Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43871   clinical trials with a EudraCT protocol, of which   7290   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2013-001543-31
    Sponsor's Protocol Code Number:LX1606.1-303-CS
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-09-11
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2013-001543-31
    A.3Full title of the trial
    A Phase 3, Randomized, Placebo-controlled, Multicenter, Double-blind Study to Evaluate the Safety and Efficacy of Telotristat Etiprate (LX1606) in Patients with Carcinoid Syndrome
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3, Randomized, Placebo-controlled, Multicenter, Double-blind Study to Evaluate the Safety and Efficacy of Telotristat Etiprate (LX1606) in Patients with Carcinoid Syndrome
    A.4.1Sponsor's protocol code numberLX1606.1-303-CS
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorLexicon Pharmaceuticals, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportLexicon Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationLexicon Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointPenny Logan
    B.5.3 Address:
    B.5.3.1Street Address8800 Technology Forest Place
    B.5.3.2Town/ cityThe Woodlands
    B.5.3.3Post codeTX 77381
    B.5.3.4CountryUnited States
    B.5.4Telephone number001281 863 3260
    B.5.5Fax number001281863 8088
    B.5.6E-mailplogan@lexpharma.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleComparator
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEMEA/OD/047/09
    D.3 Description of the IMP
    D.3.1Product nameTelotristat Etiprate
    D.3.2Product code LX1606
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTelotristat Etiprate
    D.3.9.1CAS number 1137608-69-5
    D.3.9.2Current sponsor codeLX1606 Hippurate
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Carcinoid Syndrome
    E.1.1.1Medical 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.
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    E.1.2Level PT
    E.1.2Classification code 10007270
    E.1.2Term Carcinoid syndrome
    E.1.2System Organ Class 10014698 - Endocrine disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the effect of telotristat etiprate versus placebo over the double-blind portion of the study on the incidence of treatment-emergent adverse events (TEAEs) and percent (%) change from Baseline in 24-hour urinary 5-hydroxyindoleacetic acid (5-HIAA) levels at Week 12
    E.2.2Secondary objectives of the trial
    To evaluate the effect of telotristat etiprate versus placebo over the double-blind portion of the study on:
    • Change from Baseline in the number of daily bowel movements (BMs) averaged over a 12-week treatment period
    • Change from Baseline in stool consistency, as measured by the Bristol Stool Form Scale averaged across all time points
    • Change from Baseline in the number of cutaneous flushing episodes
    • Change from Baseline in abdominal pain averaged across all time points
    • Change from Baseline in the frequency of rescue short-acting, somatostatin analog (SSA) used to treat carcinoid syndrome (CS) symptoms
    • Change from Baseline in the number of daily BMs averaged over the 12-week treatment period and at each study week, among patients who are not on an SSA at Baseline
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Pharmacogenomics Research
    E.3Principal inclusion criteria
    1. Patients ≥18 years of age at the time of the Screening visit
    2. Patients (males and females) of reproductive 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 patient or partner include condoms with spermicidal gel, diaphragm with spermicidal 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.
    Note: For females, 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 Screening are confirmatory in the absence of a clear postmenopausal history.
    3. Histopathologically-confirmed, well-differentiated metastatic neuroendocrine tumor (NET) confirmed by CT, MRI or radionuclide imaging
    4. Documented history of CS and meeting 1 of the following 2 criteria:
    • If currently receiving LAR/Depot/infusion SSA therapy approved in their country for the treatment of CS, must be currently receving a stable dose, averaging <4 BMs per day, and must have ≥1 of the following sign/symptoms of CS:
    a) Daily stool consistency of type≥5 on Bristol Stool Form Scale9 (Appendix D) for ≥50% of the days during the Run-in, or
    b) Average daily flushing frequency of ≥2, or
    c) Average daily rating of ≥3 for abdominal pain, or
    d) Nausea present ≥20% of days, or
    e) u5-HIAA > ULN
    • If not currently receiving SSA therapy, must have ≥1 of the following sign/symptoms of CS:
    a) Daily stool consistency of type ≥5 on Bristol Stool Form Scale9 (Appendix D) for ≥50% of the days during the Run-in, or
    b) Average daily flushing frequency of ≥2, or
    c) Average daily rating of ≥3 for abdominal pain, or
    d) Nausea present ≥20% of days, or
    e) u5-HIAA > ULN, or
    f) Currently averaging ≥4 BMs per day
    Note: Confirmation of eligibility will be determined by measuring the mean number BMs, stool consistency, abdominal pain, nausea, and/or flushing during the last 2 weeks of the Run-in period.
