Trial Outcomes & Findings for A Study of Teduglutide in Japanese Children With Short Bowel Syndrome Aged 4 Months or Older (NCT NCT05027308)
NCT ID: NCT05027308
Last Updated: 2024-05-16
Results Overview
An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AESI, whether serious or non-serious, is one of scientific and medical concern specific to the compound or program, for which ongoing monitoring and rapid communication by the investigator to the sponsor may be appropriate.
COMPLETED
PHASE3
3 participants
From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
2024-05-16
Participant Flow
Three participants took part in the study at six investigative sites in Japan from 4 January 2022 to 27 September 2023.
Pediatric participants with a diagnosis of short bowel syndrome (SBS) dependent on parenteral support (PS) were enrolled in the study based on the eligibility criteria to receive teduglutide.
Participant milestones
| Measure |
Teduglutide
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
|
|---|---|
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Overall Study
STARTED
|
3
|
|
Overall Study
COMPLETED
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study of Teduglutide in Japanese Children With Short Bowel Syndrome Aged 4 Months or Older
Baseline characteristics by cohort
| Measure |
Teduglutide
n=3 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
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|---|---|
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Age, Categorical
<=18 years
|
3 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
Japan
|
3 Participants
n=99 Participants
|
|
Height for Age Z-Score at Baseline
|
-3.060 Z score
STANDARD_DEVIATION 1.1601 • n=99 Participants
|
|
Weight for Age Z-Score at Baseline
|
-3.017 Z score
STANDARD_DEVIATION 1.8048 • n=99 Participants
|
PRIMARY outcome
Timeframe: From first dose of study drug until follow-up visit (4 weeks after end of treatment [EOT]/end of termination [ET] {up to 47.3-51.3 weeks})Population: Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. TEAEs were defined as any AEs whose onset occurred, severity worsened, or intensity increased after receiving the investigational product.
Outcome measures
| Measure |
Teduglutide
n=3 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
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|---|---|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
|
3 Participants
|
PRIMARY outcome
Timeframe: From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])Population: Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An SAE is defined as any untoward medical occurrence that at any dose: results in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, leads to a congenital anomaly /birth defect, is the other important medical event.
Outcome measures
| Measure |
Teduglutide
n=3 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
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|---|---|
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Number of Participants With Serious Adverse Events (SAEs)
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3 Participants
|
PRIMARY outcome
Timeframe: From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])Population: Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AESI, whether serious or non-serious, is one of scientific and medical concern specific to the compound or program, for which ongoing monitoring and rapid communication by the investigator to the sponsor may be appropriate.
Outcome measures
| Measure |
Teduglutide
n=3 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
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|---|---|
|
Number of Participants With Adverse Events of Special Interest (AESIs)
|
0 Participants
|
PRIMARY outcome
Timeframe: From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])Population: Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
Vital signs include systolic and diastolic blood pressure, heart rate and body temperature.
Outcome measures
| Measure |
Teduglutide
n=3 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
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|---|---|
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Number of Participants With Clinically Significant Abnormalities in Vital Signs Reported as an Adverse Event
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0 Participants
|
PRIMARY outcome
Timeframe: Baseline, EOT (up to 47.3-51.3 weeks)Population: Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
A Z-score is the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). According to WHO Child Growth Standards used for assessment of this outcome measure, a weight for age Z-score below -2 indicates underweight.
Outcome measures
| Measure |
Teduglutide
n=3 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
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|---|---|
|
Change From Baseline in Body Weight Z-Score at EOT
|
1.637 Z score
Standard Deviation 2.8839
|
PRIMARY outcome
Timeframe: Baseline, EOT (up to 47.3-51.3 weeks)Population: Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
A Z-score is the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). According to WHO Child Growth Standards used for assessment of this outcome measure, a height for age Z-score below -2 indicates stunted.
Outcome measures
| Measure |
Teduglutide
n=3 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
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|---|---|
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Change From Baseline in Height Z-Score at EOT
|
0.590 Z score
Standard Deviation 1.9949
|
PRIMARY outcome
Timeframe: Baseline, EOT (up to 47.3-51.3 weeks)Population: Safety Analysis Set included all participants who received at least 1 dose of study teduglutide. Overall number analyzed is the number of participants with data available for analyses.
A Z-score is the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). According to the Food and Nutrition Technical Assistance (FANTA) Guide to Anthropometry used for assessment of this outcome measure, a head circumference for age Z-score below -2 indicates small head circumference.
