Trial Outcomes & Findings for Trichuris Suis Ova Treatment in Left-sided Ulcerative Colitis (NCT NCT01953354)
NCT ID: NCT01953354
Last Updated: 2017-03-17
Results Overview
Clinical response is defined as a reduction in the Mayo score of at least 3 points and at least a 30% reduction from Baseline, along with either a decrease from Baseline in the rectal bleeding subscore of more than 1 point or a rectal bleeding subscore of 0 or 1.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
16 participants
Primary outcome timeframe
Week 12
Results posted on
2017-03-17
Participant Flow
Of all the participating sites, nine reached the study intervention randomization phase for \>=one participant.The first site was activated in November 2013 and the last participant was randomized in March 2015.
Participant milestones
| Measure |
TSO 7500
Participants were randomized to receive six doses of 7500 viable, embryonated Trichuris suis ova (TSO) in liquid suspension orally over a 10-week period.
|
Placebo
Participants were randomized to receive six doses of placebo in liquid suspension orally over a 10-week period.
|
|---|---|---|
|
Overall Study
STARTED
|
9
|
7
|
|
Overall Study
COMPLETED
|
8
|
5
|
|
Overall Study
NOT COMPLETED
|
1
|
2
|
Reasons for withdrawal
| Measure |
TSO 7500
Participants were randomized to receive six doses of 7500 viable, embryonated Trichuris suis ova (TSO) in liquid suspension orally over a 10-week period.
|
Placebo
Participants were randomized to receive six doses of placebo in liquid suspension orally over a 10-week period.
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
|
Overall Study
Decision of Sponsor
|
0
|
1
|
|
Overall Study
Disease Exacerbation
|
1
|
0
|
Baseline Characteristics
Trichuris Suis Ova Treatment in Left-sided Ulcerative Colitis
Baseline characteristics by cohort
| Measure |
TSO 7500
n=9 Participants
Participants were randomized to receive six doses of 7500 viable, embryonated Trichuris suis ova (TSO) in liquid suspension orally over a 10-week period.
|
Placebo
n=7 Participants
Participants were randomized to receive six doses of placebo in liquid suspension orally over a 10-week period.
|
Total
n=16 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
40.0 years
STANDARD_DEVIATION 9.0 • n=5 Participants
|
44.7 years
STANDARD_DEVIATION 11.4 • n=7 Participants
|
42.1 years
STANDARD_DEVIATION 10.0 • n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
14 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
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 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
|
7 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
14 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
|
9 participants
n=5 Participants
|
7 participants
n=7 Participants
|
16 participants
n=5 Participants
|
|
Percentage of Participants at Baseline Who Were Taking a Corticosteroid
|
33.3 percentage of participants
n=5 Participants
|
14.3 percentage of participants
n=7 Participants
|
25.0 percentage of participants
n=5 Participants
|
|
Percentage of Participants at Baseline Who Were Taking Thiopurine
|
11.1 percentage of participants
n=5 Participants
|
0.0 percentage of participants
n=7 Participants
|
6.3 percentage of participants
n=5 Participants
|
|
Mayo Score for Assessment of Ulcerative Colitis Activity
|
9.0 Total Mayo Score
STANDARD_DEVIATION 1.2 • n=5 Participants
|
7.4 Total Mayo Score
STANDARD_DEVIATION 1.3 • n=7 Participants
|
8.3 Total Mayo Score
STANDARD_DEVIATION 1.5 • n=5 Participants
|
PRIMARY outcome
Timeframe: Week 12Population: The Modified Intent-to-Treat (mITT) population included all randomized subjects who received at least one dose of either TSO or placebo. Only mITT subjects with clinical response results at Week 12 are included in this analysis.
Clinical response is defined as a reduction in the Mayo score of at least 3 points and at least a 30% reduction from Baseline, along with either a decrease from Baseline in the rectal bleeding subscore of more than 1 point or a rectal bleeding subscore of 0 or 1.
Outcome measures
| Measure |
TSO 7500
n=7 Participants
Participants were randomized to receive six doses of 7500 viable, embryonated Trichuris suis ova (TSO) in liquid suspension orally over a 10-week period.
|
Placebo
n=6 Participants
Participants were randomized to receive six doses of placebo in liquid suspension orally over a 10-week period.
|
|---|---|---|
|
Percentage of Participants Who Achieved a Clinical Response at Week 12
|
57.1 percentage of participants
|
50.0 percentage of participants
|
SECONDARY outcome
Timeframe: Week 12Population: The Modified Intent-to-Treat (mITT) population included all randomized subjects who received at least one dose of either TSO or placebo. Only mITT subjects with remission results at Week 12 are included in this analysis.
