Intestinal Microbiome Transplant for ALS

Overseen By Richard Bedlack, MD, PhD

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Duke University

Must be taking: Riluzole, Edaravone, Tofersen

Must not be taking: Probiotics, Nutraceuticals, Antibiotics

Disqualifiers: Pregnancy, Food allergy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 7

Duration: 8 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called MTP-101C for individuals with ALS, a disease affecting nerve cells in the brain and spinal cord, leading to muscle control issues. The researchers aim to determine if altering gut bacteria can impact the disease, using a capsule-based treatment (intestinal microbiome transplant) following antibiotics and a bowel cleanse. Participants will attend in-person and phone check-ins to monitor their safety and any symptom changes. Individuals with fast-progressing ALS who can still swallow capsules may be suitable for this study. As a Phase 1, Phase 2 trial, this research focuses on understanding the treatment's effects and measuring its effectiveness in an initial, smaller group, offering participants a chance to contribute to groundbreaking ALS research.

Will I have to stop taking my current medications?

The trial requires that if you are taking riluzole, edaravone, or tofersen, you must be on a stable dose for 30 days before the trial and continue it during the study. You cannot take probiotics, nutraceuticals, or herbal remedies within 2 weeks of screening, or antibiotics within 3 months of screening.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that fecal microbiota transplantation (FMT) is generally safe for people with ALS. One study found that FMT was well-tolerated and helped slow the loss of function in some patients. Another study involving 27 ALS patients reported no major side effects from FMT, although it did not significantly slow the disease's progression based on certain measures. Overall, these findings suggest that the treatment is relatively safe and may offer some benefits. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for ALS?

Unlike the standard treatments for ALS, which mainly focus on managing symptoms with medications like riluzole and edaravone, MTP-101C takes a completely different approach by targeting the gut microbiome. Researchers are excited about MTP-101C because it uses a freeze-dried encapsulated universal donor microbiome-based formulation, which could potentially modify disease progression by influencing the body's immune and inflammatory responses. This novel treatment is administered orally through capsules, making it a non-invasive option. By altering the intestinal microbiome, MTP-101C offers a unique mechanism that could complement or enhance the effects of existing therapies.

What evidence suggests that this treatment might be an effective treatment for ALS?

Research has shown that fecal microbiota transplantation (FMT) can restore the balance of gut bacteria, potentially improving conditions like ALS. Studies have found that FMT greatly enhances gut health by introducing beneficial bacteria. For ALS, a disease affecting the nerves, a healthier gut may help manage symptoms or slow disease progression. Early findings suggest FMT might alter disease development, but more research is needed to confirm these effects specifically for ALS. In this trial, participants will receive MTP-101C, a freeze-dried encapsulated universal donor microbiome-based formulation, to promote a healthier internal environment. The treatment transfers healthy bacteria from a donor to the patient's gut. ^{1 5 6 7 8}

Who Is on the Research Team?

Richard Bedlack, MD, PhD

Principal Investigator

Duke University

Are You a Good Fit for This Trial?

This trial is for people with ALS who can attend four in-person visits and provide blood and stool samples. They must be able to undergo antibiotic conditioning and bowel preparation before receiving the investigational product, MTP-101C.

Inclusion Criteria

I am of child-bearing age and my pregnancy test was negative.

I can sign and understand the consent form.

If sexually active, must agree to use contraceptive or abstinence for duration of treatment

Exclusion Criteria

I have been treated with MTT before.

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

I have not taken antibiotics in the last 3 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Antibiotic Conditioning

Participants undergo antibiotic conditioning with oral vancomycin and neomycin followed by a bowel preparation

10 days

1 visit (in-person)

Treatment

Participants ingest MTP-101C capsules daily following antibiotic conditioning

8 weeks

4 visits (in-person), 4 visits (phone)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

2 visits (phone)

What Are the Treatments Tested in This Trial?

Interventions

• MTP-101C

Trial Overview The study tests an intestinal microbiome transplant called MTP-101C over a period of 24 weeks. Participants will have clinical evaluations, phone check-ins for safety, and provide samples to monitor changes in their neurofilament light chain and gut microbiome.

How Is the Trial Designed?

Group I: MTP-101C administration Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Duke University

Lead Sponsor

Trials

2,495

Recruited

5,912,000+

University of Minnesota

Collaborator

Trials

1,459

Recruited

1,623,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07017946?term=intestinal%20microbiota%20transplantation&viewType=Table&rank=8

NCT07017946 | Intestinal Microbiome Transplant in ALS This is a 24-week Phase I/II study of MTT in people with Amyotrophic Lateral Sclerosis. All participants will be evaluated clinically in person at weeks 0, 4, ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11610222/

Effect of fecal microbiota transplantation on patients with ... Fecal microbiota transplantation (FMT) has emerged as a promising method for restoring intestinal microbial ecology [19], widely recognized for ...

3. tandfonline.com tandfonline.com/doi/full/10.1080/19490976.2024.2353396

Fecal microbiota transplantation significantly improved ... Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that leads to respiratory failure, and eventually death.

4. withpower.com withpower.com/trial/phase-2-amyotrophic-lateral-sclerosis-6-2025-d507c

Intestinal Microbiome Transplant for ALS · Info for Participants 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.

5. clinicaltrials.gov clinicaltrials.gov/study/NCT03766321

NCT03766321 | Fecal Microbiota Transplantation Effect on ... This interventional study aims at evaluating the biological and disease-modifying effects of Fecal Microbiota Transplant (FMT) in patients affected by ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT03766321

Fecal Microbiota Transplantation Effect on Amyotrophic ... This interventional study aims at evaluating the biological and disease-modifying effects of Fecal Microbiota Transplant (FMT) in patients affected by ...

7. gut.bmj.com gut.bmj.com/content/73/Suppl_2/A366

IDDF2024-ABS-0384 Efficacy and safety of fecal ... FMT showed efficacy and safety for ALS with slower functional decline. FMT might improve bulbar and respiratory function and ameliorate life quality.

8. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39617896/

Effect of fecal microbiota transplantation on patients with ... In this clinical trial involving 27 sporadic ALS patients, FMT did not significantly slow the decline in ALSFRS-R score.