A Study of ABC294640 Alone and in Combination With HCQ Sulfate in the Treatment of Advanced Chola… (NCT03377179) | Clinical Trial Compass
CompletedPhase 2
A Study of ABC294640 Alone and in Combination With HCQ Sulfate in the Treatment of Advanced Cholangiocarcinoma
United States65 participantsStarted 2018-03-07
Plain-language summary
ABC-108 is a single-arm Phase IIA clinical study of ABC294640 (Yeliva ®, opaganib) alone and in combination with hydroxychloroquine sulfate (HCQ) in the treatment of cholangiocarcinoma (CCA). In Part 1 of this clinical study, all participants will be receiving ABC294640 and in Part 2 all participants will be receiving ABC294640 and HCQ to explore the drugs activity signal in CCA. The study drug, ABC294640 is an orally available inhibitor of the enzyme sphingosine kinase-2 (SK2). SK2 is an innovative target for anti-cancer therapy because of its critical role in sphingolipid metabolism, which is known to regulate tumor cell death and proliferation. ABC294640 also inhibits proliferation and induces apoptosis of cholangiocarcinoma cell lines. Furthermore, in a recent Phase I trial, ABC294640 demonstrated clinical activity in CCA patients. HCQ, is an orally available, FDA approved therapy for the treatment of malaria as well as discoid and systemic lupus erythematosus and rheumatoid arthritis. It is also known as an inhibitor of autophagy, a pro-survival mechanism utilized by many cancers. Evidence indicates that inhibition of autophagy can increase the therapeutic activity of ABC294640 in CCA. In Part 1 of this study, ABC294640 will be continuously administrated orally, twice a day, in 28 day cycles. In Part 2, ABC294640 and HCQ will be continuously administrated orally (the safe and tolerable will be determined in the study) in 28 day cycles. Administration of drug/s in both parts of the study will continue until disease progression, unacceptable toxicity or voluntary withdrawal initiated by the participants or physician.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Patients with histologically confirmed intrahepatic, perihilar or extra-hepatic CCA.
. Patients with no more than 2 prior treatments with systemic anti-neoplastic therapy for CCA.
. The tumor is unresectable and not amenable to curative therapy.
. One or more tumors measurable on CT scan per RECIST 1.1.
. Eastern Cooperative Oncology Group (ECOG) performance status 0- 1.
. Life expectancy of at least 3 months.
. Age ≥18 years.
. Signed, written IRB-approved informed consent.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial tested a drug called ABC294640, sometimes alone and sometimes combined with hydroxychloroquine sulfate, for advanced cholangiocarcinoma — can you explain what each of these drugs does and why combining them was being studied for my specific type of bile duct cancer?
2Since this was a Phase 2 trial that is now completed, has the response rate data from Part 1 or the durable disease control rate from Part 2 been published yet, and do the results look promising enough to influence my treatment plan?
3Because this trial is closed to enrollment, are there any currently open trials studying similar approaches — like autophagy inhibition or sphingosine kinase inhibition — that might be worth considering for advanced cholangiocarcinoma?
4Given that my cancer may be intrahepatic, extrahepatic, or perihilar, does the location of my tumor affect how relevant these trial results are to my situation, and would standard chemotherapy still be a better first step?
5If the completed trial showed any signals of benefit or notable side effects from this drug combination, how should that information factor into the options you're recommending for me right now?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Part1 - Determine Response Rate
Timeframe: At least 4 months
2
Part 2 - Determine the Durable Disease Control Rate
. \>2 previous systemic anti-neoplastic regimens for CCA.
. Previously having received ABC294640 or HCQ (or chloroquine) for the treatment of a malignancy.
. New York Heart Association Class III or IV, cardiac disease, myocardial infarction within the past 6 months, unstable arrhythmia, or evidence of ischemia on ECG.
. Pregnant or nursing women. NOTE: If a woman became pregnant or suspects she is pregnant while participating in this study, she must inform her treating physician immediately.
. Treatment with radiation therapy, surgery, chemotherapy, or investigational therapy within 28 days prior to study entry.
. Unwillingness or inability to comply with procedures required in this protocol.
. Known infection with human immunodeficiency virus.