Intestinal Microbiome Transplant in ALS

NCT07017946 | Recruiting Phase 1 FDA Drug

• 1 other identifier: Pro00118099
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

This is a 24-week study of intestinal microbiome transplant in people with ALS. All participants will be evaluated clinically in person for 4 visits. Blood and mailed/ fresh stool samples will also be collected. Blood samples will be used to determine changes in neurofilament light chain over time. Stool samples will be processed for microbiome analysis. Participants will have phone visits to further evaluate safety and tolerability. They will then undergo antibiotic conditioning and a standard bowel preparation before being assigned to the investigational product, MTP-101C .

Conditions

Amyotrophic Lateral Sclerosis ALS

Outcome Measures

Primary Outcomes (4)

• ALS Functional Rating Scale, Revised (ALSFRS-R)

  The ALSFRS-R is a quickly administered (five minute) ordinal rating scale (ratings 0-4) used to determine patients' assessments of their capability and independence in 12 functional activities. The total score ranges from 0 to 48, where higher scores indicate better function.

  Weeks 0, 4, 8, 12, 16, 20 and 24

• ALS Quality of Life (ALSAQ-5)

  The overall ALSAQ-5 score ranges from 0 (best imaginable health state) to 100 (worst imaginable health state).

  Weeks 0, 4, 12, and 24

• Neurofilament Light Chain levels

  Neurofilament light chain levels are elevated in the spinal fluid and the blood of patients with ALS and other neurodegenerative diseases, and higher levels predict more severe disease progression.

  Weeks 0, 4, 12 and 24

• The Gastrointestinal Symptom Rating Scale (GSRS)

  The Gastrointestinal Symptom Rating Scale (GSRS) is a 15-item questionnaire that can be used weekly to assess the severity of gastrointestinal symptoms experienced over the past week. The GSRS is designed to evaluate symptoms associated with common gastrointestinal disorders, such as reflux, abdominal pain, indigestion, diarrhea, and constipation. The questionnaire uses a seven-point Likert-type scale, with 1 representing no symptoms and 7 representing very troublesome symptoms. Higher scores on the subscales indicate more discomfort.

  Weeks 0, 4, 12, and 24

Secondary Outcomes (1)

• Microbiome composition

  Screening/Baseline, Week 4, Week 12, and Week 24

Study Arms (1)

MTP-101C administration

EXPERIMENTAL

MTP-101C is a freeze-dried encapsulated universal donor microbiome-based formulation. Participants will ingest 2 capsules daily for 3 days then 1 capsule daily. This is preceded by antibiotic conditioning with a combination of vancomycin and neomycin, followed by a bowel purgative with magnesium citrate depending upon underlying comorbidities.

Drug: MTP-101C

Interventions

MTP-101C DRUG

MTP-101C is a freeze-dried encapsulated formulation of fecal microbiota purified from the stool of healthy donors.

MTP-101C administration

Eligibility Criteria

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

You may qualify if:

• Diagnosis of ALS according to Gold Coast Criteria
• Age: 18+ years at enrollment
• Fast-progressing (ALSFRS-R change of at least 1.5 points per month between last 2 measurements at screening)
• Modestly but not severely affected (ALSFRS-R score at or above 24 at screening)
• Able to swallow capsules and expected to be able to for the duration of the trial (ALSFRS-R "swallowing" score of 3 or 4 at screening)
• Expected to survive for the duration of the trial
• Taking any combination of riluzole, edaravone, and/or tofersen at a stable dose for 30 days prior to screening, or not taking any of these and not expected to during the study.
• Capable of giving written consent.
• If sexually active, must agree to use contraceptive or abstinence for duration of treatment.
• Females of child-bearing age must have negative pregnancy test at screening.

You may not qualify if:

• Concurrent illness or laboratory abnormalities that could confound the measurement of ALS progression or the microbiome or interfere with the ability to complete the study.
• Taking probiotics, nutraceuticals, or herbal remedies within 2 weeks of screening
• Taking antibiotics within 3 months of screening.
• Taking any investigational study drug within 30 days of screening or five half-lives of the prior agent.
• Previous exposure to MTT.
• Pregnancy.
• Known specific food allergy with anaphylaxis
• Other co-morbid conditions that, in the opinion of the study investigator, place the participant at increased risk of complications, interfere with study participation or compliance, or confound study objectives.

Sponsors & Collaborators

• Duke University: lead
• University of Minnesota: collaborator

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27705, United States

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

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

Study Officials

• Richard Bedlack, MD, PhD

  Duke University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Richard Bedlack, MD, PhD

CONTACT

919-613-2681; ALSresearch@duke.edu

Michelle Ward, RN

CONTACT

919-613-2681; ALSresearch@duke.edu

Study Design

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

Model Details: All participants receive the investigational product.

Study Record Dates

• First Submitted: June 4, 2025
• First Posted: June 12, 2025
• Study Start: January 27, 2026
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): November 1, 2026
• Last Updated: April 13, 2026

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)