Remission is defined as a Mayo score of less than or equal to 1 with absence of rectal bleeding and endoscopy score of 0 or 1.
Outcome measures
| Measure |
TSO 7500
n=7 Participants
Participants were randomized to receive six doses of 7500 viable, embryonated Trichuris suis ova (TSO) in liquid suspension orally over a 10-week period.
|
Placebo
n=6 Participants
Participants were randomized to receive six doses of placebo in liquid suspension orally over a 10-week period.
|
|---|---|---|
|
Percent of Participants Who Achieved Remission at Week 12
|
14.3 percentage of participants
|
16.7 percentage of participants
|
SECONDARY outcome
Timeframe: Week 12Population: The Modified Intent-to-Treat (mITT) population included all randomized subjects who received at least one dose of either TSO or placebo. Only mITT subjects with a Mayo endoscopy score at Week 12 are included in this analysis.
Healed colonic mucosa is defined as a Mayo endoscopy score of 0 or 1.
Outcome measures
| Measure |
TSO 7500
n=6 Participants
Participants were randomized to receive six doses of 7500 viable, embryonated Trichuris suis ova (TSO) in liquid suspension orally over a 10-week period.
|
Placebo
n=6 Participants
Participants were randomized to receive six doses of placebo in liquid suspension orally over a 10-week period.
|
|---|---|---|
|
Percent of Participants With Healed Colonic Mucosa at Week 12
|
66.7 percentage of participants
|
16.7 percentage of participants
|
SECONDARY outcome
Timeframe: From Day 0 through time of first clinical response or end of follow-up, whichever comes first, up to 12 WeeksPopulation: The Modified Intent-to-Treat (mITT) population included all randomized subjects who received at least one dose of either TSO or placebo. Only mITT subjects with baseline and at least one post-baseline modified clinical response result are included in this analysis.
Modified clinical response is defined as a reduction in the modified Mayo score (i.e., minus the endoscopy component) of at least 2 points from baseline.
Outcome measures
| Measure |
TSO 7500
n=9 Participants
Participants were randomized to receive six doses of 7500 viable, embryonated Trichuris suis ova (TSO) in liquid suspension orally over a 10-week period.
|
Placebo
n=7 Participants
Participants were randomized to receive six doses of placebo in liquid suspension orally over a 10-week period.
|
|---|---|---|
|
Percent of Participants With a Modified Clinical Response
|
88.9 percentage of participants
|
71.4 percentage of participants
|
SECONDARY outcome
Timeframe: From Baseline through the day that modified clinical response is reached. Week 16 is the last visit that the modified Mayo score is assessed.Population: The Modified Intent-to-Treat (mITT) population included all randomized subjects who received at least one dose of either TSO or placebo. Only mITT subjects who achieved a modified clinical response are included in this analysis.
Number of days to reach a modified clinical response. Modified clinical response is defined as a reduction in the modified Mayo score (i.e., minus the endoscopy component) of at least 2 points from baseline.
Outcome measures
| Measure |
TSO 7500
n=8 Participants
Participants were randomized to receive six doses of 7500 viable, embryonated Trichuris suis ova (TSO) in liquid suspension orally over a 10-week period.
|
Placebo
n=5 Participants
Participants were randomized to receive six doses of placebo in liquid suspension orally over a 10-week period.
|
|---|---|---|
|
Time to Modified Clinical Response
|
34.5 Days
Interval 14.0 to 125.0
|
28.0 Days
Interval 15.0 to 126.0
|
SECONDARY outcome
Timeframe: From Day 0 through end of follow-up, up to 36 weeksPopulation: The Safety population included all subjects for whom study treatment was initiated.
Stool evaluations for ova and parasites confirmed the absence of T. suis. If evidence suggested a presence of T. suis, a colonoscopy would be performed to confirm invasion with a visible worm.
Outcome measures
| Measure |
TSO 7500
n=9 Participants
Participants were randomized to receive six doses of 7500 viable, embryonated Trichuris suis ova (TSO) in liquid suspension orally over a 10-week period.
|
Placebo
n=7 Participants
Participants were randomized to receive six doses of placebo in liquid suspension orally over a 10-week period.
|
|---|---|---|
|
Percent of Participants With Colonoscopic Evidence of Visible Worm
|
0 percentage of participants
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: From Day 0 through end of follow-up, up to 36 weeksPopulation: The Safety population included all subjects for whom study treatment was initiated.
