Gut Microbial Transplantation in Pediatric Inflammatory Bowel Diseases

NCT01560819 | Completed Phase 1 Results DMC

• 1 other identifier: 2011-233
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

Inflammatory bowel disease (IBD) is a chronic, debilitating, relapsing inflammatory disorder affecting the gastrointestinal tract which does not have a medical cure. IBD consists of 2 different forms: Crohn's Disease (CD) and Ulcerative Colitis (UC). In the last 2 decades, Gut Microbial Transplantation (GMT), also known as fecal transplantation, has been used as a treatment option for Clostridium difficile colitis and UC. The literature supports strong evidence for the plausibility of using GMT for patients with IBD associated colitis, especially for patients with UC. This research will be conducted in the Helen DeVos Children's Hospital (HDVCH) Pediatric gastrointestinal outpatient clinic. A pilot study of ten patients will be conducted to evaluate if GMT improves clinical symptoms in patients with IBD. Patients with IBD colitis (UC and CD with colonic involvement only) will be approached for GMT as a treatment option for their disease. Each subject will undergo 5 sessions (1 session/day, and not necessarily on consecutive days) of GMT within a period of 10 days. Post treatment evaluation will be done at their regularly scheduled clinic follow up. Healthy donors \>18 years of age will be chosen by the family, inclusive of immediate family members and friends. Donors will be required to complete a screening questionnaire, provide medical history, and undergo blood and stool tests.

Conditions

Inflammatory Bowel Disease (IBD)

Keywords

ulcerative colitis, gut microbial transplantation

Outcome Measures

Primary Outcomes (1)

• Clinical Response

  Clinical response (i.e. improvement in Pediatric Ulcerative Colitis Activity Index (PUCAI) score by greater than or equal to 15 points from baseline) at 4 weeks following GMT treatment

  4 weeks following GMT Treatment

Study Arms (1)

Study Participants

EXPERIMENTAL

Participants did not receive any bowel preparation before Gut Microbial Transplantation (GMT). Audio-visual aids were used to help reduce participants' anxiety about GMT.

Drug: Gut Microbial Transplantation

Interventions

Gut Microbial Transplantation DRUG

Each participant received Gut Microbial Transplantation (GMT) as retention enema over a period of 1 hour (60mL enema every 15 minutes) daily for 5 days. Although 240mL of GMT solution was prepared for each participant, the final administered dose was dependent on the subject's comfort and willingness to proceed with the next enema, which was assessed after each enema infusion. Subjects were monitored for 30 minutes after GMT for any immediate adverse events and discharged.

Also known as: GMT

Study Participants

Eligibility Criteria

• Age: 7 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Children ages \>7 and ≤21 years of age with established diagnosis of Inflammatory Bowel Disease (IBD) colitis (patient with ulcerative colitis (UC) or patients with crohn's disease (CD) with colonic involvement only)
• Have clinical disease (mild to moderate disease: 10≤ Pediatric Ulcerative Colitis Activity Index (PUCAI) \<65)
• Have stable disease activity and therapy for two months prior to Gut Microbial Transplantation (GMT) procedure.

You may not qualify if:

• Fulminant colitis
• Indication or scheduled for surgery
• Pregnancy
• Use of probiotic supplements during the study period (subjects who have stopped use of probiotic supplements will be eligible as long as they stop taking it 2 weeks prior to Day 1 of GMT)
• anemia (hemoglobin \< 6.0 g/dL) in last one month
• Graft versus host disease (GVHD)
• Severely immunocompromised - defined as
• History of opportunistic infection (tuberculosis, Pneumocystis jirovecii or systemic fungal infections) in last one year or
• Neutropenia: Absolute neutrophil count (ANC) \<500
• Major intra-abdominal surgery within 90 days prior to Day 1 of GMT
• Administration of any investigational drug within 30 days prior to Day 1 of GMT.
• Have received infliximab or other tumor necrosis factor (TNF) inhibitors within 2 months prior to Day 1 of GMT or are expected to receive such therapy within 1 month post final GMT.
• Based on patient's and parents'/guardian's decision
• Will be chosen from immediate adult (≥18 years) family members or close friends
• Positive or abnormal screening test to include:

Sponsors & Collaborators

• Corewell Health West: lead

Study Sites (1)

Helen DeVos Children's Hospital of Spectrum Health Hospitals

Grand Rapids, Michigan, 49503, United States

Location

Related Publications (1)

• Kunde S, Pham A, Bonczyk S, Crumb T, Duba M, Conrad H Jr, Cloney D, Kugathasan S. Safety, tolerability, and clinical response after fecal transplantation in children and young adults with ulcerative colitis. J Pediatr Gastroenterol Nutr. 2013 Jun;56(6):597-601. doi: 10.1097/MPG.0b013e318292fa0d.

  PMID: 23542823 RESULT

MeSH Terms

Conditions

Inflammatory Bowel Diseases; Colitis, Ulcerative

Condition Hierarchy (Ancestors)

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

Limitations and Caveats

This short-term pilot study is limited by its small sample size; therefore, studies with a larger sample size and longer follow-up period will be required to determine true efficacy of GMT in patients with ulcerative colitis (UC).

Results Point of Contact

• Title: Dr. Sachin Kunde
• Organization: Helen DeVos Children's Hospital of Spectrum Health Hospitals

sachin.kunde@helendevoschildrens.org

616-267-2100

Study Officials

• Sachin Kunde, MD, MPH

  Helen DeVos Children's Hospital of Spectrum Health Hospitals

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

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: March 20, 2012
• First Posted: March 22, 2012
• Study Start: March 1, 2012
• Primary Completion: December 1, 2012
• Study Completion: June 1, 2013
• Last Updated: June 2, 2023
• Results First Posted: December 4, 2013

Record last verified: 2023-05

Locations

US (1)