Trial Outcomes & Findings for MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders (NCT NCT04132427)
NCT ID: NCT04132427
Last Updated: 2024-08-26
Results Overview
The DSR is a daily record of their bowel movements including Bristol Stool Form scale. It is rated as the number of days (out of 14 days) with an abnormal report (abnormal stool, no stool, or the use of a gastrointestinal treatment). A higher percentage indicates worse symptoms.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
6 participants
Primary outcome timeframe
change in % abnormal days from baseline (for 2 weeks) vs. week 14 (2 weeks from week 13-14)
Results posted on
2024-08-26
Participant Flow
Participant milestones
| Measure |
Group A: Treatment
Vancomycin, magnesium citrate, microbiota
vancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota
|
Group B: Placebo
placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota
placebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota
|
|---|---|---|
|
Overall Study
STARTED
|
3
|
3
|
|
Overall Study
COMPLETED
|
3
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
Baseline characteristics by cohort
| Measure |
Group A: Treatment
n=3 Participants
Vancomycin, magnesium citrate, microbiota
vancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota
|
Group B: Placebo
n=3 Participants
placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota
placebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota
|
Total
n=6 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
13.0 years
STANDARD_DEVIATION 3.6 • n=5 Participants
|
12.7 years
STANDARD_DEVIATION 3.1 • n=7 Participants
|
12.8 years
STANDARD_DEVIATION 3.0 • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
3 participants
n=7 Participants
|
6 participants
n=5 Participants
|
|
Daily Stool Record
|
14 Number of abnormal days out of 14 days
STANDARD_DEVIATION 0 • n=5 Participants
|
14 Number of abnormal days out of 14 days
STANDARD_DEVIATION 0 • n=7 Participants
|
14 Number of abnormal days out of 14 days
STANDARD_DEVIATION 0 • n=5 Participants
|
PRIMARY outcome
Timeframe: change in % abnormal days from baseline (for 2 weeks) vs. week 14 (2 weeks from week 13-14)The DSR is a daily record of their bowel movements including Bristol Stool Form scale. It is rated as the number of days (out of 14 days) with an abnormal report (abnormal stool, no stool, or the use of a gastrointestinal treatment). A higher percentage indicates worse symptoms.
Outcome measures
| Measure |
Group A: Treatment
n=3 Participants
Vancomycin, magnesium citrate, microbiota
vancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota
|
Group B: Placebo
n=3 Participants
placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota
placebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota
|
|---|---|---|
|
Daily Stool Record (DSR(
|
-2.3 days
Standard Deviation 2.5
|
0 days
Standard Deviation 0
|
PRIMARY outcome
Timeframe: weeks 0-14number of adverse events and serious adverse events likely associated with treatment
Outcome measures
| Measure |
Group A: Treatment
n=3 Participants
Vancomycin, magnesium citrate, microbiota
vancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota
|
Group B: Placebo
n=3 Participants
placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota
placebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota
|
|---|---|---|
|
Safety Measures
|
8 number of AEs among all participants
|
10 number of AEs among all participants
|
SECONDARY outcome
Timeframe: change in score between baseline and week 14Clinical Global Impressions of GI Disorders, including severity, change in severity, and side effects. The severity is rated on a scale of 1-7, with 1 being normal and 7 being the worst possible.
Outcome measures
| Measure |
Group A: Treatment
n=3 Participants
Vancomycin, magnesium citrate, microbiota
vancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota
|
Group B: Placebo
n=3 Participants
placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota
placebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota
|
|---|---|---|
|
CGI for GI Disorders
|
-1 units on a scale
Standard Deviation 1
|
-.67 units on a scale
Standard Deviation 1.15
|
SECONDARY outcome
Timeframe: change in score between baseline and week 14Clinical Global Impressions of Pitt Hopkins Syndrome symptoms, including severity, change in severity, and side effects. The severity is rated on a scale of 1-7, with 1 being normal and 7 being the worst possible.