Outcome measures
| Measure |
Teduglutide
n=2 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
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|---|---|
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Change From Baseline in Head Circumference Z-Score at EOT
|
1.130 Z score
Standard Deviation 0.4101
|
PRIMARY outcome
Timeframe: Baseline, EOT (up to 47.3-51.3 weeks)Population: Safety Analysis Set included all participants who received at least 1 dose of study teduglutide. Overall number analyzed is the number of participants with data available for analyses.
A Z-score is the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). According to WHO Child Growth Standards used for assessment of this outcome measure, a weight for length Z-score below -2 indicates wasted (recent and severe weight loss).
Outcome measures
| Measure |
Teduglutide
n=1 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
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|---|---|
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Change From Baseline in Weight-for-Length Z-Score at EOT
|
4.240 Z score
Standard Deviation NA
The standard deviation was not estimable for a single participant.
|
PRIMARY outcome
Timeframe: From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])Population: Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
Laboratory safety parameters included biochemistry, hematology, and urinalysis.
Outcome measures
| Measure |
Teduglutide
n=3 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
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|---|---|
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Number of Participants With Any Laboratory Safety Finding Reported as an Adverse Event
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1 Participants
|
PRIMARY outcome
Timeframe: From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])Population: Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
Urine and stool output was recorded and calculated in the output diary over a 48-hour period of parenteral support (PS) and enteral nutrition (EN) stability before every site visit and within 1 week of implementing a change in the PS prescription.
Outcome measures
| Measure |
Teduglutide
n=3 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
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|---|---|
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Number of Participants With a Change in Urine Output Reported as an Adverse Event
|
1 Participants
|
PRIMARY outcome
Timeframe: From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])Population: Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
Urine and stool output was recorded and calculated in the output diary over a 48-hour period of PS and EN stability before every site visit and within 1 week of implementing a change in the PS prescription.
Outcome measures
| Measure |
Teduglutide
n=3 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
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|---|---|
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Number of Participants With a Change in Stool Output Reported as an Adverse Event
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline, Cycle 1 = Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 2: Week 0, 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 3 = Week 0, 1, 2, and EOT and overall EOT (for up to 47.3-51.3 weeks) [cycle length=28 weeks]Population: Full Analysis Set included all enrolled participants, who were not screen failures, regardless of whether participants took any dose of teduglutide in the study. Number analyzed is the number of participants with data available for analysis at the specified time point.
PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume and calories during the treatment period. An end of treatment (EOT) was defined as the last determination of endpoint of the last cycle.
Outcome measures
| Measure |
Teduglutide
n=3 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
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|---|---|
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Change From Baseline in PS Volume
Cycle 1, Week 1
|
