Microbiota Transplant Therapy for Pulmonary Arterial Hypertension: Early Safety and Feasibility Study

NCT04884971 | Completed Phase 1 FDA Drug

• 1 other identifier: CV-2021-29604
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 1

Brief Summary

This pilot clinical trial will evaluate the initial safety and feasibility of intestinal microbiota transplantation (IMT) in patients with pulmonary arterial hypertension (PAH). This trial will inform development of future trials in treatment of PAH. Active drug in capsule form composed of freeze-dried, encapsulated intestinal microbiota from healthy donors will be administered to patients with PAH. This study will also allow for limited evaluation of pharmacokinetics in terms of donor microbiota engraftment and pharmacodynamics in terms of potential mechanisms. It will also allow for limited evaluation of cardiac endurance and function prior to and after IMT.

Conditions

Pulmonary Arterial Hypertension

Outcome Measures

Primary Outcomes (2)

• Frequency of Serious Adverse Events

  In order to assess the safety of the trial, the frequency of adverse events will be reported. Outcome will be reported as the mean number of serious adverse events per participant.

  6 months

• Proportion of IMT Compliance

  In order to assess the feasibility of the trial, the proportion of subjects taking 100% of the intestinal microbiota transplantation (IMT) doses per protocol will be reported. Outcome is reported as the percent of participants who consume 100% of IMT doses.

  6 months

Study Arms (1)

Microbiota Treatment Arm

EXPERIMENTAL

Participants will receive the encapsulated microbiota intervention daily for seven days and will be subsequently monitored for six months.

Drug: Intestinal microbiota transplant (IMT)

Interventions

Intestinal microbiota transplant (IMT) DRUG

Two size 00 capsules from a single lot will be taken daily. Approximately 2.0 x 10\^11 bacteria from a healthy donor are contained in each capsule.

Microbiota Treatment Arm

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of pulmonary arterial hypertension (PAH)
• On stable treatment for PAH for one month prior to enrollment
• Able to swallow capsultes
• Able to provide blood sample and fecal sample

You may not qualify if:

• Dysphagia to pills
• Clinically active inflammatory bowel disease
• Pregnancy or breastfeeding
• Life expectancy of \<6 months
• Presence of ileostomy or colostomy
• Taking immunosuppressants (calcineurin inhibitors, prednisone greater than or equal to 20mg/day, methotrexate, azathioprine, immunosuppressive biologics, JAK inhibitors)
• Neurotropenia (an absolute neurotrophil count \< 0.5 x 10\^9 cells/L)
• History of solid organ or bone marrow transplant
• Anticipated recurrent antibiotic use (participants with frequent urinary tract infections or sinusitis)
• History of severe anaphylactic food allergy
• History of celiac disease
• History of receiving cancer chemotherapy, immunotherapy, or radiation

Sponsors & Collaborators

• University of Minnesota: lead

Study Sites (1)

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Related Publications (1)

• Moutsoglou D, Blake M, Belhasan DC, Peichel G, Vang BM, Weir EK, Lopez S, Prins KW, Kabage AJ, Prisco SZ, Kremer BP, Khoruts A, Thenappan T. Microbiota Transplant Therapy Is Safe and Feasible in Pulmonary Arterial Hypertension. JACC Basic Transl Sci. 2025 Sep;10(9):101347. doi: 10.1016/j.jacbts.2025.101347. Epub 2025 Aug 12.

  PMID: 40803054 DERIVED

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Interventions

Fecal Microbiota Transplantation

Condition Hierarchy (Ancestors)

Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics

Study Officials

• Thenappan Thenappan, MD

  University of Minnesota Division of Cardiology

  PRINCIPAL INVESTIGATOR

• Kurt Prins, MD, PhD

  University of Minnesota Division of Cardiology

  PRINCIPAL INVESTIGATOR

• Edward Weir, MD

  University of Minnesota Division of Cardiology

  PRINCIPAL INVESTIGATOR

• Alexander Khoruts, MD

  University of Minnesota Division of Gastroenterology

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: May 7, 2021
• First Posted: May 13, 2021
• Study Start: November 3, 2021
• Primary Completion: December 31, 2023
• Study Completion: December 31, 2023
• Last Updated: February 12, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)