Outcome measures
| Measure |
Group A: Treatment
n=3 Participants
Vancomycin, magnesium citrate, microbiota
vancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota
|
Group B: Placebo
n=3 Participants
placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota
placebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota
|
|---|---|---|
|
CGI for PTHS Symptoms
|
0 units on a scale
Standard Deviation 0
|
-.33 units on a scale
Standard Deviation .58
|
SECONDARY outcome
Timeframe: change in score between baseline and week 14Parent Global Impressions of Pitt Hopkins Syndrome symptoms. This is a rating of 29 symptoms, and an average is computed. The scale ranges from -3 (much worse) to +3 (much better).
Outcome measures
| Measure |
Group A: Treatment
n=3 Participants
Vancomycin, magnesium citrate, microbiota
vancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota
|
Group B: Placebo
n=3 Participants
placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota
placebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota
|
|---|---|---|
|
PGI-PTHS
|
.68 -3 to +3
Standard Deviation .97
|
.29 -3 to +3
Standard Deviation .44
|
SECONDARY outcome
Timeframe: change in score between baseline and week 14Gastrointestinal Symptom Rating Scale. This is a 15-item questionnaire, with each item rated on a scale of 1 (no symptoms) to 7 (very severe discomfort). The average score for all 15 items is reported here.
Outcome measures
| Measure |
Group A: Treatment
n=3 Participants
Vancomycin, magnesium citrate, microbiota
vancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota
|
Group B: Placebo
n=3 Participants
placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota
placebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota
|
|---|---|---|
|
GSRS
|
-2.3 units on a scale
Standard Deviation .87
|
-1.07 units on a scale
Standard Deviation 1.91
|
SECONDARY outcome
Timeframe: change in score between baseline and week 14Revised Face Legs Activity Crying Consolability Pain Questionnaire for Children with Cognitive Impairment (FLACC). This is a rating scale of 5 symptoms of pain, with each item rated on a scale of 0 (no symptom) to 2 (maximum symptom), and the scores for each item are summed to create a total score (zero to 10).
Outcome measures
| Measure |
Group A: Treatment
n=3 Participants
Vancomycin, magnesium citrate, microbiota
vancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota
|
Group B: Placebo
n=3 Participants
placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota
placebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota
|
|---|---|---|
|
FLACC
|
-4.0 units on a scale
Standard Deviation 2.65
|
-2.67 units on a scale
Standard Deviation 2.08
|
Adverse Events
Group A: Treatment
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Group B: Placebo
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Group A: Treatment
n=3 participants at risk
Vancomycin, magnesium citrate, microbiota
vancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota
|
Group B: Placebo
n=3 participants at risk
placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota
placebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota
|
|---|---|---|
|
Psychiatric disorders
Hyperactivity
|
33.3%
1/3 • Number of events 1 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
66.7%
2/3 • Number of events 2 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
|
Psychiatric disorders
Irritability
|
33.3%
1/3 • Number of events 1 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
66.7%
2/3 • Number of events 2 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
|
Gastrointestinal disorders
Gassy
|
66.7%
2/3 • Number of events 2 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
66.7%
2/3 • Number of events 2 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
|
General disorders
Insomnia
|
33.3%
1/3 • Number of events 1 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
66.7%
2/3 • Number of events 2 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
|
Gastrointestinal disorders
Loss of appetite
|
33.3%
1/3 • Number of events 1 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
33.3%
1/3 • Number of events 1 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
|
Gastrointestinal disorders
Diarrhea
|
66.7%
2/3 • Number of events 2 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
66.7%
2/3 • Number of events 2 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
|
Skin and subcutaneous tissue disorders
Redness- Genitalia
|
33.3%
1/3 • Number of events 1 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
0.00%
0/3 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
|
Gastrointestinal disorders
Change in Stool Color
|
66.7%
2/3 • Number of events 2 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
33.3%
1/3 • Number of events 1 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
|
Gastrointestinal disorders
Abdominal Pain
|
33.3%
1/3 • Number of events 1 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
100.0%
3/3 • Number of events 3 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
|
Gastrointestinal disorders
Bloating
|
0.00%
0/3 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
100.0%
3/3 • Number of events 3 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
|
Gastrointestinal disorders
Constipation
|
33.3%
1/3 • Number of events 1 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
100.0%
3/3 • Number of events 3 • AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place