Trial Outcomes & Findings for Teduglutide for Enterocutaneous Fistula (ECF) (NCT NCT02889393)
NCT ID: NCT02889393
Last Updated: 2021-10-06
Results Overview
Percent fistula volume will be reported from the participant
COMPLETED
PHASE2
6 participants
Baseline, Up to 8 weeks
2021-10-06
Participant Flow
Participant milestones
| Measure |
Standard of Care Followed by Teduglutide
Participants in this group will receive standard of care treatment for the first 8 weeks followed by daily Teduglutide for the next 8 weeks.
Subjects with a favorable response will be offered an open label extension during which they will continue to receive teduglutide for up to another 12 months or permanent fistula closure.
Teduglutide: Daily 0.05 mg/kg Teduglutide administered subcutaneously.
Standard of Care: The standard of care treatment of ECF which includes meticulous wound care, optimizations of nutrition, use of acid-suppression medications and anti-motility agents as per treating physician discretion.
|
Teduglutide Followed by Standard of Care
Participants in this group will receive daily Teduglutide treatment for the first 8 weeks followed by standard of care for the next 8 weeks.
Subjects with a favorable response will be offered an open label extension during which they will continue to receive teduglutide for up to another 12 months or permanent fistula closure
Teduglutide: Daily 0.05 mg/kg Teduglutide administered subcutaneously.
Standard of Care: The standard of care treatment of ECF which includes meticulous wound care, optimizations of nutrition, use of acid-suppression medications and anti-motility agents as per treating physician discretion.
|
|---|---|---|
|
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
Teduglutide for Enterocutaneous Fistula (ECF)
Baseline characteristics by cohort
| Measure |
Standard of Care Followed by Teduglutide
n=3 Participants
Participants in this group will receive standard of care treatment for the first 8 weeks followed by daily Teduglutide for the next 8 weeks.
Subjects with a favorable response will be offered an open label extension during which they will continue to receive teduglutide for up to another 12 months or permanent fistula closure.
Teduglutide: Daily 0.05 mg/kg Teduglutide administered subcutaneously.
Standard of Care: The standard of care treatment of ECF which includes meticulous wound care, optimizations of nutrition, use of acid-suppression medications and anti-motility agents as per treating physician discretion.
|
Teduglutide Followed by Standard of Care
n=3 Participants
Participants in this group will receive daily Teduglutide treatment for the first 8 weeks followed by standard of care for the next 8 weeks.
Subjects with a favorable response will be offered an open label extension during which they will continue to receive teduglutide for up to another 12 months or permanent fistula closure
Teduglutide: Daily 0.05 mg/kg Teduglutide administered subcutaneously.
Standard of Care: The standard of care treatment of ECF which includes meticulous wound care, optimizations of nutrition, use of acid-suppression medications and anti-motility agents as per treating physician discretion.
|
Total
n=6 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 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
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 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
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 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
|
PRIMARY outcome
Timeframe: Baseline, Up to 8 weeksPopulation: This is a cross-over study. Each participant received both teduglutide intervention and the standard of care intervention for 8 weeks each.
Percent fistula volume will be reported from the participant
Outcome measures
| Measure |
Teduglutide Intervention
n=6 Participants
Participants that received Teduglutide for 8 weeks
|
Standard of Care Intervention
n=6 Participants
Participants that received Standard of Care for 8 weeks
|
|---|---|---|
|
Change in Average 3-day Fistula Percent Volume
|
76.33 Percent change in fistula volume
Standard Deviation 355.36
|
441.67 Percent change in fistula volume
Standard Deviation 824.87
|
SECONDARY outcome
Timeframe: Up to 16 weeksPopulation: Ceased fistula output was reported for all participants at the end of treatment after receiving both interventions.
As evaluated by treating physician of having at least 1 ceased fistula output
Outcome measures
| Measure |
Teduglutide Intervention
n=6 Participants
Participants that received Teduglutide for 8 weeks
|
Standard of Care Intervention
Participants that received Standard of Care for 8 weeks
|
|---|---|---|
|
Number of Participants With Ceased Fistula Output
|
3 Participants
|
—
|
SECONDARY outcome
Timeframe: Up to 16 weeksPopulation: This is a cross-over study. Each participant received both teduglutide intervention and the standard of care intervention for 8 weeks each.
Adult Sickle Cell Quality of Life Measurement (ASCQ-Me) Version 2.0 Social Functioning - Short Form questionnaire has a total score ranging from 5-25 with the higher score indicating a healthier status.
