A Safety and Efficacy Study of XERMELO® + First-line Chemotherapy in Patients With Advanced Biliary Tract Cancer

NCT03790111 | Terminated Phase 2 Results FDA Drug

• 2 other identifiers: LX1606.1-207-BTCLX1606.207
• Study Type: interventional
• Target: 53
• Locations: 1 country
• Sites: 14

Brief Summary

A Phase 2, multicenter, open-label, 2-stage study to assess the safety, tolerability, and efficacy of XERMELO in combination with first-line (1L) therapy (cisplatin \[cis\] plus gemcitabine \[gem\])

Conditions

Biliary Tract Cancer (BTC)

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Progression-free Survival (PFS) as Evaluated by Central Radiologist's Assessment

  Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions", or similar definition as accurate and appropriate.

  Month 6

• Project Overall Survival Rate at Month 6

  Overall Survival (OS) was defined as the time from the frist dose of study treatment until the date of death due to any cause. Duration of Overall Survival (OS) in days is defined as (Date of event/censoring- date of First dose +1). Use Kaplan Meier method to project survival rate at month 6.

  6 Months

Secondary Outcomes (30)

• Overall Survival (OS)

  First dose of study treatment until the date of death due to any cause, whichever came first, a median of approximately 17.67 months

• Project Overall Survival Rate at Month 12

  12 Months

• Median Progression Free Survival

  Month 12

• Disease Control Rate (DCR), Central Radiologist's Assessment

  Month 6

• Disease Control Rate (DCR), Central Radiologist's Assessment

  Month 12

Study Arms (1)

Xermelo 250mg plus first line therapy for a week, then Xermelo 500mg

EXPERIMENTAL

Xermelo 250 milligram (mg) plus first line therapy for a week, then Xermelo 500mg plus first line therapy for the duration of the study

Drug: telotristat ethyl

Interventions

telotristat ethyl DRUG

XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) three times a day plus first line therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) three times a day plus first line therapy for the duration of the study

Also known as: telotristat ethyl + (cisplatin [cis] plus gemcitabine [gem])

Xermelo 250mg plus first line therapy for a week, then Xermelo 500mg

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female adults, ≥18 years of age. Patients of childbearing potential must agree to use an adequate method of contraception during the study and for 30 days after the last dose of XERMELO
• Histopathologically or cytologically-confirmed, unresectable, locally advanced, recurrent, or metastatic biliary tract cancer (BTC)
• Naïve to tumor-directed therapy in locally advanced, unresectable, or metastatic setting
• Measurable disease
• Eastern Cooperative Oncology Group (ECOG) performance status 0-1
• Plans to initiate treatment with 1L therapy (cisplatin plus gemcitabine)
• Ability to provide written informed consent prior to participation in any study-related procedure

You may not qualify if:

• Prior exposure to XERMELO, telotristat ethyl, telotristat etiprate, LX1032, or LX1606
• Primary tumor site in the ampulla of Vater
• Treatment with photodynamic therapy for localized disease or to relieve biliary obstruction in the presence of metastatic disease within the past 30 days
• Hematology laboratory values of: a. Absolute neutrophil count (ANC) ≤1,500 cells/mm\^3; or b. Platelets ≤100,000 cells/mm\^3; or c. Hemoglobin (Hgb) ≤9 g/dL; or d. White blood count (WBC) ≤3,000 cells/mm\^3
• Hepatic laboratory values of aspartate aminotransferase (AST) or alanine aminotransferase (ALT): a. \>5 x upper limit of normal (ULN) if patient has documented history of hepatic metastases; or b. \>2.5 x ULN if no liver metastases are present
• Serum albumin \<2.8 g/dL
• Total bilirubin \>1.5 x ULN or \>1.5 mg/dL
• Prothrombin time (PT) or international normalized ratio (INR) \>1.5 x ULN
• Serum creatinine or serum urea \>1.5 x ULN
• Estimated glomerular filtration rate (eGFR) \<50 mL/min
• Positive pregnancy test, pregnant, or breastfeeding
• Any other clinically significant laboratory abnormality that would compromise patient safety or the outcome of the study
• Any clinically significant and/or uncontrolled cardiac-related abnormality that would compromise patient safety or the outcome of the study
• Myocardial infarction within the past 6 months
• Active bleeding diathesis

Sponsors & Collaborators

• TerSera Therapeutics LLC: lead

Study Sites (14)

TerSera Investigational Site

Gilbert, Arizona, 85234, United States

Location

TerSera Investigational Site

Fullerton, California, 92835, United States

Location

TerSera Investigational Site

Gainesville, Florida, 32610, United States

Location

TerSera Investigational Site

Tampa, Florida, 33612, United States

Location

TerSera Investigational Site

Chicago, Illinois, 60637, United States

Location

TerSera Investigational Site

Westwood, Kansas, 66205, United States

Location

TerSera Investigational Site

Lexington, Kentucky, 40536, United States

Location

TerSera Investigational Site

Boston, Massachusetts, 02118, United States

Location

TerSera Investigational Site

Grand Rapids, Michigan, 49503, United States

Location

TerSera Investigational Site

Buffalo, New York, 14263, United States

Location

TerSera Investigational Site

Lake Success, New York, 11042, United States

Location

TerSera Investigational Site

Charlotte, North Carolina, 28204, United States

Location

TerSera Investigational Site

Oklahoma City, Oklahoma, 73104, United States

Location

TerSera Investigational Site

San Antonio, Texas, 78229, United States

Location

MeSH Terms

Conditions

Biliary Tract Neoplasms

Interventions

telotristat ethyl; Cisplatin; cytokine inducible SH2-containing protein

Condition Hierarchy (Ancestors)

Digestive System Neoplasms; Neoplasms by Site; Neoplasms; Biliary Tract Diseases; Digestive System Diseases

Intervention Hierarchy (Ancestors)

Chlorine Compounds; Inorganic Chemicals; Nitrogen Compounds; Platinum Compounds

Limitations and Caveats

This study was halted prematurely as it did not meet pre-specified Progression-Free Survival at Month 6 for Stage 2 Analysis. Due to early discontinuation of the study with limited data, the study results (data) at Month 12 and End of Study may be unreliable and uninterpretable.

Results Point of Contact

• Title: Janine North, Executive Director-Clinical Development
• Organization: TerSera Therapeutics Inc.

jnorth@tersera.com

888 600 8116

Study Officials

• Renuka Iyer, M.D.

  Roswell Park Cancer Institute

  STUDY CHAIR

• Richard Kim, M.D.

  H. Lee Moffitt Cancer Center and Research Institute

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 25, 2018
• First Posted: December 31, 2018
• Study Start: March 13, 2019
• Primary Completion: January 13, 2022
• Study Completion: January 13, 2022
• Last Updated: April 19, 2023
• Results First Posted: April 19, 2023

Record last verified: 2023-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (14)