Trial · Interventional

CompletedPhase 3Results postedRegistry data

A Study of Teduglutide in Japanese Children With Short Bowel Syndrome Aged 4 Months or Older

NCTNCT05027308Other idTAK-633-3008Other idU1111-1267-3327Other idjRCT2021210035Other id2022-003572-16

Open at ClinicalTrials.govAsk about this trial

Overview

Status: Completed
Phase: Phase 3
Enrollment: 3 participants (actual)
Sponsor: Takeda · Industry
Design: Allocation not applicable · Open label (no masking)
Countries: 1 country
Japan
Start: 4 Jan 2022
Primary completion: 27 Sept 2023
Completion: 27 Sept 2023
First posted: 30 Aug 2021
Last update posted: 16 May 2024
Conditions: Short Bowel Syndrome

Timeline

1 trial · 1 completed · 1 with posted results

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

202220232024

Ph 3
A Study of Teduglutide in Japanese Children With Short Bowel Syndrome Aged 4 Months or Older
NCT05027308
4 Jan 2022 to 27 Sept 2023

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

The main aims of the study are to check for side effects from teduglutide. Participants will receive a daily injection of teduglutide just under the skin (subcutaneous) for 24 weeks. Then they are followed up for another 4 weeks. Participants may be able to repeat this treatment if they meet specific criteria. The study doctors will check for side effects from teduglutide until it becomes commercially available. The maximum duration of treatment is approximately 51.3 weeks.

Interventions

TeduglutideDrug

Arms1

Experimental
Teduglutide 0.05 milligram per kilogram (mg/kg)
Participants will receive 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.

Primary outcomes11

Outcomes the sponsor listed as primary.

Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame · 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})
Registry description
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.
Number of Participants With Serious Adverse Events (SAEs)
Time frame · From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Registry description
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.
Number of Participants With Adverse Events of Special Interest (AESIs)
Time frame · From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Registry description
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.
Number of Participants With Clinically Significant Abnormalities in Vital Signs Reported as an Adverse Event
Time frame · From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Registry description
Vital signs include systolic and diastolic blood pressure, heart rate and body temperature.
Change From Baseline in Body Weight Z-Score at EOT
Time frame · Baseline, EOT (up to 47.3-51.3 weeks)
Registry description
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.
Change From Baseline in Height Z-Score at EOT
Time frame · Baseline, EOT (up to 47.3-51.3 weeks)
Registry description
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.
Change From Baseline in Head Circumference Z-Score at EOT
Time frame · Baseline, EOT (up to 47.3-51.3 weeks)
Registry description
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.
Change From Baseline in Weight-for-Length Z-Score at EOT
Time frame · Baseline, EOT (up to 47.3-51.3 weeks)
Registry description
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).
Number of Participants With Any Laboratory Safety Finding Reported as an Adverse Event
Time frame · From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Registry description
Laboratory safety parameters included biochemistry, hematology, and urinalysis.
Number of Participants With a Change in Urine Output Reported as an Adverse Event
Time frame · From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Registry description
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.
Number of Participants With a Change in Stool Output Reported as an Adverse Event
Time frame · From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Registry description
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.

Secondary outcomes(5)

Change From Baseline in PS Volume
Time frame · 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]
Registry description
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.
Percent Change From Baseline in PS Volume
Time frame · 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]
Registry description
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.
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
Time frame · 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]
Registry description
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.
Number of Participants Who Achieved Enteral Autonomy
Time frame · 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]
Registry description
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.
Change From Baseline in Number of Days Per Week of PS Usage at EOT
Time frame · 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]
Registry description
PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume and calories during the treatment period.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeCount of participants· 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})

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Group	Value (Participants)
Teduglutide	3

Primary outcomeCount of participants· From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])

Number of Participants With Serious Adverse Events (SAEs)

Group	Value (Participants)
Teduglutide	3

Primary outcomeCount of participants· From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])

Number of Participants With Adverse Events of Special Interest (AESIs)

Group	Value (Participants)
Teduglutide	0

Primary outcomeCount of participants· From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])

Number of Participants With Clinically Significant Abnormalities in Vital Signs Reported as an Adverse Event

Group	Value (Participants)
Teduglutide	0

Primary outcomeMean· Baseline, EOT (up to 47.3-51.3 weeks)

Change From Baseline in Body Weight Z-Score at EOT

Group	Value (Z score)	Spread
Teduglutide	1.637	2.8839

Primary outcomeMean· Baseline, EOT (up to 47.3-51.3 weeks)

Change From Baseline in Height Z-Score at EOT

Group	Value (Z score)	Spread
Teduglutide	0.590	1.9949

Primary outcomeMean· Baseline, EOT (up to 47.3-51.3 weeks)

Change From Baseline in Head Circumference Z-Score at EOT

Group	Value (Z score)	Spread
Teduglutide	1.130	0.4101

Primary outcomeMean· Baseline, EOT (up to 47.3-51.3 weeks)

Change From Baseline in Weight-for-Length Z-Score at EOT

Group	Value (Z score)	Spread
Teduglutide	4.240	NA

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
Group	Deaths	Serious events	Other events
TeduglutideParticipants 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.	0 / 3	3 / 3	3 / 3

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies1

Studies that cite this trial.

Studies with results

1 study on this page

Indexing hint: Clinical trial · 2026

8 results

Teduglutide in pediatric patients under 10 kg with short bowel syndrome on parenteral support: An open-label study.

Masumoto et al. 2026Pediatrics international : official journal of the Japan Pediatric Societycited by 2

PMID 41454663PMC12743259DOI 10.1111/ped.70301PUB-41454663

CC BY8 results

Download PDF See results (8)

Eligibility

Who could join, as posted.

Sex: All
Age: from 4 Months
Healthy volunteers: Not accepted

Inclusion and exclusion criteria, as posted

Inclusion Criteria: 1. Male or female pediatric patient of corrected gestational age 4 months or older. 2. Body weight at the time of screening and baseline visits of at least 5 kg and \<10 kg for participants with normal renal function or mild renal impairment (estimated glomerular filtration rate \>=50 mL/min/1.73 m\^2), OR at least 10 kg and \<20 kg for participants with moderate or greater renal impairment (estimated glomerular filtration rate \<50 mL/min/1.73 m\^2). 3. Diagnosis of short bowel syndrome (SBS) with intestinal failure, defined as dependence on PS to provide at least 30% of fluid or caloric needs. 4. Participants to have stable PS for at least 1 month prior to screening as assessed by the investigator. Stable PS is defined as inability to significantly reduce parenteral nutrition/intravenous fluid (PN/IV) support, usually associated with minimal or no advance in enteral feeds (ie, 10% or less change in PN or advance in feeds), assessed by the investigator. Exclusion Criteria: 1. A parent/guardian who is not capable of understanding or not willing to adhere to the study visit schedule and other protocol requirements. 2. Clinically significant intestinal obstruction, active or recurrent pancreatic or biliary disease, or dysmotility that prevents the advancement of enteral intake. 3. Intestinal malabsorption due to a genetic condition, such as cystic fibrosis, microvillus inclusion disease, etc. 4. Severe, known dysmotility syndrome, such as pseudo-obstruction or persistent, severe, active gastroschisis-related dysmotility, that is the primary contributing factor to feeding intolerance and inability to reduce PS, prior to screening. Dysmotility is defined as severe if it is expected to limit the advancement of enteral feeding. 5. Major gastrointestinal (GI) surgical intervention including significant intestinal resection or bowel lengthening procedure within 3 months prior to screening (insertion of feeding tube, anastomotic ulcer repair, minor intestinal resections =\<10 cm and endoscopic procedures are allowed). 6. Cardiac disease that makes the patient vulnerable to changes in fluid status. 7. History of cancer or known cancer predisposition syndrome, such as juvenile polyposis or Beckwith-Wiedemann syndrome, or first degree relative with early onset of GI cancer (including hepatobiliary and pancreatic cancer). 8. Concurrent treatment with glucagon-like peptide-2 (GLP-2), human growth hormone, or analogs of these hormones within 6 months prior to the screening visit, or concurrent treatment with octreotide, or GLP-1 analogs within 30 days prior to the screening visit. 9. Concurrent treatment with biological therapy (eg, anti-tumor necrosis factor \[anti-TNF\]) for active Crohn's disease within 6 months prior to the screening visit. 10. Participation in a clinical study using an experimental drug (other than glutamine or omegaven) within 3 months or 5.5 half-lives of the experimental drug, whichever is longer, prior to the screening visit and for the duration of the study. 11. Known or suspected intolerance or hypersensitivity to the study drug, closely related compounds, or any of the stated ingredients. 12. Signs of active, severe, or unstable clinically significant hepatic impairment during the screening period as meeting at least 2 of any of the following parameters: 1. International normalized ratio \>1.5 not corrected with parenteral vitamin K 2. Platelet count \<100×10\^3/mcrL due to portal hypertension 3. Presence of clinically significant gastric or esophageal varices 4. Cirrhosis 5. Persistent cholestasis defined as conjugated bilirubin \>4 mg/dL (\>68 mcr mol/L) over a 2-week period during screening 6. Total bilirubin \>=2x upper limit of normal (ULN) 7. Aspartate aminotransferase (AST) \>=3x ULN 8. Alanine aminotransferase (ALT) \>=3x ULN

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