E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
Short Bowel Syndrome (SBS) is a disease where nutrients are not adequately absorbed by the body due to surgical removal or
malfunctioning of the intestines |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10049416 |
E.1.2 | Term | Short-bowel syndrome |
E.1.2 | System Organ Class | 10017947 - Gastrointestinal disorders |
|
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 this clinical study is to further study long term
safety and efficacy in subjects who have completed 24-weeks of dosing or participated in or qualified for Study CL0600-020: A 24-Week Study of the Efficacy and Safety of Teduglutide in Subjects with Parenteral Nutrition (PN/IV) Dependent Short Bowel Syndrome (SBS).
Long-term safety data and data on PN/IV volume (L/week) will be collected at each study visit. Numbers of subjects who achieve 20, 50, 75 and 100% reduction in PN/IV volume from baseline will be calculated for each visit during this study. |
|
E.2.2 | Secondary objectives of the trial |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Subjects who meet all of the following criteria can be enrolled in this study:
1. Completion of 24-weeks of dosing in Study CL0600-020 or based on investigator and sponsor decision if the subject was required to stop dosing prematurely due to a non-drug related adverse experience or successful completion of stage I (optimization/stabilization) in Study CL0600-020 (ie., qualification for randomization) after approximately 86 subjects have been randomized.
2. Signed and dated informed consent form (ICF) to participate before any study-related procedures of Study CL0600-021 are performed. |
|
E.4 | Principal exclusion criteria |
Exclusion criteria do not apply as only those subjects who have participated in Study CL0600-020 and do not meet the events necessitating withdrawal from the study as listed in Section 4.4.2 of this protocol are eligible. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Data on the PN/IV volume (actual volume, L/week) will be collected at study visits to explore the long-term effects of teduglutide treatement.
Subjects' quality of life (QoL) will be evaluated by using a subject-reported outcome SBS specific QoL scale.
Adverse events, 12-lead ECG, vital signs laboratory safety data, antibodies to teduglutide and/or E.coli protein (ECP), and changes in urine output and body weight will be evaluated. Plasma concentrations of teduglutide will also be measured. Colonoscopy will be performed at the end of the study. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Weeks 2 and 4. Months 2, 3, 6,9,12,15,18,21, and 24 |
|
E.5.2 | Secondary end point(s) |
Blood concentration levels of teduglutide by time and visits will be measured for all subjects who provide samples. In addition, the relationship between PK parameters and
antibody outcomes will be explored. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
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 | Yes |
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 | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
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 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 20 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
|
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
|
Study end is defined as the hard-lock of the database. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 0 |