Trial Outcomes & Findings for Gut Microbial Transplantation in Pediatric Inflammatory Bowel Diseases (NCT NCT01560819)
NCT ID: NCT01560819
Last Updated: 2023-06-02
Results Overview
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
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
20 participants
Primary outcome timeframe
4 weeks following GMT Treatment
Results posted on
2023-06-02
Participant Flow
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.
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.
Participant milestones
| Measure |
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.
|
|---|---|---|
|
Overall Study
STARTED
|
10
|
10
|
|
Overall Study
COMPLETED
|
9
|
10
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
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.
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|---|---|---|
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Overall Study
Physician Decision
|
1
|
0
|
Baseline Characteristics
Gut Microbial Transplantation in Pediatric Inflammatory Bowel Diseases
Baseline characteristics by cohort
| Measure |
Study Participants
n=10 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
n=10 Participants
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
n=20 Participants
Total of all reporting groups
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|---|---|---|---|
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Age, Customized
|
15.2 Years
STANDARD_DEVIATION 4.4 • n=99 Participants
|
39.6 Years
STANDARD_DEVIATION 12.7 • n=107 Participants
|
27.4 Years
STANDARD_DEVIATION 15.6 • n=206 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
12 Participants
n=206 Participants
|
|
Disease Extent
Extensive
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Disease Extent
Left-sided
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Disease Extent
Pancolitis
|
6 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
Disease Extent
Proctitis
|
2 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Disease Activity before Treatment
0-9: Remission
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Disease Activity before Treatment
10-34: Mild Disease
|
3 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
|
Disease Activity before Treatment
35-64: Moderate Disease
|
7 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
7 Participants
n=206 Participants
|
|
Disease Activity before Treatment
65-85: Severe Disease
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: 4 weeks following GMT TreatmentPopulation: One participant showed intolerance to the treatment (immediate leaking of enema) and was not included in post-treatment disease activity evaluation.
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
Outcome measures
| Measure |
Study Participants
n=9 Participants
Ten participants between the ages of 7 to 21 years with mild-to moderate ulcerative colitis (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.
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|---|---|
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Clinical Response
|
9 Participants
|
Adverse Events
Study Participants
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Study Participants
n=10 participants at risk
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. 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.
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|---|---|
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Gastrointestinal disorders
Bloating/flatulance
|
90.0%
9/10 • Number of events 11 • Adverse events (AEs) were recorded during and until 4 weeks after GMT using a symptom diary that participants completed. Common Terminology Criteria for Adverse Events and Toxicity Grading Guidance from Vaccine Clinical Trials (FDA, 2007) were used.
AEs were collected for study participants, but not donors. Intensity of AEs was classified as mild, moderate, severe, or disabling. AEs were categorized as unrelated or possibly, probably, or definitely related to GMT. Data safety monitoring board was established to monitor AEs and review halting criteria after every 3 participants completed GMT.
|
|
Gastrointestinal disorders
Abdominal pain/cramping
|
60.0%
6/10 • Number of events 11 • Adverse events (AEs) were recorded during and until 4 weeks after GMT using a symptom diary that participants completed. Common Terminology Criteria for Adverse Events and Toxicity Grading Guidance from Vaccine Clinical Trials (FDA, 2007) were used.
AEs were collected for study participants, but not donors. Intensity of AEs was classified as mild, moderate, severe, or disabling. AEs were categorized as unrelated or possibly, probably, or definitely related to GMT. Data safety monitoring board was established to monitor AEs and review halting criteria after every 3 participants completed GMT.
|
|
Gastrointestinal disorders
Diarrhea
|
60.0%
6/10 • Number of events 9 • Adverse events (AEs) were recorded during and until 4 weeks after GMT using a symptom diary that participants completed. Common Terminology Criteria for Adverse Events and Toxicity Grading Guidance from Vaccine Clinical Trials (FDA, 2007) were used.
AEs were collected for study participants, but not donors. Intensity of AEs was classified as mild, moderate, severe, or disabling. AEs were categorized as unrelated or possibly, probably, or definitely related to GMT. Data safety monitoring board was established to monitor AEs and review halting criteria after every 3 participants completed GMT.
|
|
Gastrointestinal disorders
Blood in stool
|
30.0%
3/10 • Number of events 5 • Adverse events (AEs) were recorded during and until 4 weeks after GMT using a symptom diary that participants completed. Common Terminology Criteria for Adverse Events and Toxicity Grading Guidance from Vaccine Clinical Trials (FDA, 2007) were used.
AEs were collected for study participants, but not donors. Intensity of AEs was classified as mild, moderate, severe, or disabling. AEs were categorized as unrelated or possibly, probably, or definitely related to GMT. Data safety monitoring board was established to monitor AEs and review halting criteria after every 3 participants completed GMT.
|
|
General disorders
Fatigue
|
30.0%
3/10 • Number of events 3 • Adverse events (AEs) were recorded during and until 4 weeks after GMT using a symptom diary that participants completed. Common Terminology Criteria for Adverse Events and Toxicity Grading Guidance from Vaccine Clinical Trials (FDA, 2007) were used.
AEs were collected for study participants, but not donors. Intensity of AEs was classified as mild, moderate, severe, or disabling. AEs were categorized as unrelated or possibly, probably, or definitely related to GMT. Data safety monitoring board was established to monitor AEs and review halting criteria after every 3 participants completed GMT.
|
|
General disorders
Fever
|
20.0%
2/10 • Number of events 2 • Adverse events (AEs) were recorded during and until 4 weeks after GMT using a symptom diary that participants completed. Common Terminology Criteria for Adverse Events and Toxicity Grading Guidance from Vaccine Clinical Trials (FDA, 2007) were used.
AEs were collected for study participants, but not donors. Intensity of AEs was classified as mild, moderate, severe, or disabling. AEs were categorized as unrelated or possibly, probably, or definitely related to GMT. Data safety monitoring board was established to monitor AEs and review halting criteria after every 3 participants completed GMT.
|
Additional Information
Dr. Sachin Kunde
Helen DeVos Children's Hospital of Spectrum Health Hospitals
Phone: 616-267-2100
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place