Symptomatic Carcinoid Syndrome MedDRA version: 15.1 Level: PT Classification code 10007270 Term: Carcinoid syndrome System Organ Class: 10014698 - Endocrine disorders
Conditions
Interventions
Product Name: LX1606
Product Code: LP-778914
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Telotristat etiprate
CAS Number: 1033805-22-9
Current Sponsor code: LX1606
Other descriptive name:
Sponsors
Lexicon Pharmaceuticals, Inc
Eligibility
Sex/Gender
All
Inclusion criteria
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. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to evaluate the safety and tolerability of orally administered LX1606 in subjects with symptomatic carcinoid syndrome.;Secondary Objective: - 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.;Primary 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 leve | — |
Countries
Germany, United Kingdom
Contacts
Public ContactPablo Lapuerta
Lexicon Pharmaceuticals Inc
Outcome results
None listed