Outcome measures
| Measure |
Teduglutide Intervention
n=6 Participants
Participants that received Teduglutide for 8 weeks
|
Standard of Care Intervention
n=6 Participants
Participants that received Standard of Care for 8 weeks
|
|---|---|---|
|
Quality of Life as Measured by ASCQ-Me® v2.0 Social Functioning - Short Form
|
20.5 score on a scale
Standard Deviation 5.2
|
20 score on a scale
Standard Deviation 7.7
|
SECONDARY outcome
Timeframe: Up to 16 weeksPopulation: This is a cross-over study. Each participant received both teduglutide intervention and the standard of care intervention for 8 weeks each.
Adult Sickle Cell Quality of Life Measurement (ASCQ-Me) Version 2.0 Sleep Impact - Short Form questionnaire has a total score ranging from 5-25 with the higher score indicating a healthier status
Outcome measures
| Measure |
Teduglutide Intervention
n=6 Participants
Participants that received Teduglutide for 8 weeks
|
Standard of Care Intervention
n=6 Participants
Participants that received Standard of Care for 8 weeks
|
|---|---|---|
|
Quality of Life as Measured by the ASCQ-Me® v2.0 Sleep Impact - Short Form
|
19.5 score on a scale
Standard Deviation 1.3
|
19.5 score on a scale
Standard Deviation 4.1
|
SECONDARY outcome
Timeframe: Up to 16 weeksPopulation: This is a cross-over study. Each participant received both teduglutide intervention and the standard of care intervention for 8 weeks each. 1 participant in the teduglutide arm did not complete the global health questionnaire.
Patient Reported Outcome Measures Information Systems Global Health has a total score ranging from 10-50 with the higher score indicating a healthier status
Outcome measures
| Measure |
Teduglutide Intervention
n=5 Participants
Participants that received Teduglutide for 8 weeks
|
Standard of Care Intervention
n=6 Participants
Participants that received Standard of Care for 8 weeks
|
|---|---|---|
|
Quality of Life as Measured by the PROMIS Scale v1.2 - Global Health
|
30 score on a scale
Standard Deviation 6.3
|
35 score on a scale
Standard Deviation 10.2
|
SECONDARY outcome
Timeframe: Up to 16 weeksPopulation: This is a cross-over study. Each participant received both teduglutide intervention and the standard of care intervention for 8 weeks each.
Patient Reported Outcome Measures Information Systems (PROMIS-GI) Gastrointestinal Belly Pain 5a questionnaire has a total score ranging from 1-25 with the lower score indicating a healthier status
Outcome measures
| Measure |
Teduglutide Intervention
n=6 Participants
Participants that received Teduglutide for 8 weeks
|
Standard of Care Intervention
n=6 Participants
Participants that received Standard of Care for 8 weeks
|
|---|---|---|
|
Quality of Life as Measured by the PROMIS Scale v1.0 - Gastrointestinal Belly Pain 5a
|
6.0 score on a scale
Standard Deviation 8.2
|
7.5 score on a scale
Standard Deviation 6.9
|
SECONDARY outcome
Timeframe: Up to 16 weeksPopulation: This is a cross-over study. Each participant received both teduglutide intervention and the standard of care intervention for 8 weeks each.
Patient Reported Outcome Measures Information Systems (PROMIS-GI) Gastrointestinal Diarrhea 6a questionnaire has a total score ranging from 1-30 with the lower score indicating a healthier status
Outcome measures
| Measure |
Teduglutide Intervention
n=6 Participants
Participants that received Teduglutide for 8 weeks
|
Standard of Care Intervention
n=6 Participants
Participants that received Standard of Care for 8 weeks
|
|---|---|---|
|
Quality of Life as Measured by the PROMIS Scale v1.0 - Gastrointestinal Diarrhea 6a
|
8.0 score on a scale
Standard Deviation 4.4
|
6.0 score on a scale
Standard Deviation 4.0
|
SECONDARY outcome
Timeframe: Up to 16 weeksPopulation: This is a cross-over study. Each participant received both teduglutide intervention and the standard of care intervention for 8 weeks each.
