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Trial record 1 of 1 for:    MAAT033 ALS
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Safety and Gut Microbiota Analysis of an Oral Microbiotherapy in Patients With Amyotrophic Lateral Sclerosis (IASO)

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ClinicalTrials.gov Identifier: NCT05889572
Recruitment Status : Recruiting
First Posted : June 5, 2023
Last Update Posted : February 15, 2024
Sponsor:
Information provided by (Responsible Party):
MaaT Pharma

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.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis ALS Drug: MaaT033 Phase 1

Detailed Description:

This is a prospective, single arm, open-label study.

The target population includes subjects with a recent disease onset defined as the time from first motor deficit at screening of at least 6 months and up to 24 months and removing very rapid/slow progressors based on the ALS Functional Rating Scale - Revised (ALSFRS-R) progression slope.

After a screening period (clinical examination, blood sampling), subject will come for a baseline visit (clinical examination, blood and feces sampling) and to initiate a bowel preparation phase. Five days later, subject will come back to the study site (clinical examination, blood sampling) to initiate a first Maat033 treatment period of 28-day. Ten days after MaaT033 treatment initiation a remote visit is included (feces sampling) to check the subject safety/tolerability. After the first Maat033 treatment period, subject will come to the study site (clinical examination, blood and feces sampling) to initiate the second MaaT033 treatment period of 28-day. At the end of the second Maat033 treatment period subjects will come to the study site (clinical examination, blood and feces sampling) and start a 28-day follow-up period without treatment.

Study completion is defined when all subjects enrolled completed the study follow-up period (clinical examination, blood and feces sampling) or earlier if a subject discontinued the study.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety, Tolerability and Gut microbIota AnalysiS of an Oral Microbiotherapy in Amyotrophic Lateral Sclerosis; an Open-label Phase 1b Pilot Trial
Actual Study Start Date : June 8, 2023
Estimated Primary Completion Date : September 2024
Estimated Study Completion Date : December 2024


Arm Intervention/treatment
Experimental: MaaT033

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




Primary Outcome Measures :
  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 [ Time Frame: Day 84 ]
    To assess the safety and tolerability of MaaT033 treatment


Secondary Outcome Measures :
  1. Changes in gut microbiota profile [ Time Frame: From Day -5 to Day 84 (at Day -5, Day 10, Day 28, Day 56 and Day 84) ]
    Analysis of fecal samples to assess gut microbiota alpha- and beta-diversity indices

  2. Changes in levels of biomarkers in blood [ Time Frame: From Day -5 to Day 84 ]
    • Changes from baseline (Day -5) of neutrophil/ lymphocyte ratio, Interleukins (IL): IL-2, IL-6 and IL-8, Interferon gamma (IFNg), Tumor Necrosis Factor alpha (TNFa), Monocyte Chemoattractant Protein-1 (MCP-1), Transforming Growth Factor-beta (TGFb), the soluble subtype of CD14 (sCD14), C-reactive protein (CRP), erythrocyte sedimentation rate, plasma soluble Lipopolysaccharide Binding Protein (LBP), creatinin and serum Short-Chain Fatty Acids (SCFA) at Day 28, Day 56 and Day 84.
    • Changes from baseline (Day -5) of serum Neurofilament light (NfL) at Day 56 and Day 84.

  3. Changes in levels of fecal calprotectin [ Time Frame: From Day -5 to Day 84 ]
    Changes from baseline (Day -5) of fecal calprotectin at Day 10, Day 28, Day 56 and Day 84



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
  • Subjects with negative IgG serology for Epstein Barr virus (EBV)
  • Women of childbearing potential1 without effective contraceptive protection
  • Nursing or pregnant women
  • Any condition that, in the opinion of the investigator, may interfere with full participation in the study, including administration of study drug (and its preparation procedure) and attendance at required study visits; represent a significant risk to the subject; or interfere with the interpretation of study data
  • Enrollment in another trial or expanded access program that may interfere with this study
  • Guardianship/legal protection/curatorship of subjects
  • Vulnerable subjects such as: persons deprived of liberty, persons in intensive care units unable to provide informed consent prior to the procedure

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05889572


Contacts
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Contact: Juliette Jouve +33 4 28 29 14 00 contact@maat-pharma.com

Locations
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France
Centre Hospitalier Universitaire de Lille - CIC Recruiting
Lille, France, 59037
Contact: Véronique DANEL, MD         
Hôpital de la Pitié-Salpêtrière - CIC Neuroscience Recruiting
Paris, France, 75013
Contact: Gaelle Bruneteau, MD, PhD         
Sponsors and Collaborators
MaaT Pharma
Investigators
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Principal Investigator: Gaelle Bruneteau, MD, PhD Hôpital de la Pitié-Salpêtrière - CIC Neuroscience
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Responsible Party: MaaT Pharma
ClinicalTrials.gov Identifier: NCT05889572    
Other Study ID Numbers: MPNS01
First Posted: June 5, 2023    Key Record Dates
Last Update Posted: February 15, 2024
Last Verified: February 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by MaaT Pharma:
Microbiotherapy
Amyotrophic Lateral Sclerosis
Neurodegenerative disease
ALS
Additional relevant MeSH terms:
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Amyotrophic Lateral Sclerosis
Motor Neuron Disease
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases