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Completed

NCT Number: NCT05027308

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

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.

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Key information

Conditions

Short Bowel Syndrome Digestive System Diseases Gastrointestinal Diseases Intestinal Diseases Malabsorption Syndromes Pathologic Processes Pathological Conditions, Signs and Symptoms Postoperative Complications

Age range

4 month and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

University of Tsukuba Hospital, Tsukuba, Ibaraki, Japan

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About this study

A study of teduglutide in Japanese children with short bowel syndrome aged 4 months or older.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Male or female pediatric patient of corrected gestational age 4 months or older.
  • 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).
  • Diagnosis of short bowel syndrome (SBS) with intestinal failure, defined as dependence on PS to provide at least 30% of fluid or caloric needs.
  • 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

  • A parent/guardian who is not capable of understanding or not willing to adhere to the study visit schedule and other protocol requirements.
  • Clinically significant intestinal obstruction, active or recurrent pancreatic or biliary disease, or dysmotility that prevents the advancement of enteral intake.
  • Intestinal malabsorption due to a genetic condition, such as cystic fibrosis, microvillus inclusion disease, etc.
  • 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.
  • 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).
  • Cardiac disease that makes the patient vulnerable to changes in fluid status.
  • 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).
  • 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.
  • 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.
  • 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.
  • Known or suspected intolerance or hypersensitivity to the study drug, closely related compounds, or any of the stated ingredients.
  • 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:
  • International normalized ratio >1.5 not corrected with parenteral vitamin K
  • Platelet count <100×10^3/mcrL due to portal hypertension
  • Presence of clinically significant gastric or esophageal varices
  • Cirrhosis
  • Persistent cholestasis defined as conjugated bilirubin >4 mg/dL (>68 mcr mol/L) over a 2-week period during screening
  • Total bilirubin >=2x upper limit of normal (ULN)
  • Aspartate aminotransferase (AST) >=3x ULN
  • Alanine aminotransferase (ALT) >=3x ULN

Treatment and study plan

Teduglutide

Drug

Teduglutide 0.05 mg/kg SC injection

Other names: TAK-633

Primary outcomes

  1. 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})

    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.

  2. 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])

    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.

  3. 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])

    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.

  4. 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])

    Vital signs include systolic and diastolic blood pressure, heart rate and body temperature.

  5. Change From Baseline in Body Weight Z-Score at EOT

    Time frame: Baseline, EOT (up to 47.3-51.3 weeks)

    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.

  6. Change From Baseline in Height Z-Score at EOT

    Time frame: Baseline, EOT (up to 47.3-51.3 weeks)

    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.

  7. Change From Baseline in Head Circumference Z-Score at EOT

    Time frame: Baseline, EOT (up to 47.3-51.3 weeks)

    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.

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

    Time frame: Baseline, EOT (up to 47.3-51.3 weeks)

    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).

  9. 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])

    Laboratory safety parameters included biochemistry, hematology, and urinalysis.

  10. 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])

    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.

  11. 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])

    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

  1. 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]

    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.

  2. 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]

    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.

  3. 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]

    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.

  4. 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]

    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.

  5. 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]

    PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume and calories during the treatment period.

Sponsors and collaborators

Lead sponsor

Takeda

Industry

Registry information

Official study title

A Phase 3, Open-label Safety Study of Teduglutide in Japanese Pediatric Patients With Short Bowel Syndrome Who Are Dependent on Parenteral Support, Aged 4 Months of Corrected Gestational Age or Older, and Requiring the Dosing of 1.25 mg Formulation

Important dates

Study start
2022
Primary completion
2023
Study completion
2023
First posted
Aug 30, 2021
Registry last updated
May 16, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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