3.13 milliliters per kilograms (mL/kg)/day
Standard Deviation 10.041
|
|
Change From Baseline in PS Volume
Cycle 1, Week 2
|
7.82 milliliters per kilograms (mL/kg)/day
Standard Deviation 19.334
|
|
Change From Baseline in PS Volume
Cycle 1, Week 4
|
-1.31 milliliters per kilograms (mL/kg)/day
Standard Deviation 6.491
|
|
Change From Baseline in PS Volume
Cycle 1, Week 8
|
10.27 milliliters per kilograms (mL/kg)/day
Standard Deviation 13.788
|
|
Change From Baseline in PS Volume
Cycle 1, Week 12
|
5.83 milliliters per kilograms (mL/kg)/day
Standard Deviation 15.979
|
|
Change From Baseline in PS Volume
Cycle 1, Week 16
|
1.89 milliliters per kilograms (mL/kg)/day
Standard Deviation 16.386
|
|
Change From Baseline in PS Volume
Cycle 1, Week 20
|
1.82 milliliters per kilograms (mL/kg)/day
Standard Deviation 6.673
|
|
Change From Baseline in PS Volume
Cycle 1, Week 24
|
0.66 milliliters per kilograms (mL/kg)/day
Standard Deviation 9.511
|
|
Change From Baseline in PS Volume
Cycle 1, EOT
|
0.66 milliliters per kilograms (mL/kg)/day
Standard Deviation 9.511
|
|
Change From Baseline in PS Volume
Cycle 2, Week 0
|
-0.07 milliliters per kilograms (mL/kg)/day
Standard Deviation 12.808
|
|
Change From Baseline in PS Volume
Cycle 2 Week 1
|
0.11 milliliters per kilograms (mL/kg)/day
Standard Deviation 15.080
|
|
Change From Baseline in PS Volume
Cycle 2, Week 2
|
-2.32 milliliters per kilograms (mL/kg)/day
Standard Deviation 13.465
|
|
Change From Baseline in PS Volume
Cycle 2, Week 4
|
-0.71 milliliters per kilograms (mL/kg)/day
Standard Deviation 12.202
|
|
Change From Baseline in PS Volume
Cycle 2, Week 8
|
-4.81 milliliters per kilograms (mL/kg)/day
Standard Deviation 13.214
|
|
Change From Baseline in PS Volume
Cycle 2, Week 12
|
-8.42 milliliters per kilograms (mL/kg)/day
Standard Deviation 11.909
|
|
Change From Baseline in PS Volume
Cycle 2, Week 16
|
-10.14 milliliters per kilograms (mL/kg)/day
Standard Deviation 10.452
|
|
Change From Baseline in PS Volume
Cycle 2, Week 20
|
-8.20 milliliters per kilograms (mL/kg)/day
Standard Deviation 0.509
|
|
Change From Baseline in PS Volume
Cycle 2, Week 24
|
-9.28 milliliters per kilograms (mL/kg)/day
Standard Deviation 2.092
|
|
Change From Baseline in PS Volume
Cycle 2, EOT
|
-9.11 milliliters per kilograms (mL/kg)/day
Standard Deviation 1.508
|
|
Change From Baseline in PS Volume
Cycle 3, Week 0
|
-12.86 milliliters per kilograms (mL/kg)/day
Standard Deviation NA
The standard deviation was not estimable for a single participant.
|
|
Change From Baseline in PS Volume
Cycle 3, Week 1
|
-13.67 milliliters per kilograms (mL/kg)/day
Standard Deviation NA
The standard deviation was not estimable for a single participant.
|
|
Change From Baseline in PS Volume
Cycle 3, Week 2
|
-5.04 milliliters per kilograms (mL/kg)/day
Standard Deviation NA
The standard deviation was not estimable for a single participant.
|
|
Change From Baseline in PS Volume
Cycle 3, EOT
|
-5.04 milliliters per kilograms (mL/kg)/day
Standard Deviation NA
The standard deviation was not estimable for a single participant.
|
|
Change From Baseline in PS Volume
Overall EOT
|
-10.99 milliliters per kilograms (mL/kg)/day
Standard Deviation 27.093
|
SECONDARY outcome
Timeframe: Baseline, Cycle 1 = Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 2 = Week 0, 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 3 = Week 0, 1, 2, and EOT and overall EOT (for up to 47.3-51.3 weeks) [cycle length=28 weeks]Population: Full Analysis Set included all enrolled participants, who were not screen failures, regardless of whether participants took any dose of teduglutide in the study. Number analyzed is the number of participants with data available for analysis at the specified time point.
Percent change from baseline in PS volume was calculated as follows; (PS volume at each point \[Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT\] - PS volume at baseline)/ PS volume at baseline \*100 (percent). PS (parenteral nutrition or intravenous fluids) was to be considered for managing nutritional support in terms of volume and calories during the treatment period. An EOT was defined as the last determination of endpoint of the last cycle.