An increase in diarrhea is defined as an increase in the Mayo Score's Stool Frequency score by at least 1 point from baseline at any time during follow-up.
Outcome measures
| Measure |
TSO 7500
n=9 Participants
Participants were randomized to receive six doses of 7500 viable, embryonated Trichuris suis ova (TSO) in liquid suspension orally over a 10-week period.
|
Placebo
n=7 Participants
Participants were randomized to receive six doses of placebo in liquid suspension orally over a 10-week period.
|
|---|---|---|
|
Percent of Participants With Increase in Diarrhea
|
11.1 percentage of participants
|
28.6 percentage of participants
|
SECONDARY outcome
Timeframe: From Day 0 through Week 16Population: The Safety population included all subjects for whom study treatment was initiated.
New or increase in UC medications is defined as a need for dose-escalation of concurrent medications or need for rescue medications to treat UC through Week 16.
Outcome measures
| Measure |
TSO 7500
n=9 Participants
Participants were randomized to receive six doses of 7500 viable, embryonated Trichuris suis ova (TSO) in liquid suspension orally over a 10-week period.
|
Placebo
n=7 Participants
Participants were randomized to receive six doses of placebo in liquid suspension orally over a 10-week period.
|
|---|---|---|
|
Percent of Participants With Increase in Concurrent Ulcerative Colitis (UC) Medications or New Rescue Medications Added
|
44.4 percentage of participants
|
28.6 percentage of participants
|
Adverse Events
TSO 7500
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
TSO 7500
n=8 participants at risk
Participants were randomized to receive six doses of 7500 viable, embryonated Trichuris suis ova (TSO) in liquid suspension orally over a 10-week period.
|
Placebo
n=7 participants at risk
Participants were randomized to receive six doses of placebo in liquid suspension orally over a 10-week period.
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
12.5%
1/8 • Number of events 1 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
0.00%
0/7 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
Blood and lymphatic system disorders
Lymphopenia
|
0.00%
0/8 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
28.6%
2/7 • Number of events 2 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
Gastrointestinal disorders
Abdominal pain
|
12.5%
1/8 • Number of events 1 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
28.6%
2/7 • Number of events 4 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
Gastrointestinal disorders
Diarrhoea
|
25.0%
2/8 • Number of events 2 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
14.3%
1/7 • Number of events 1 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/8 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
14.3%
1/7 • Number of events 1 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
Gastrointestinal disorders
Stomatitis
|
12.5%
1/8 • Number of events 1 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
0.00%
0/7 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
General disorders
Condition aggravated
|
12.5%
1/8 • Number of events 1 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
0.00%
0/7 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
General disorders
Fatigue
|
12.5%
1/8 • Number of events 1 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
0.00%
0/7 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
General disorders
Influenza like illness
|
12.5%
1/8 • Number of events 1 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
0.00%
0/7 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
General disorders
Pyrexia
|
12.5%
1/8 • Number of events 1 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
0.00%
0/7 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
Infections and infestations
Clostridium difficile infection
|
12.5%
1/8 • Number of events 1 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
0.00%
0/7 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
Infections and infestations
Pharyngitis streptococcal
|
12.5%
1/8 • Number of events 1 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
0.00%
0/7 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
Nervous system disorders
Headache
|
25.0%
2/8 • Number of events 3 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
0.00%
0/7 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
Renal and urinary disorders
Urinary tract pain
|
0.00%
0/8 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
14.3%
1/7 • Number of events 1 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/8 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
14.3%
1/7 • Number of events 1 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
12.5%
1/8 • Number of events 1 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
0.00%
0/7 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
Respiratory, thoracic and mediastinal disorders
Rales
|
12.5%
1/8 • Number of events 1 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
0.00%
0/7 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
|
Skin and subcutaneous tissue disorders
Macule
|
12.5%
1/8 • Number of events 1 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
0.00%
0/7 • From the time of signed informed consent to the required follow-up time (Week 36) or until 30 days after the participant prematurely withdrew from the study.
CTCAE V4.0
|
Additional Information
Director, Clinical Research Operations Program
DAIT/NIAID
Phone: 301-594-7669
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place