NCT07319832 - A Study of Teduglutide in Chinese Children and Teenagers With Short Bowel Syndrome | Crick

Eligibility

Sex: ALLMin age: 1 YearMax age: 17 Years

Medical Language ↔ Plain English

Inclusion criteria * Children and adolescents greater than or equal to (\>=)1 through 17 years of age at Day 1 (D1). * Documented diagnosis of SBS. * Received or plan to receive Teduglutide treatment for a minimum of 24 weeks. * Stable PN/IV support, defined as inability to significantly reduce PN/IV support, usually associated with minimal or no advance in enteral feeds (i.e., 10% or less change in PN or advance in feeds) for at least 3 months prior to D1, as assessed by the investigator. Transient instability for events such as interruption of central access or treatment for sepsis is allowed if the PN/IV support returns to within 10% of baseline prior to the event. * Informed consent obtained from the patient aged 8 to 17 years and their guardians, while informed consent from the guardians for participants under 8 years old, unless waived by the Institution's Ethics Committee. Exclusion criteria * Participants who are not expected to be able to advance oral or tube feeding regimens. * Serial Transverse Enteroplasty (STEP) or any other bowel lengthening procedure performed within 3 months prior to baseline. * Known clinically significant untreated intestinal obstruction contributing to feeding intolerance and inability to reduce PS. * Evidence of clinically significant obstruction on upper GI series done within 6 months prior to baseline. * Previous use of octreotide or Dipeptidyl peptidase-4 (DPP-4) inhibitors within 3 months prior to baseline. * Signs of active, severe, or unstable clinically significant hepatic impairment during the screening or baseline period, indicative by any of the following laboratory test results: 1. Total Bilirubin Level (TBL) \>= 2 × upper limit of normal (ULN) 2. Aspartate Aminotransferase (AST) \>=7 × ULN 3. Alanine Aminotransferase (ALT) \>=7 × ULN For Participants with Gilbert's disease: 4. Indirect (unconjugated) bilirubin \>=2 × ULN * Signs of known continuous active or unstable, clinically significant renal dysfunction shown by results of an estimated glomerular filtration rate (eGFR) below 50 millilitres per minutes per 1.73 meter square (mL/min/1.73 m\^2). * Known hypersensitivity of the active substance or excipient of teduglutide. * Body weight less than (\<) 10 kg at baseline. * Previous use of teduglutide or native/synthetic Glucagon-like Peptide-2 (GLP-2). * Previous use of GLP-1 analog or human growth hormone within 3 months prior to baseline. * Any condition, disease, illness, or circumstance that in the investigator's opinion puts the patient at any undue risk, prevents completion of the study, or interferes with analysis of the study results.

Outcomes

Primary Outcomes

Percentage of Participants Who Achieve at Least a 20% Reduction from Baseline in Weekly Parenteral Support (PS) Volume at Week 24

Percentage of participants who achieve at least a 20 percent (%) reduction from baseline in weekly PS volume at Week 24 will be reported.

Time frame: At Week 24

Secondary Outcomes

Number of Participants Able to Completely Wean off PS Support at Week 24

Number of participants who are able to completely wean off PS support at Week 24 will be reported.

Time frame: At Week 24

Absolute Change from Baseline in PN/Intravenous (IV) Support Volume

Absolute change from baseline in PN/IV support volume will be reported.

Time frame: Baseline up to Week 24

Percent Change From Baseline in PN/IV Support Volume

Percent change from baseline in PN/IV support volume will be reported.

Time frame: Baseline up to Week 24

Absolute Change From Baseline in PN/IV Support Calories

Absolute change from baseline in PN/IV support calories will be reported.

Time frame: Baseline up to Week 24

Percent Change From Baseline in PN/IV Support Calories

Percent change from baseline in PN/IV support calories will be reported.

Time frame: Baseline up to Week 24

Absolute Change From Baseline in Plasma Citrulline

Absolute change from baseline in plasma citrulline will be reported.

Time frame: Baseline, Week 24

Percent Change From Baseline in Plasma Citrulline

Percent change from baseline in plasma citrulline will be reported.

Time frame: Baseline, Week 24

Absolute Change From Baseline in Enteral Nutrition (EN) Support Volume

Absolute change from baseline in EN support volume will be reported.

Time frame: Baseline up to Week 24

Percent Change From Baseline in EN Support Volume

Percent change from baseline in EN support volume will be reported.

Time frame: Baseline up to Week 24

Absolute Change From Baseline in EN Support Calories

Absolute change from baseline in EN support calories will be reported.

Time frame: Baseline up to Week 24

Percent Change From Baseline in EN Support Calories

Percent change from baseline in EN support calories will be reported.

Time frame: Baseline up to Week 24

Change From Baseline in Hours per day of PN/IV Support

Change from baseline in hours per day of PN/IV support will be reported.

Time frame: Baseline up to Week 24

Change From Baseline in Days per Week of PN/IV Support

Change from baseline in days per week of PN/IV support will be reported.