Outcome measures
| Measure |
Teduglutide
n=3 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
|
|---|---|
|
Percent Change From Baseline in PS Volume
Cycle 1, Week 1
|
4.82 percent change
Standard Deviation 13.391
|
|
Percent Change From Baseline in PS Volume
Cycle 1, Week 2
|
11.27 percent change
Standard Deviation 26.175
|
|
Percent Change From Baseline in PS Volume
Cycle 1, Week 4
|
-1.51 percent change
Standard Deviation 8.881
|
|
Percent Change From Baseline in PS Volume
Cycle 1, Week 8
|
13.48 percent change
Standard Deviation 19.471
|
|
Percent Change From Baseline in PS Volume
Cycle 1, Week 12
|
6.97 percent change
Standard Deviation 22.006
|
|
Percent Change From Baseline in PS Volume
Cycle 1, Week 16
|
1.61 percent change
Standard Deviation 22.136
|
|
Percent Change From Baseline in PS Volume
Cycle 1, Week 20
|
2.94 percent change
Standard Deviation 8.794
|
|
Percent Change From Baseline in PS Volume
Cycle 1, Week 24
|
1.24 percent change
Standard Deviation 12.938
|
|
Percent Change From Baseline in PS Volume
Cycle 1, EOT
|
1.24 percent change
Standard Deviation 12.938
|
|
Percent Change From Baseline in PS Volume
Cycle 2, Week 0
|
0.07 percent change
Standard Deviation 17.520
|
|
Percent Change From Baseline in PS Volume
Cycle 2, Week 1
|
0.26 percent change
Standard Deviation 20.634
|
|
Percent Change From Baseline in PS Volume
Cycle 2, Week 2
|
-2.81 percent change
Standard Deviation 18.432
|
|
Percent Change From Baseline in PS Volume
Cycle 2, Week 4
|
-0.62 percent change
Standard Deviation 16.671
|
|
Percent Change From Baseline in PS Volume
Cycle 2, Week 8
|
-6.00 percent change
Standard Deviation 18.175
|
|
Percent Change From Baseline in PS Volume
Cycle 2, Week 12
|
-10.21 percent change
Standard Deviation 16.645
|
|
Percent Change From Baseline in PS Volume
Cycle 2, Week 16
|
-13.03 percent change
Standard Deviation 14.937
|
|
Percent Change From Baseline in PS Volume
Cycle 2, Week 20
|
-9.73 percent change
Standard Deviation 3.073
|
|
Percent Change From Baseline in PS Volume
Cycle 2, Week 24
|
-10.50 percent change
Standard Deviation 6.059
|
|
Percent Change From Baseline in PS Volume
Cycle 2, EOT
|
-10.98 percent change
Standard Deviation 4.363
|
|
Percent Change From Baseline in PS Volume
Cycle 3, Week 0
|
-17.69 percent change
Standard Deviation NA
The standard deviation was not estimable for a single participant.
|
|
Percent Change From Baseline in PS Volume
Cycle 3, Week 1
|
-18.79 percent change
Standard Deviation NA
The standard deviation was not estimable for a single participant.
|
|
Percent Change From Baseline in PS Volume
Cycle 3, Week 2
|
-6.93 percent change
Standard Deviation NA
The standard deviation was not estimable for a single participant.
|
|
Percent Change From Baseline in PS Volume
Cycle 3, EOT
|
-6.93 percent change
Standard Deviation NA
The standard deviation was not estimable for a single participant.
|
|
Percent Change From Baseline in PS Volume
Overall EOT
|
-13.13 percent change
Standard Deviation 37.259
|
SECONDARY outcome
Timeframe: Baseline, Cycle 1 = Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 2 = Week 0, 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 3 = Week 0, 1, 2, and EOT and overall EOT (for up to 47.3-51.3 weeks) [cycle length=28 weeks]Population: Full Analysis Set included all enrolled participants, who were not screen failures, regardless of whether participants took any dose of teduglutide in the study. Number analyzed is the number of participants with data available for analysis at the specified time point.
PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume and calories during the treatment period. An EOT was defined as the last determination of endpoint of the last cycle.
Outcome measures
| Measure |
Teduglutide
n=3 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
|
|---|---|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 1, Week 1
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 1, Week 2
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 1, Week 4
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 1, Week 8
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 1, Week 12
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 1, Week 16
|
1 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 1, Week 20
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 1, Week 24
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 1, EOT
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 2, Week 0
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 2, Week 1
|
1 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 2, Week 2
|
1 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 2, Week 4
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 2, Week 8
|
1 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 2, Week 12
|
1 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 2, Week 16
|
1 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 2, Week 20
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 2, Week 24
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 2, EOT
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 3, Week 0
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 3, Week 1
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 3, Week 2
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Cycle 3, EOT
|
0 Participants
|
|
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Overall EOT
|
1 Participants
|
SECONDARY outcome
Timeframe: Cycle 1 = Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 2 = Week 0, 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 3 = Week 0, 1, 2, and EOT and overall EOT (for up to 47.3-51.3 weeks) [cycle length=28 weeks]Population: Full Analysis Set included all enrolled participants, who were not screen failures, regardless of whether participants took any dose of teduglutide in the study. Number analyzed is the number of participants with data available for analysis at the specified time point.
