Fecal Microbiota Transplant (FMT) in Pediatric Active Ulcerative Colitis and Pediatric Active Crohn's Colitis

NCT02330653 | Completed Phase 1 Results DMC FDA Drug

• 1 other identifier: P00014876
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

The primary aims of this phase I/II, randomized, placebo controlled study are the assessment of safety and tolerability of universal donor FMT compared to placebo in pediatric and young adult subjects (ages 5 years through 30 years) with active ulcerative colitis (UC) or active Crohn's colitis (CD) who have failed, are intolerant to, or have refused traditional first-line maintenance therapy. Secondary objectives include the identification biomarkers in both donor and recipient that may confer a clinical response and to establish whether or not ongoing FMT maintenance therapy is required for maintenance of clinical benefit in pediatric UC or pediatric CD.

Conditions

Inflammatory Bowel Diseases; Ulcerative Colitis; Crohn Disease

Keywords

Colitis; Fecal Microbiota Transplant; Ulcerative colitis; inflammatory bowel disease; Crohn's Disease

Outcome Measures

Primary Outcomes (1)

• 1. Safety and Tolerability of Universal Donor FMT Compared to Placebo: FMT-related Adverse Events Grade 2 or Above

  Number of participants with FMT-related adverse events grade 2 or above experienced in each arm.

  At 8 weeks after start of FMT up to 6 months post treatment, an average of 10 months

Secondary Outcomes (4)

• Remission of Disease

  At all intermediate timepoints until 6 month follow up post intervention, an average of 10 months

• Improvement in Inflammatory Biomarkers

  At End of Treatment (8 weeks) and at 6 month post treatment

• Percentage of Donor Microbiome Present in Transplant Recipient

  At two weeks and seven weeks post induction enema

• Number of Participants With Improvement in Disease Activity

  At 8 weeks after start of FMT

Study Arms (2)

Fecal Microbiota Transplant (FMT)

EXPERIMENTAL

Induction retention enema for the first week of treatment followed by once weekly administration of 15 capsules of study treatment (the equivalent of 7.5 grams of human stool) will be (administered within 60 minutes of thawing once weekly) for 7 weeks. After completing 8 weeks of blinded study treatment, study subjects on FMT who have shown improvement will be given the option to receive open-label maintenance FMT weekly for an additional 8 weeks of once weekly FMT capsule administration.

Biological: Fecal Microbiota Transplant (FMT)

Placebo

PLACEBO COMPARATOR

Induction placebo enema for the first week of treatment followed by once weekly administration of 15 capsules of study placebo (administered within 60 minutes of thawing once weekly) for 7 weeks. After completing 8 weeks of blinded study placebo, study subjects on placebo who DO NOT demonstrate improvement will be given the option to receive open-label maintenance FMT weekly for an additional 8 weeks, beginning with a FMT induction enema followed by 7 weeks of weekly FMT capsule administration.

Biological: Placebo

Interventions

Fecal Microbiota Transplant (FMT) BIOLOGICAL

The study intervention consists of frozen, bottled or encapsulated fecal microbiota preparations that have been screened and prepared to a uniform and rigorous standard by OpenBiome. FMT is performed by patients receiving a retention enema and swallowing capsules, introducing stool from a healthy donor into their intestinal tract.

Also known as: Screened, healthy human donor stool

Fecal Microbiota Transplant (FMT)

Placebo BIOLOGICAL

Placebo administration will consist of both a placebo retention enema and placebo capsules.

Placebo

Eligibility Criteria

• Age: 5 Years - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Two initial subsets will be created: an initial subset of 20 subjects limited to patients greater than or equal to 12 years of age with mild to moderate ulcerative colitis (i.e., PUCAI \< 65) patients with mild to moderate Crohn's disease (i.e., PDCAI less than or equal to 30).
• All patients must satisfy below criteria:
• Have UC (PUCAI \>9) or CD (PDCAI \>10) and have failed, are intolerant to, or have refused first-line maintenance therapy.
• Have had visual or histologic evidence of inflammation confirmed through colonoscopy no more than 105 days prior to randomization.
• Have negative test results for Hepatitis B (HBV), Hepatitis C (HCV), and Human Immunodeficiency Virus (HIV).
• Have a negative urine hCG test if female of childbearing potential.
• Able to swallow antibiotic, FMT or placebo capsules.
• Able to give informed consent and/or assent as appropriate (patients 12-17 will be asked to provide written assent, patients 5-11 will be observed for assent or dissent behaviorally, or with verbal/written communication)
• Willing and able to participate in the study requirements, including serial stool collection, survey completion and clinic visits.
• Willing to undergo telephone follow-up to assess for safety and adverse events.
• Must be free of any known food allergy.
• Agrees and willing to have an enema for purposes of induction therapy.
• Patients who have disease that has required other medications (including steroids, immunosuppressives, and biologics) will be included.

You may not qualify if:

• Patients with extensive and/or severe CD (i.e. fistulizing disease, abscess, small bowel obstruction, fevers).
• Patients in a clinical remission (PUCAI \< 9) or (PCDAI \<10).
• Patients with recent (within 4 weeks) dose change of biologics, 5-ASA, steroids or immunomodulators
• Patients considered to have toxic megacolon.
• Patients with a known drug allergy to vancomycin, metronidazole or polymyxin.
• Patients with a history of aspiration, gastroparesis, surgery involving the upper gastrointestinal tract (that might affect upper gastrointestinal motility) or unable to swallow pills.
• Patients with esophageal dysmotility or swallowing dysfunction.
• Patients with known food allergies.
• Patients with positive test results for HBV, HCV, or HIV.
• Female patients with a positive test result on a urine hCG test.
• Patients unwilling or unable to give consent or participate in all study requirements.
• Patients unable or unwilling to receive a retention enema for purposes of induction therapy.
• Patients with recent (within 6 weeks) systemic antibiotic use.
• Patients who have testing consistent with active clostridium difficile.
• Patients with known prior experience with donor FMT.

Sponsors & Collaborators

• Stacy A. Kahn: lead

Study Sites (1)

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

MeSH Terms

Conditions

Inflammatory Bowel Diseases; Colitis, Ulcerative; Crohn Disease; Colitis

Interventions

Fecal Microbiota Transplantation

Condition Hierarchy (Ancestors)

Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases; Colonic Diseases

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics

Results Point of Contact

• Title: Dr. Stacy A. Kahn
• Organization: Boston Children's Hospital

stacy.kahn@childrens.harvard.edu

617-355-6058

Study Officials

• Stacy A Kahn, MD

  Boston Childrens Hospital - GI & Nutrition

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Associate Director of the Inflammatory Bowel Disease Center

Study Record Dates

• First Submitted: December 29, 2014
• First Posted: January 5, 2015
• Study Start: November 1, 2015
• Primary Completion: April 8, 2019
• Study Completion: April 8, 2019
• Last Updated: October 16, 2023
• Results First Posted: October 16, 2023

Record last verified: 2023-10

Locations

US (1)