This site became the new ClinicalTrials.gov on June 19th. Learn more.
Show more
ClinicalTrials.gov Menu IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu IMPORTANT: Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu
Give us feedback

Study of AG-120 in Previously Treated Advanced Cholangiocarcinoma With IDH1 Mutations (ClarIDHy) (ClarIDHy)

This study is currently recruiting participants.
See Contacts and Locations
Verified November 2016 by Agios Pharmaceuticals, Inc.
Sponsor:
Information provided by (Responsible Party):
Agios Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT02989857
First received: December 5, 2016
Last updated: August 21, 2017
Last verified: November 2016
  Purpose
Study AG120-C-005 is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study of orally administered AG-120. Subjects, all personnel involved in the evaluation of subjects' response to treatment (e.g., Investigators, study coordinators, study pharmacists), and designated Sponsor team members will be blinded to study treatment. Subjects are required to have a histologically-confirmed diagnosis of IDH1 gene-mutated cholangiocarcinoma that is not eligible for curative resection, transplantation, or ablative therapies prior to enrollment.IDH1 mutation testing will be performed at participating investigative sites. Subjects must have progression of disease and have received at least 1 but not more than 2 prior treatment regimens for advanced disease (nonresectable or metastatic). All subjects must have received either a gemcitabine or a 5 fluorouracil (5-FU) based chemotherapy regimen.

Condition Intervention Phase
Advanced Cholangiocarcinoma Metastatic Cholangiocarcinoma Drug: AG-120 Drug: AG-120 matched placebo Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-controlled Study of AG-120 in Previously-treated Subjects With Nonresectable or Metastatic Cholangiocarcinoma With an IDH1 Mutation

Resource links provided by NLM:


Further study details as provided by Agios Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Progression Free Survival (PFS) [ Time Frame: Up to 26 weeks, on average ]

Secondary Outcome Measures:
  • Adverse Event (AE) and Serious Adverse Event (SAE) analysis [ Time Frame: Up to 26 weeks, on average ]
  • Overall Survival (OS) [ Time Frame: Up to 52 weeks, on average ]
  • Overall Response Rate (ORR) [ Time Frame: Up to 26 weeks, on average ]
  • Quality of Life (QOL) [ Time Frame: Up to 52 weeks, on average ]
    Method of assessment will be questionnaires. Questionnaire: EORTC QLQ-C30

  • Quality of Life (QOL) [ Time Frame: Up to 52 weeks, on average ]
    Method of assessment will be questionnaires. Questionnaire: EORTC QLQ-Bil21

  • Quality of Life (QOL) [ Time Frame: Up to 26 weeks, on average ]
    Method of assessment will be questionnaires. Questionnaire: EuroQOL EQ-5D-5L


Estimated Enrollment: 186
Actual Study Start Date: December 2016
Estimated Study Completion Date: August 2020
Estimated Primary Completion Date: November 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: AG-120 experimental study drug
AG-120, 500mg daily continuous dosing
Drug: AG-120
Placebo Comparator: AG-120 matched placebo
AG-120 matched placebo, daily continuous dosing. Subjects who experience disease progression and were receiving placebo, will be allowed to cross-over and receive AG-120
Drug: AG-120 matched placebo

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Be ≥18 years of age.
  2. Have a histopathological diagnosis (fresh or banked tumor biopsy sample, preferably collected within the last 3 years) of nonresectable or metastatic cholangiocarcinoma and are not eligible for curative resection, transplantation, or ablative therapies.
  3. Have documented IDH1 gene-mutated disease (from a fresh tumor biopsy or the most recent banked tumor tissue available) based on central laboratory testing (R132C/L/G/H/S mutation variants tested).
  4. Have an ECOG PS score of 0 or 1
  5. Have an expected survival of ≥3 months.
  6. Have at least one evaluable and measurable lesion as defined by RECIST v1.1. Subjects who have received prior local therapy (including but not limited to embolization, chemoembolization, radiofrequency ablation, or radiation therapy) are eligible provided measurable disease falls outside of the treatment field or within the field and has shown ≥20% growth in size since post-treatment assessment.
  7. Have documented disease progression following at least 1 and no more than 2 prior systemic regimens for advanced disease (nonresectable or metastatic). Subjects must have received at least 1 gemcitabine- or 5-FU-containing regimen for advanced cholangiocarcinoma. Subjects who have received systemic adjuvant chemotherapy will be permitted provided there is documented disease progression during or within 6 months of completing the therapy.

