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Protocol No. UW17015

Principal Investigator Lubner, Sam

Phase III

Age Group Adult

Scope National

Sponsor Type Industry

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

Objective AG-120-C-005 is a clinical research study that is designed to evaluate the efficacy and safety of AG-120 as compared to placebo in patients with advanced cholangiocarcinoma. We want to find out what effects, good or bad, either AG-120 or placebo has on you and your disease.

Treatment The use of AG-120 in this study is experimental, which means that it is not approved by the U.S. Food and Drug Administration (FDA) or other regulatory authorities around the world for use alone or in combination with any drug.

Description Isocitrate dehydrogenase 1 (IDH1) is a type of protein involved in normal cell metabolism, the process of providing your body’s cells with energy. In certain types of diseases such as cholangiocarcinoma, an abnormal form of the IDH1 protein is present in the cancer cells. When IDH1 is present in this form, it produces an excess amount of 2-hydroxyglutarate (2-HG), which is a substance that is present in low levels in normal cells. When 2-HG is present in excessive amounts, it results in changes within the cells which may result in cholangiocarcinoma. AG-120 may stop the abnormal IDH1 protein and may reduce 2-HG levels in diseased cells to normal levels. Your cells will be tested to see if they carry the abnormal IDH1 gene which results in the production of the abnormal IDH1 protein. You will only be enrolled in this study and treated with AG-120 or placebo if your diseased cells carry the abnormal IDH1 gene and you meet the other eligibility criteria required. AG-120 or placebo treatment is to be taken by mouth once daily without interruption.

Key Eligibility
  • Be ≥18 years of age
  • 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
  • Have documented IDH1 gene-mutated disease based on central laboratory testing
  • Have at least one evaluable and measurable lesion
  • 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
  • If patient received a prior IDH inhibitor, they are ineligible
  • If patient received systemic anticancer therapy or an investigational agent <2 weeks prior to Day 1 (washout from prior immune based anticancer therapy is 4 weeks) they are ineligible
  • If patient received radiotherapy to metastatic sites of disease <2 weeks prior to Day 1, they are ineligible
  • If patient underwent hepatic radiation, chemoembolization, and radiofrequency ablation <4 weeks prior to Day 1, they are ineligible
  • If patient has known symptomatic brain metastases requiring steroids, they are ineligible
  • Women who are pregnant or breastfeeding are ineligible
  • Patients who have known active hepatitis B (HBV) or hepatitis C (HCV) infections, known positive human immunodeficiency virus (HIV) antibody results, or acquired immunodeficiency syndrome (AIDS) related illness are ineligible
  • Patients who have known active inflammatory gastrointestinal disease, chronic diarrhea, previous gastric resection or lap band dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally are ineligible

Applicable Disease Sites Gastrointestinal cancers, other

Status Open

Participating Institutions UW Hospital and Clinics