Achieving enteral autonomy is defined as complete weaning off PS. PS (parenteral nutrition or intravenous fluids) was to be considered for managing nutritional support in terms of volume and calories during the treatment period.
Outcome measures
| Measure |
Teduglutide
n=3 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
|
|---|---|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 1, Week 1
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 1, Week 2
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 1, Week 4
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 1, Week 8
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 1, Week 12
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 1, Week 16
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 1, Week 20
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 1, Week 24
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 1, EOT
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 2, Week 0
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 2, Week 1
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 2, Week 2
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 2, Week 4
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 2, Week 8
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 2, Week 12
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 2, Week 16
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 2, Week 20
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 2, Week 24
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 2, EOT
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 3, Week 0
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 3, Week 1
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 3, Week 2
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Cycle 3, EOT
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy
Overall EOT
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline, Cycle 1 = Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 2 = Week 0, 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 3 = Week 0, 1, 2, and EOT, and overall EOT (for 47.3-51.3 weeks) [cycle length=28 weeks]Population: Full Analysis Set included all enrolled participants, who were not screen failures, regardless of whether participants took any dose of teduglutide in the study. Number analyzed is the number of participants with data available for analysis at the specified time point.
PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume and calories during the treatment period.
Outcome measures
| Measure |
Teduglutide
n=3 Participants
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
|
|---|---|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 1, Week 1
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 1, Week 2
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 1, Week 4
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 1, Week 8
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 1, Week 12
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 1, Week 16
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 1, Week 20
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 1, Week 24
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 1, EOT
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 2, Week 0
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 2, Week 1
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 2, Week 2
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 2, Week 4
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 2, Week 8
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 2, Week 12
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 2, Week 16
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 2, Week 20
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 2, Week 24
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 2, EOT
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 3, Week 0
|
0.0 days per week
Standard Deviation 0.00
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 3, Week 1
|
0.0 days per week
Standard Deviation NA
The standard deviation was not estimable for a single participant.
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 3, Week 2
|
0.0 days per week
Standard Deviation NA
The standard deviation was not estimable for a single participant.
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Cycle 3, EOT
|
0.0 days per week
Standard Deviation NA
The standard deviation was not estimable for a single participant.
|
|
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Overall EOT
|
0.0 days per week
Standard Deviation 0.00
|
Adverse Events
Teduglutide
Serious adverse events
| Measure |
Teduglutide
n=3 participants at risk
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
|
|---|---|
|
Infections and infestations
Bacteraemia
|
33.3%
1/3 • From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
|
|
General disorders
Catheter site pruritus
|
33.3%
1/3 • From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
|
|
Product Issues
Device breakage
|
33.3%
1/3 • From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
|
|
Infections and infestations
Device related infection
|
66.7%
2/3 • From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
|
|
General disorders
Vascular device occlusion
|
33.3%
1/3 • From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
|
Other adverse events
| Measure |
Teduglutide
n=3 participants at risk
Participants received teduglutide 0.05 milligram per kilogram \[mg/kg\] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous \[SC\] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles.
|
|---|---|
|
Investigations
Blood iron increased
|
33.3%
1/3 • From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
|
|
Infections and infestations
COVID-19
|
66.7%
2/3 • From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
|
|
General disorders
Catheter site rash
|
33.3%
1/3 • From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
|
|
Gastrointestinal disorders
Enterocolitis
|
66.7%
2/3 • From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
|
|
Metabolism and nutrition disorders
Lactic acidosis
|
33.3%
1/3 • From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
|
|
Hepatobiliary disorders
Liver disorder
|
33.3%
1/3 • From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
|
|
Renal and urinary disorders
Oliguria
|
33.3%
1/3 • From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
|
|
Infections and infestations
Parainfluenzae virus infection
|
33.3%
1/3 • From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
|
|
Infections and infestations
Respiratory syncytial virus infection
|
66.7%
2/3 • From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
|
|
Infections and infestations
Skin candida
|
33.3%
1/3 • From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract inflammation
|
33.3%
1/3 • From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The first study related publication will be a multi-center publication submitted within 24 months after conclusion or termination of a study at all sites. After such multi site publication, all proposed site publications and presentations will be submitted to sponsor for review 60 days in advance of publication. Site will remove Sponsor confidential information unrelated to study results. Sponsor can delay a proposed publication for another 60 days to preserve intellectual property.
- Publication restrictions are in place
Restriction type: OTHER