Time frame: Baseline up to Week 24

Percentage of Participants with at Least a 20% Reduction From Baseline in PN/IV Volume

Percentage of participants with at least a 20% reduction from baseline in PN/IV volume will be reported.

Time frame: Baseline up to Week 24

Change in Body Weight

Change in body weight will be reported.

Time frame: Baseline up to Week 24

Change in Height

Change in height will be reported.

Time frame: Baseline up to Week 24

Change in Head Circumference

Head circumference will be measured in participants 36 months of age and younger.

Time frame: Baseline up to Week 24

Change in Height Z-score

Height is measured using Z-score. Z score is calculated as (\[observed value/M\]\^L-1)/S\*L, where observed value is the child's height. The L, M, and S values vary according to the child's sex and age.

Time frame: Baseline up to Week 24

Change in Weight Z-score

Weight is measured using Z-score. Z score is calculated as (\[observed value/M\]\^L-1)/S\*L, where observed value is the child's weight. The L, M, and S values vary according to the child's sex and age.

Time frame: Baseline up to Week 24

Change in Body Mass Index (BMI)

BMI is a simple measure that uses a participant's weight and height to estimate whether they are underweight, normal weight, overweight, or obese. It is calculated as weight in kilograms (kg) divided by height in meters squared (kg/m\^2). Change in BMI will be reported.

Time frame: Baseline up to Week 24

Change in BMI Z-score

BMI is measured using Z-score. Z score is calculated as (\[observed value/M\]\^L-1)/S\*L, where observed value is the child's BMI. The L, M, and S values vary according to the child's sex and age.

Time frame: Baseline up to Week 24

Number of Participants with Adverse Events (AEs). Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)

An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product. An AE does not necessarily have a causal relationship withthis treatment. An AE can therefore be any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product (investigational or marketed), whether or not considered related to treatment with the medicinal product. SAE is any untoward medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of hospitalization, results in persistent or significant disability/incapacity, results in a congenital abnormality/birth defect, or is an important medical event. An AESI is an AE (serious or nonserious) of scientific and medical concern specific to the product or program.

Time frame: Up to 50 weeks

Number of Participants with Clinically Significant Change in Vital Sign Values

Vital signs will include measurement of temperature, heart rate, blood pressure and respiratory rate. Any clinically significant change in vital signs will be determined at the investigator's discretion.

Time frame: Up to 24 weeks

Body Weight in Participants Dependent on PS

Body weight in kg will be collected and reported.

Time frame: Baseline up to Week 24

Head Circumference in Participants Dependent on PS

Head circumference in participants 36 months of age and younger will be collected and reported.

Time frame: Baseline up to Week 24

Height in Participants Dependent on PS

Height in centimeters (cm) will be collected and reported.

Time frame: Baseline up to Week 24

Trends on Growth Chart

Trends on growth chart will be collected and reported.

Time frame: Baseline up to Week 24

Urine Output Determined up to Week 24

Collected and calculated values of urine output will be recorded over a 48-hour period of PN/IV and EN stability before every clinic visit and within 1 week of implementing a change in the PN/IV prescription.

Time frame: Baseline up to Week 24

Fecal Output Measured by Volume

Collected and calculated values of fecal output should be recorded over a 48-hour period of PN/IV and EN stability before every clinic visit and within 1 week of implementing a change in the PN/IV prescription.

Time frame: Baseline up to Week 24

Fecal Output Measured by Number of Bowel Movements per Day

Fecal output should be recorded over a 48-hour period of PN/IV and EN stability before every clinic visit and within 1 week of implementing a change in the PN/IV prescription.

Time frame: Baseline up to Week 24

Number of Participants with Gastrointestinal-specific Testing

Gastrointestinal-specific testing will include colonoscopy or sigmoidoscopy or upper gastrointestinal (GI) endoscopy, abdominal ultrasound, fecal occult blood testing (FOBT), upper gastrointestinal series small bowel follow-through (UGI SBFT).

Time frame: Baseline up to Week 24

Number of Participants With Clinically Significant Changes in Physical Examinations

Physical examination will include assessing the participants physical status. (general surface, skin, head and neck, chest \[heart, lungs, breasts\], abdomen \[gastrointestinal, liver and gallbladder\], back, urinary system, limbs, nervous system, lymph nodes, etc.). Any clinically significant change in physical examination will be determined at the investigator's discretion.

Time frame: Up to 24 weeks

Number of Participants With Clinically Significant Changes in Laboratory Values

Laboratory parameters will include hematology, chemistry and urinalysis. Any clinically significant change in laboratory values will be determined at the investigator's discretion.

Time frame: Up to 24 weeks

Number of Participants With Clinically Significant Changes in 12-Lead Electrocardiogram (ECG) Parameters

The 12-lead ECG will be evaluated. Any clinically significant change in ECG assessment will be determined at the investigator's discretion.

Time frame: Up to 24 weeks

A Study of Teduglutide in Chinese Children and Teenagers With Short Bowel Syndrome

Overview

Conditions

Interventions

Eligibility

Locations (4)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts