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    Summary
    EudraCT Number:2009-016973-13
    Sponsor's Protocol Code Number:LX1606.1-203-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:2009-12-17
    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 number2009-016973-13
    A.3Full title of the trial
    A Phase 2, Open-Label, Multi-Center, Serial Ascending-Dose, Dose Finding Study to Evaluate the Safety and Tolerability of LX1606 in Subjects with Symptomatic Carcinoid Syndrome
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2, Open-Label, Multi-Center, Serial Ascending-Dose, Dose Finding Study to Evaluate the Safety and Tolerability of LX1606 in Subjects with Symptomatic Carcinoid Syndrome
    A.4.1Sponsor's protocol code numberLX1606.1-203-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 pointPablo Lapuerta
    B.5.3 Address:
    B.5.3.1Street Address350 Carter Road
    B.5.3.2Town/ cityPrinceton
    B.5.3.3Post codeNJ 08540
    B.5.3.4CountryUnited States
    B.5.4Telephone number001 609 466-5590
    B.5.5Fax number001 609 466-3562
    B.5.6E-mailplapuerta@lexpharma.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    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 nameLX1606
    D.3.2Product code LP-778914
    D.3.4Pharmaceutical form Capsule, hard
    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 1033805-22-9
    D.3.9.2Current sponsor codeLX1606
    D.3.9.3Other descriptive nameLP-778914
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50 to mg
    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.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    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 nameLX1606
    D.3.2Product code LP-778914
    D.3.4Pharmaceutical form Capsule, hard
    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 1033805-22-9
    D.3.9.2Current sponsor codeLX1606
    D.3.9.3Other descriptive nameLP-778914
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250 to mg
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Symptomatic Carcinoid Syndrome
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 15.1
    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
    The primary objective of this study is to evaluate the safety and tolerability of orally administered LX1606 in subjects with symptomatic carcinoid syndrome.
    E.2.2Secondary objectives of the trial
    - To assess the effects of LX1606 on symptomatic response
    - To evaluate the effects of LX1606 over a range of multiple oral doses on the number of cutaneous flushing episodes.
    - To assess the proportion of subjects achieving clinically meaningful symptom reduction over a range of multiple oral doses of LX1606.
    - To assess the concentrations of LX1606 and its active moiety, LP-778902, in plasma over a range of multiple oral doses in subjects with symptomatic carcinoid syndrome.
    - To assess the pharmacodynamic effects of LX1606 over a range of multiple oral doses on 5 HT levels in blood over time versus baseline.
    - To assess the pharmacodynamic effects of LX1606 over a range of multiple oral doses on 5 hydroxyindoleacetic acid (5 HIAA) levels in urine over time versus baseline.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Adult subjects aged ≥18 years at the time of the Screening visit.
    2. Male or female; males and females of childbearing potential must agree to use an adequate method of contraception during the study and for 30 days after the last Follow-Up visit.
    3. Biopsy-proven metastatic carcinoid tumor of the GI tract with disease extent confirmed by computed tomography (CT), magnetic resonance imaging (MRI), or radionuclide imaging.
    4. Symptomatic carcinoid syndrome, defined as experiencing an average of ≥ 4 bowel movements per day. Confirmation of eligibility will be determined by measuring the mean number of bowel movements during the Run-In period.
    5. Ability and willingness to provide written informed consent prior to participation in any study-related activities and to participate in and comply with the study requirements.
    E.4Principal exclusion criteria
    1. Presence of volume contraction, dehydration, or hypotension compatible with a “pancreatic cholera”-type clinical syndrome, as judged by the Investigator.
    2. Karnofsky Performance Status ≤ 70% (see Appendix D).
    3. Clinical laboratory values for hematology (at Screening):
    a. Absolute neutrophil count (ANC) ≤ 1500 cells/mm3; or
    b. Platelets ≤ 100,000 cells/mm3; or
    c. Hemoglobin (Hgb) ≤ 9 g/dL.
    4. Liver function test levels (at Screening) such that:
    a. Asparate transaminase (AST), alanine aminotransferase (ALT) ≥ five times the upper limit of normal (2 x ULN);or
    b. Total bilirubin > ULN; or
    c. Alkaline Phosphatase (ALP) ≥ 1.5 x ULN;Higher values will be considered if the investigator deems the elevation in ALP is due to the underlying carcinoid tumor burden and not progressive liver failure.; or
    d. Serum creatinine ≥ 1.5 x ULN.
    5. Any other clinically significant laboratory abnormality at Screening.
    6. Any clinically significant abnormalities on resting ECG (relative to subject population) at Screening.
    7. Surgery within 60 days prior to Screening.
    8. A history of short bowel syndrome (SBS).
    9. History of positive hepatitis B surface antigen (HBsAg), hepatitis C antibody (HCV Ab), human immunodeficiency virus (HIV) 1, or HIV 2.
    10. Pregnant or nursing (lactating) women.
    11. Positive pregnancy test at Screening.
    12. A history of bleeding diathesis.
    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 subject population) that, in the Investigator’s or Sponsor’s opinion, would compromise the outcome of the study.
    15. A history of, or the existence of, any surgical or medical condition that, in the judgment of the Investigator, might interfere with the absorption, distribution, metabolism, or excretion of LX1606.
    16. Any concurrent conditions that could interfere with safety and/or tolerability measurements.
    17. A history of substance or alcohol abuse within 2 years prior to Screening.
    18. Participation in any other investigational drug study within 30 days prior to Screening.
    19. Administration of any investigational agent within 30 days of Screening or any therapeutic protein or antibody within 90 days prior to Screening.
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy Endpoints
    • Number of daily bowel movements;
    • Improvement in stool form/consistency;
    • Reduction in sensation of urgency to defecate;
    • Reduction in sensation/severity of nausea;
    • Improvement in subject global assessment of symptoms associated with carcinoid syndrome;
    • Improvement in levels of abdominal pain or discomfort;
    • Reduction in the number of flushing episodes per day;
    • Proportion of subjects who achieve a clinically meaningful symptom reduction.

    Safety Endpoints
    • Incidence of treatment-emergent AEs, drug-related AEs, deaths, SAEs, and AEs leading to discontinuation from the study;
    • Changes from baseline in clinical laboratory results; shifts from baseline to abnormal clinical laboratory results;
    • Changes from baseline in vital signs results; shifts from baseline to abnormal vital signs results;
    • Clinically significant changes from baseline in ECG findings

    Pharmacokinetic and Pharmacodynamic Endpoints
    The pharmacokinetic endpoints will be trough levels of LX1606 and LP-778902 for each dose group.

    Other Endpoints
    • Change from baseline in CgA levels;
    • Change in the frequency of concomitant medications used to treat symptoms of carcinoid syndrome.
    • Change from baseline in symptom improvement as assessed by Investigator
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 EEA13
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    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
    End of trial is defined as last patient last visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months11
    E.8.9.2In all countries concerned by the trial days0
    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.1.3Elderly (>=65 years) Yes
    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 state16
    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 completion of the clinical trial, 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 Decision2010-03-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2010-02-12
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-02-12
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    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
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