (Slow Vital Capacity) equal to or greater than 70% of the predicted normal value for sex, height, and age at the screening visit
• If taking riluzole, subject must be on a stable dose for ≥30 days
• Signature of written informed consent by subject

You may not qualify if:

• Subjects with a non-invasive ventilation, a tracheotomy and /or a gastrostomy
• Known autoimmune diseases, inflammatory disorders (SLE, Rheumatoid arthritis, connective tissue disorder) or chronic infections (HIV, hepatitis B, or C infection, Tuberculosis)
• Known hypersensitivity to rifaximin or macrogol or any of its components
• Known allergy or intolerance to trehalose, maltodextrin or Polyethylene Glycol (PEG)
• Documented hepatic impairment (Alanine Transaminase/ Aspartate Transaminase \> 5N)
• Subject with white blood cells \< 4000/ mm3; Polynuclear neutrophils \< 1.5 G/ L
• Active infection requiring systemic antimicrobial therapy within 2-week prior to screening visit
• Active infection requiring systemic antimicrobial therapy between screening and baseline
• Medical condition requiring proton pump inhibitors (PPIs)
• Gastrointestinal obstruction or perforation
• Any gastro-intestinal bleeding in the past 3 months
• Gastric emptying disorders (gastroparesis)
• Toxic megacolon
• Severe forms of inflammation of the intestinal tract, including Crohn's disease and ulcerative colitis
• Severe vital organ dysfunctions unrelated to ALS and not compatible with experimental treatment, as per the investigator's assessment

Sponsors & Collaborators

• MaaT Pharma: lead

Study Sites (2)

Centre Hospitalier Universitaire de Lille - CIC

Lille, 59037, France

Location

Hôpital de la Pitié-Salpêtrière - CIC Neuroscience

Paris, 75013, France

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Neurodegenerative Diseases

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Gaelle Bruneteau, MD, PhD

  Hôpital de la Pitié-Salpêtrière - CIC Neuroscience

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 17, 2023
• First Posted: June 5, 2023
• Study Start: June 8, 2023
• Primary Completion: July 22, 2024
• Study Completion: July 22, 2024
• Last Updated: June 15, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

FR (2)