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Gut Microbial Transplantation in Pediatric Inflammatory Bowel Diseases (GMT)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Spectrum Health Hospitals
ClinicalTrials.gov Identifier:
NCT01560819
First received: March 20, 2012
Last updated: December 4, 2013
Last verified: December 2013
Results First Received: July 15, 2013  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Inflammatory Bowel Disease (IBD)
Intervention: Drug: Gut Microbial Transplantation

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
An open-label, uncontrolled, single-center, prospective pilot study was conducted at Helen DeVos Children's Hospital's outpatient gastroenterology center. Participants were enrolled between April and December 2012. Each participant took part in the study for 6 weeks. The study was not advertised.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Fifteen patients with ulcerative colitis (UC) were assessed for eligibility; all showed interest in the study. Ten participants met the eligibility criteria and the donors were identified by the participants.

Reporting Groups
  Description
Study Participants Ten participants between the ages of 7 to 21 years with mild-to moderate UC (pediatric UC activity index [PUCAI] between 15 and 65) were enrolled in the study. PUCAI is a validated tool to measure disease activity in pediatric UC based on clinical symptoms: a score of <10 = remission; 10-34 = mild disease; 35-64 = moderate disease; 65-85 = severe disease. Participants had stable disease activity and medical treatment for UC for 2 months prior to enrollment. Participants' ongoing treatment for UC was not changed. None of the subjects had concurrent C difficile infection.
Donors Healthy donors (>18 years of age) were chosen by the participants. Donors were required to complete a screening questionnaire, provide medical history, and undergo blood and stool tests.

Participant Flow:   Overall Study
    Study Participants     Donors  
STARTED     10     10  
COMPLETED     9     10  
NOT COMPLETED     1     0  
Physician Decision                 1                 0  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Study Participants Ten participants between the ages of 7 to 21 years with mild-to moderate UC (pediatric UC activity index [PUCAI] between 15 and 65) were enrolled in the study. PUCAI is a validated tool to measure disease activity in pediatric UC based on clinical symptoms: a score of <10 = remission; 10-34 = mild disease; 35-64 = moderate disease; 65-85 = severe disease. Participants had stable disease activity and medical treatment for UC for 2 months prior to enrollment. Participants' ongoing treatment for UC was not changed. None of the subjects had concurrent C difficile infection.
Donors Healthy donors (>18 years of age) were chosen by participants. Donors were required to complete a screening questionnaire, provide medical history, and undergo blood and stool tests.
Total Total of all reporting groups

Baseline Measures
    Study Participants     Donors     Total  
Number of Participants  
[units: participants]
  10     10     20  
Age, Customized  
[units: Years]
Mean ± Standard Deviation
  15.2  ± 4.4     39.6  ± 12.7     27.4  ± 15.6  
Gender  
[units: Participants]
     
Female     4     4     8  
Male     6     6     12  
Disease Extent [1]
[units: Participants]
     
Extensive     1     0     1  
Left-sided     1     0     1  
Pancolitis     6     0     6  
Proctitis     2     0     2  
Disease Activity before Treatment [2]
[units: Participants]
     
0-9: Remission     0     0     0  
10-34: Mild Disease     3     0     3  
35-64: Moderate Disease     7     0     7  
65-85: Severe Disease     0     0     0  
[1] Disease extent: extensive = involving left and transverse colon; left-sided = involving rectum and sigmoid or descending colon; pancolitis = involving the entire colon; proctitis = involving rectum.
[2] Disease activity: measured by pediatric ulcerative colitis activity index (PUCAI). PUCAI is a validated tool to measure disease activity in pediatric UC based on clinical symptoms: a score of <10 = remission; 10-34 = mild disease; 35-64 = moderate disease; 65-85 = severe disease.



  Outcome Measures

1.  Primary:   Clinical Response   [ Time Frame: 4 weeks following GMT Treatment ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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).


  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Dr. Sachin Kunde
Organization: Helen DeVos Children's Hospital of Spectrum Health Hospitals
phone: 616-267-2100
e-mail: sachin.kunde@helendevoschildrens.org


Publications of Results:

Responsible Party: Spectrum Health Hospitals
ClinicalTrials.gov Identifier: NCT01560819     History of Changes
Other Study ID Numbers: 2011-233
Study First Received: March 20, 2012
Results First Received: July 15, 2013
Last Updated: December 4, 2013
Health Authority: United States: Food and Drug Administration