Exclusion criteria:

  1. Received a prior IDH inhibitor.
  2. Received systemic anticancer therapy or an investigational agent <2 weeks prior to Day 1 (washout from prior immune based anticancer therapy is 4 weeks). In addition, the first dose of study treatment should not occur before a period ≥5 half-lives of the investigational agent has elapsed.
  3. Received radiotherapy to metastatic sites of disease <2 weeks prior to Day 1.
  4. Underwent hepatic radiation, chemoembolization, and radiofrequency ablation <4 weeks prior to Day 1.
  5. Have known symptomatic brain metastases requiring steroids. Subjects with previously diagnosed brain metastases are eligible if they have completed their treatment and have recovered from the acute effects of radiation therapy or surgery prior to study entry, have discontinued corticosteroid treatment for these metastases for at least 4 weeks and have radiographically stable disease for at least 3 months prior to study entry. Note: up to 10 mg per day of prednisone equivalent will be allowed.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02989857

Contacts
Contact: Medical Affairs Agios Pharmaceuticals, Inc. 844-633-2332 Medinfo@agios.com

  Hide Study Locations
Locations
United States, Arizona
Mayo Cancer Center Recruiting
Scottsdale, Arizona, United States
United States, California
University of Southern California Recruiting
Los Angeles, California, United States
University of California, San Francisco Recruiting
San Francisco, California, United States
United States, Florida
Mayo Cancer Center Recruiting
Jacksonville, Florida, United States
United States, Illinois
University of Chicago Medical Center Recruiting
Chicago, Illinois, United States
United States, Maryland
Johns Hopkins University Recruiting
Baltimore, Maryland, United States
United States, Massachusetts
Dana-Farber Cancer Institute Recruiting
Boston, Massachusetts, United States
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States
United States, Michigan
University of Michigan Recruiting
Ann Arbor, Michigan, United States
United States, Missouri
Washington University Recruiting
Saint Louis, Missouri, United States
United States, New York
Memorial Sloan-Kettering Cancer Center Recruiting
New York, New York, United States
United States, Pennsylvania
Fox Chase Cancer Center Recruiting
Philadelphia, Pennsylvania, United States
United States, South Carolina
Gibbs Cancer Center Recruiting
Spartanburg, South Carolina, United States
United States, Tennessee
Sarah Cannon Research Institute Recruiting
Nashville, Tennessee, United States
Vanderbilt University Medical Center Recruiting
Nashville, Tennessee, United States
United States, Texas
University of Texas, SouthWestern Recruiting
Dallas, Texas, United States
UT MD Anderson Cancer Center Recruiting
Houston, Texas, United States
United States, Utah
University of Utah, Huntsman Cancer Institute Recruiting
Salt Lake City, Utah, United States
United States, Washington
Seattle Cancer Care Alliance Recruiting
Seattle, Washington, United States
United States, Wisconsin
University of Wisconsin, Carbone Cancer Center Recruiting
Madison, Wisconsin, United States
Spain
Hospital Vall d'Hebrón Recruiting
Barcelona, Spain
Centro Integral Oncologico Clara Campal Recruiting
Madrid, Spain
Hospital General Universitario Gregorio Marañón Recruiting
Madrid, Spain
Hospital Universitario 12 de Octubre Recruiting
Madrid, Spain
Sponsors and Collaborators
Agios Pharmaceuticals, Inc.
Investigators
Study Chair: Medical Affairs Agios Pharmaceuticals, Inc. Agios Pharmaceuticals, Inc.
  More Information

Responsible Party: Agios Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02989857     History of Changes
Other Study ID Numbers: AG120-C-005
Study First Received: December 5, 2016
Last Updated: August 21, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Agios Pharmaceuticals, Inc.:
IDH1

Additional relevant MeSH terms:
Cholangiocarcinoma
Adenocarcinoma
Carcinoma
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms

ClinicalTrials.gov processed this record on September 19, 2017