Patient Reported Outcome Measures Information Systems (PROMIS-GI) Gastrointestinal Nausea and Vomiting 4a questionnaire has a total score ranging from 1-20 with the lower score indicating a healthier status
Outcome measures
| Measure |
Teduglutide Intervention
n=6 Participants
Participants that received Teduglutide for 8 weeks
|
Standard of Care Intervention
n=6 Participants
Participants that received Standard of Care for 8 weeks
|
|---|---|---|
|
Quality of Life as Measured by the PROMIS Scale v1.0 - Gastrointestinal Nausea and Vomiting 4a
|
3.0 score on a scale
Standard Deviation 2.8
|
3.5 score on a scale
Standard Deviation 3.3
|
SECONDARY outcome
Timeframe: Up to 16 weeksPopulation: This is a cross-over study. Each participant received both teduglutide intervention and the standard of care intervention for 8 weeks each.
Patient Reported Outcome Measures Information Systems (PROMIS) Ability to Participate in Social Roles and Activities - Short Form 8a questionnaire has a total score ranging from 8-40 with the lower score indicating a healthier status
Outcome measures
| Measure |
Teduglutide Intervention
n=6 Participants
Participants that received Teduglutide for 8 weeks
|
Standard of Care Intervention
n=6 Participants
Participants that received Standard of Care for 8 weeks
|
|---|---|---|
|
Quality of Life as Measured by the PROMIS Scale v2.0 - Ability to Participate in Social Roles and Activities - Short Form 8a
|
32.0 score on a scale
Standard Deviation 7.8
|
34.0 score on a scale
Standard Deviation 13.0
|
Adverse Events
On Teduglutide
On Standard of Care
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
On Teduglutide
n=6 participants at risk
The study is a crossover design. All participants received 8 weeks of daily Teduglutide.
Teduglutide: Daily 0.05 mg/kg Teduglutide administered subcutaneously.
|
On Standard of Care
n=6 participants at risk
The study is a crossover design. All participants received 8 weeks of daily Standard of Care Therapy.
Standard of Care: The standard of care treatment of ECF which includes meticulous wound care, optimizations of nutrition, use of acid-suppression medications and anti-motility agents as per treating physician discretion.
|
|---|---|---|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/6 • Up to 19 months
|
16.7%
1/6 • Number of events 1 • Up to 19 months
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/6 • Up to 19 months
|
16.7%
1/6 • Number of events 1 • Up to 19 months
|
|
Skin and subcutaneous tissue disorders
Stoma hypertrophy
|
16.7%
1/6 • Number of events 1 • Up to 19 months
|
0.00%
0/6 • Up to 19 months
|
|
Skin and subcutaneous tissue disorders
Pain at fistula site
|
0.00%
0/6 • Up to 19 months
|
16.7%
1/6 • Number of events 1 • Up to 19 months
|
|
Musculoskeletal and connective tissue disorders
Leg Cramps
|
0.00%
0/6 • Up to 19 months
|
16.7%
1/6 • Number of events 1 • Up to 19 months
|
|
Skin and subcutaneous tissue disorders
Lip Swelling
|
16.7%
1/6 • Number of events 1 • Up to 19 months
|
0.00%
0/6 • Up to 19 months
|
|
General disorders
Headache
|
16.7%
1/6 • Number of events 1 • Up to 19 months
|
0.00%
0/6 • Up to 19 months
|
|
Infections and infestations
Upper Respiratory Infection
|
0.00%
0/6 • Up to 19 months
|
16.7%
1/6 • Number of events 1 • Up to 19 months
|
|
Musculoskeletal and connective tissue disorders
Right Sided Chest Pain
|
0.00%
0/6 • Up to 19 months
|
16.7%
1/6 • Number of events 1 • Up to 19 months
|
|
Skin and subcutaneous tissue disorders
Small Bleeding from Fistula
|
16.7%
1/6 • Number of events 1 • Up to 19 months
|
0.00%
0/6 • Up to 19 months
|
|
Surgical and medical procedures
IV Infiltration
|
16.7%
1/6 • Number of events 1 • Up to 19 months
|
0.00%
0/6 • Up to 19 months
|
|
Renal and urinary disorders
Increased Creatinine
|
16.7%
1/6 • Number of events 1 • Up to 19 months
|
0.00%
0/6 • Up to 19 months
|
|
Skin and subcutaneous tissue disorders
Edema around ostomy site
|
16.7%
1/6 • Number of events 1 • Up to 19 months
|
0.00%
0/6 • Up to 19 months
|
|
Skin and subcutaneous tissue disorders
Cellulitis
|
0.00%
0/6 • Up to 19 months
|
16.7%
1/6 • Number of events 1 • Up to 19 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place