E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Treatment of short bowel syndrome |
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E.1.1.1 | Medical condition in easily understood language |
Treatment of short bowel syndrome |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nutritional and Metabolic Diseases [C18] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective of the study is to evaluate the long-term safety and tolerability of teduglutide treatment in Japanese pediatric subjects with SBS who completed Study SHP633-302. |
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E.2.2 | Secondary objectives of the trial |
The secondary objective of this study is to evaluate the long-term efficacy of teduglutide treatment in Japanese pediatric subjects with SBS who completed Study SHP633-302. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Informed consent by a parent or guardian prior to any study-related procedures. - When applicable, informed assent (as deemed appropriate by the Institutional Review Board [IRB]) by the participant prior to any study-related procedures. - Participant completed Study SHP633-302 (NCT02980666). - Participant (and/or parent/guardian) understands and is willing and able to fully adhere to study requirements as defined in this protocol. |
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E.4 | Principal exclusion criteria |
There are no exclusion criteria for this study. |
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E.5 End points |
E.5.1 | Primary end point(s) |
- Adverse events - Vital signs, including temperature, heart rate, blood pressure - Laboratory safety data (ie, biochemistry, hematology, and urinalysis) - Urine output - Stool output - Antibodies to teduglutide - Gastrointestinal-specific testing, including fecal occult blood testing and colonoscopy or sigmoidoscopy - Z-scores for weight, height (or length), head circumference (up to 36 months of age), and BMI |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Baseline to each study visit for approximately 3 years |
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E.5.2 | Secondary end point(s) |
- Reduction in parenteral support (PS) volume of at least 20% - Absolute and relative change in PS volume - Complete weaning off PS - Change in days per week of PS |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Baseline to each study visit for approximately 3 years |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 4 |