NCT03720392

Fecal Microbiota Transplantation (FMT) in Recipients After Allogeneic Hematopoietic Cell Transplantation (HCT)

Terminated Phase 2 Results posted Last updated 1 December 2021

What this trial tests

Phase 2 trial testing FMT in Allogeneic Hematopoietic Cell Transplantation (HCT) in 8 participants. Terminated before completion.

Timeline

15 January 2019

Primary endpoint
28 May 2020

28 May 2020

Quick facts

Lead sponsor	Zachariah Michael DeFilipp
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	double
Primary purpose	treatment
Enrollment	8
Start date	15 January 2019
Primary completion	28 May 2020
Estimated completion	28 May 2020
Sites	1 location across United States

Drugs / interventions tested

• FMT — full drug profile →
• Placebo

Conditions studied

• Allogeneic Hematopoietic Cell Transplantation (HCT) — all drugs for Allogeneic Hematopoietic Cell Transplantation (HCT) →

Sponsor

Zachariah Michael DeFilipp

Who can join

Adults 18 to 80, any sex, with Allogeneic Hematopoietic Cell Transplantation (HCT). Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants With Acute Graft-Versus-Host-Disease (GvHD) Secondary · 6 months

The cumulative incidence of overall grades II-IV and grades III-IV acute GVHD will be assessed through six months after last dose of FMT or placebo. Acute GVHD will be assessed using the Mount Sinai Acute GVHD International Consortium (MAGIC) criteria.

Group	Value	95% CI
FMT Capsules	2
Placebo Capsules	0

Number of Participants With Non-relapse-mortality Secondary · 6 Months and 12 months

Non-relapse mortality (NRM) is defined as the time from first dose of FMT or placebo to death without relapse or progression or underlying disease.

at 6 months

Group	Value	95% CI
FMT Capsules	1
Placebo Capsules	0

at 12 months (cumulative)

Group	Value	95% CI
FMT Capsules	2
Placebo Capsules	0

Number of Participants With Infection at 100 Days Secondary · 100 Days

Group	Value	95% CI
FMT Capsules	2
Placebo Capsules	0

Number of Participants With Progression Free Survival at 12 Months Post-treatment Secondary · 12 Months

Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Progression-Free Survival (PFS) is defined as the time from first dose of FMT or placebo to the earlier of underlying disease progression or death due to any cause. PFS was assessed at 12 months for all participants.

Group	Value	95% CI
FMT Capsules	1
Placebo Capsules	1

Overall Survival Secondary · 12 Months

Overall survival is measured as the amount of time from the first dose of Fecal Microbiota Transplantation (FMT) or placebo, that participants in this study are still alive. Overall survival is reported as number of participants alive at 12 months.

Group	Value	95% CI
FMT Capsules	1
Placebo Capsules	2

Graft Versus Host Disease(GVHD)/ Relapse Free Survival (GRFS) Secondary · 12 Months

GVHD-Free/Relapse-Free Survival (GRFS) is measured as the amount of time from the first dose of Fecal Microbiota Transplantation (FMT) or placebo, that participants in this study are not diagnosed with GVHD, disease progression, or relapse. GRFS is reported as number of patients without GVHD, disease progression, or relapse at 12 months.

Group	Value	95% CI
FMT Capsules	0
Placebo Capsules	2

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse event data collection time frame was approximately 13 months, starting 1 week after allogeneic hematopoietic cell transplantation (HCT) until 12 months after the second fecal microbiota transplantation (FMT) or placebo administration.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

FMT Capsules

Serious: 1/3 (33%)

Deaths: 2/3

Placebo Capsules

Serious: 0/3 (0%)

Deaths: 1/3

Serious adverse events (1 terms)

Reaction	System	FMT Capsules	Placebo Capsules
Sepsis	Infections and infestations	—	—

Other adverse events (10 terms — click to expand)

Reaction	System	FMT Capsules	Placebo Capsules
Platelet count decreased	Investigations	—	—
Febrile neutropenia	Blood and lymphatic system disorders	—	—
Nausea	Gastrointestinal disorders	—	—
Neutrophil count decreased	Investigations	—	—
Abdominal pain	Gastrointestinal disorders	—	—
Diarrhea	Gastrointestinal disorders	—	—
Hypertension	Vascular disorders	—	—
Hyponatremia	Metabolism and nutrition disorders	—	—
Myalgia	Musculoskeletal and connective tissue disorders	—	—
Skin ulceration	Skin and subcutaneous tissue disorders	—	—

Most-reported serious reactions: Sepsis.

Data from ClinicalTrials.gov NCT03720392 adverse events section.

Sponsor's own description

This research study is studying the role fecal microbiota transplantation may play in post-Hematopoietic Cell Transplantation (HCT) recipients

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Modulating the microbiome to improve therapeutic response in cancer.
   McQuade JL, Daniel CR, Helmink BA, Wargo JA. · · 2019 · cited 283× · PMID 30712808 · DOI 10.1016/s1470-2045(18)30952-5
2. Introduction to host microbiome symbiosis in health and disease.
   Malard F, Dore J, Gaugler B, Mohty M. · · 2021 · cited 137× · PMID 33299088 · DOI 10.1038/s41385-020-00365-4
3. Fecal microbiota transplantation beyond Clostridioides difficile infections.
   Wortelboer K, Nieuwdorp M, Herrema H. · · 2019 · cited 111× · PMID 31201141 · DOI 10.1016/j.ebiom.2019.05.066
4. The Gut-CNS Axis in Multiple Sclerosis.
   Kadowaki A, Quintana FJ. · · 2020 · cited 72× · PMID 32650957 · DOI 10.1016/j.tins.2020.06.002
5. Adjunctive fecal microbiota transplantation in supportive oncology: Emerging indications and considerations in immunocompromised patients.
   Wardill HR, Secombe KR, Bryant RV, Hazenberg MD, et al · · 2019 · cited 48× · PMID 30940601 · DOI 10.1016/j.ebiom.2019.03.070
6. Impact of the microbiome on cancer progression and response to anti-cancer therapies.
   Buchta Rosean C, Feng TY, Azar FN, Rutkowski MR. · · 2019 · cited 32× · PMID 31202360 · DOI 10.1016/bs.acr.2019.03.005
7. Fecal Microbiota Transplantation for Treatment of Acute Graft-<i>versus</i>-Host Disease.
   Shouval R, Geva M, Nagler A, Youngster I. · · 2019 · cited 26× · PMID 34595408 · DOI 10.2991/chi.d.190316.002
8. Microbiota modification in hematology: still at the bench or ready for the bedside?
   Severyn CJ, Brewster R, Andermann TM. · · 2019 · cited 22× · PMID 31714965 · DOI 10.1182/bloodadvances.2019000365