Amyotrophic Lateral SclerosisPhase 1IASONCT05889572
Safety and Gut Microbiota Analysis of an Oral Microbiotherapy in Patients With Amyotrophic Lateral Sclerosis
A Phase 1 study testing MaaT033 in people with Amyotrophic Lateral Sclerosis and ALS, run by MaaT Pharma at 2 sites in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, ALS
- What is being tested?
- MaaT033
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 2 sitesFrance
- Study dates
- 8 Jun 2023to 22 Jul 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 22Show fewer
- 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
Trial locations and contacts
2 sites in 1 country.
France2 sites
Centre Hospitalier Universitaire de Lille - CIC
Lille, 59037
Hôpital de la Pitié-Salpêtrière - CIC Neuroscience
Paris, 75013
About this study
Official title: Safety, Tolerability and Gut microbIota AnalysiS of an Oral Microbiotherapy in Amyotrophic Lateral Sclerosis; an Open-label Phase 1b Pilot Trial
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.
Read the 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.
- Conditions
- Amyotrophic Lateral Sclerosis, ALS
- Being tested
- MaaT033
- Keywords
- Microbiotherapy, Amyotrophic Lateral Sclerosis, Neurodegenerative disease, ALS
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Neurodegenerative Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MaaT033Drug
MaaT033 is a Microbiome Ecosystem Therapy (MET), composed of allogeneic, full-ecosystem pooled biotherapeutic gut microbiota.
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: MaaT033 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Day 84
Secondary outcomes (3)
Changes in gut microbiota profile
Analysis of fecal samples to assess gut microbiota alpha- and beta-diversity indices
Measured: 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
- 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.
Measured: From Day -5 to Day 84
Changes in levels of fecal calprotectin
Changes from baseline (Day -5) of fecal calprotectin at Day 10, Day 28, Day 56 and Day 84
Measured: From Day -5 to Day 84
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2023
- Study start8 Jun 2023
- Primary completion22 Jul 2024
- Study completion22 Jul 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- MaaT Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Gaelle Bruneteau, MD, PhD, Principal investigator, Hôpital de la Pitié-Salpêtrière - CIC Neuroscience
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05889572
- Sponsor's protocol ID
- MPNS01
- Acronym
- IASO
- Status last verified
- Jun 2025
- First submitted
- 17 Apr 2023
- Last updated
- 15 Jun 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety, Tolerability and Gut microbIota AnalysiS of an Oral Microbiotherapy in Amyotrophic Lateral Sclerosis; an Open-label Phase 1b Pilot Trial
- ClinicalTrials.gov ID
- NCT05889572
- Acronym / sponsor ID
- IASO / MPNS01
- Conditions
- Amyotrophic Lateral Sclerosis, ALS
- Keywords
- Microbiotherapy, Amyotrophic Lateral Sclerosis, Neurodegenerative disease, ALS
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- MaaT PharmaIndustry (pharma, biotech, devices)
- Study start
- 8 Jun 2023 (actual)
- Primary completion
- 22 Jul 2024 (actual)
- Study completion
- 22 Jul 2024 (actual)
- First posted
- 5 Jun 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MaaT033 | Experimental | Drug: MaaT033Route 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 |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (22 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MaaT033MaaT033 is a Microbiome Ecosystem Therapy (MET), composed of allogeneic, full-ecosystem pooled biotherapeutic gut microbiota. | Drug | MaaT033 |
Primary outcome
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
3 secondary outcomes
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)
Changes in levels of biomarkers in blood
Time frame: From Day -5 to Day 84
Changes in levels of fecal calprotectin
Time frame: From Day -5 to Day 84
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jun 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gaelle Bruneteau, MD, PhD principal investigator
Hôpital de la Pitié-Salpêtrière - CIC Neuroscience
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MaaT033 in people with Amyotrophic Lateral Sclerosis and ALS, run by MaaT Pharma at 2 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jun 2025.