E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
A complex of symptoms due to a carcinoid tumor producing too many hormones which cause multiple symptoms like diarrhea, flushing, urgent need for a bowel movement. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10007270 |
E.1.2 | Term | Carcinoid syndrome |
E.1.2 | System Organ Class | 10014698 - Endocrine disorders |
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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 study is to evaluate the long-term safety and tolerability of orally administered telotristat etiprate |
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E.2.2 | Secondary objectives of the trial |
To evaluate changes in patients’ quality of life (QOL) through Week 84 |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients must meet all of the following criteria to be considered eligible to participate in the study:
1. Ongoing participation in a Phase 2 study (ie, LX1606.1-202-CS, LX1606.1-203-CS) or Phase 3 study (ie, LX1606.1-301-CS, LX1606.1-303-CS) study
2. Patients of childbearing potential must agree to use an adequate method of contraception (defined as having a failure rate of <1% per year) during the study and for 12 weeks after the Follow-up visit. Adequate methods of contraception for patients or partner include condoms with spermicide gel, diaphragm with spermicide gel, coil (intrauterine device), surgical sterilization, vasectomy, oral contraceptive pill, depot progesterone injections, progesterone implant, and abstinence during the study and for 12 weeks after the Follow-up Visit.
a. Childbearing potential is defined as those who have not undergone surgical sterilization, or those who are not considered postmenopausal. Postmenopause is defined as absence of menstruation for at least 2 years. If necessary, follicle-stimulating hormone (FSH) results >50 IU/L at Baseline Day 1 are confirmatory in the absence of a clear postmenopausal history.
3. Ability and willingness to provide written informed consent prior to participation in any study-related procedure
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E.4 | Principal exclusion criteria |
Patients who meet any of the following criteria will be excluded from participating in the study:
1. Major protocol violations in regard to dosing compliance or telotristat etiprate tolerability concerns in a Phase 2 study (ie, LX1606.1-202-CS, LX1606.1-203-CS) or Phase 3 study (ie, LX1606.1-301-CS, LX1606.1-303-CS) study
2. Positive pregnancy test
3. Presence of any clinically significant findings at entry for medical history, laboratory values, or physical examination (relative to patient population) that, in the Investigator’s or Medical Monitor’s opinion, would compromise patient safety or the outcome of the study
4. Patients who are currently committed to an institution by virtue of an order issued either by judicial or administrative authorities |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is to evaluate the long-term safety and tolerability of orally administered telotristat etiprate.
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
at each visit and the final analysis will be done at the end of the study |
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E.5.2 | Secondary end point(s) |
The secondary endpoint is to evaluate changes in patients’ QOL over 84 weeks of therapy.
Safety endpoints are as follows:
• Incidence of TEAEs, suspected adverse reaction, AEs leading to discontinuation from the study, SAEs, and deaths
• Actual and change from Baseline in clinical laboratory results
• Actual and change from Baseline in vital signs results
• Actual and change from Baseline in physical examinations
• Actual and change from Baseline in ECG findings
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
at each visit and the final analysis will be done at the end of the study |
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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 | Yes |
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 | Yes |
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.8.2.4 | Number of treatment arms in the trial | 1 |
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.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 35 |
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 |
Australia |
Belgium |
Canada |
France |
Germany |
Israel |
Italy |
Netherlands |
Spain |
Sweden |
United Kingdom |
United States |
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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.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 22 |