    5. Ability and willingness to provide written informed consent prior to participation in any study-related procedure
    E.4Principal exclusion criteria
    1. Presence of diarrhea attributed to any condition(s) other than CS including, but not limited to, fat malabsorption or bile acid malabsorption
    2. Presence of >12 watery BMs per day associated with voume contraction, dehydration, or hypotension compatible with a "pancreatic cholera"-type clinical syndrome, as judged by the Investigator
    3. Positive stool examination for enteric pathogens, pathogenic ova or parasites, or Clostridium difficile at Screening
    4. Karnofsky Performance Status ≤60% (see Appendix C)
    5. Clinical laboratory values for hematology (at Screening):
    o Absolute neutrophil count (ANC) ≤1500 cells/mm3; or
    o Platelets ≤75,000 cells/mm3; or
    o Hemoglobin (Hgb) ≤9 g/dL for males and ≤8 g/dL for females
    6. Hepatic laboratory values (at Screening):
    • Asparate transaminase (AST) or alanine aminotransferase (ALT):
    a. ≥5.5 x ULN if patient has documented history of hepatic metastases, or
    b. ≥2.5 x ULN if patient does not have documented history of hepatic metastases
    • Total bilirubin >1.5 x ULN (unless patient has a documented history of Gilbert’s Syndrome), or
    • Alkaline phosphatase (ALP) ≥5 x ULN, if total bilirubin is >ULN
    a. No upper limit on the ALP value if the total bilirubin is ≤ULN
    7. Serum creatinine ≥1.5 x ULN
    8. Treatment with any tumor-directed therapy including, but not limited to: interferon, chemotherapy, mTOR inhibitors ≤4 weeks prior to Screening. or hepatic embolization, radiotherapy, radiolabelled SSA, and/or tumor debulking ≤12 weeks prior to Screening
    9. Major surgery defined as procedures requiring general anesthesia or major regional anesthesia within 8 weeks prior to Screening
    10. A history of short bowel syndrome (SBS)
    11. Current complaints of constipation or history of chronic or idiopathic constipation within 2 years prior to Screening
    12. Positive pregnancy test, pregnant or nursing (lactating) female patients only
    13. Life expectancy <12 months from the Screening visit
    14. Presence of any clinically significant findings at Screening medical history, 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
    15. Any other clinically significant laboratory abnormality at Screening that would compromise patient safety or the outcome of the study
    16. Clinically significant cardiac arrhythmia, bradycardia, or tachycardia that would compromise patient safety or the outcome of the study
    17. A history of substance or alcohol abuse (DSM-IV Criteria for Substance-Related Disorders12) within 2 years prior to Screening
    18. Administration of any investigational agent within 30 days of Screening or investigational therapeutic protein or antibody within 90 days prior to Screening
    19. Patients who are currently committed to an institution by virtue of an order issued either by judicial or administrative authorities
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is the change from Baseline in the number of daily BMs averaged over a 12-week treatment period.

    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

    E.5.1.1Timepoint(s) of evaluation of this end point
    at each visit and the final analysis will be done at the end of the study
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints include:
    • Change from Baseline in stool consistency averaged across all time points
    • Change from Baseline in the number of cutaneous flushing episodes
    • Change from baseline in abdominal pain averaged across all time points
    • Change in the frequency of rescue short-acting SSA is used to treat CS symptoms
    • Change from Baseline in the number of daily BMs averaged over the 12-week treatment period and at each study week, among patients who are not on an SSA at Baseline
    E.5.2.1Timepoint(s) of evaluation of this end point
    at each visit and the final analysis will be done at the end of the study
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    There is a 12-week double blind treatment phase and then a 36 week open label treatment phase
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Two different doses of LX1606
    E.8.2.4Number of treatment arms in the trial3
    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.8.4.1Number of sites anticipated in Member State concerned8
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA37
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Belgium
    Brazil
    Canada
    France
    Germany
    Israel
    Italy
    Netherlands
    Spain
    Sweden
    United Kingdom
    United States
    E.8.7Trial 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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months8
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 42
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 18
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state11
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 39
    F.4.2.2In the whole clinical trial 60
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    After the end of this study the patients will continue the standard of care available for this indication in the respective country. Another option, assuming adequate clinical benefit is observed, patients may receive further treatment of study drug in an extension study.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2014-01-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-12-18
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-03-29
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Sat May 04 18:40:47 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA