Mercaptopurine for the Treatment of Hereditary Leiomyomatosis and Renal Cell Carcinoma (HLRCC), U… (NCT07716735) | Clinical Trial Compass
Not Yet RecruitingPhase 1
Mercaptopurine for the Treatment of Hereditary Leiomyomatosis and Renal Cell Carcinoma (HLRCC), Uterine/Cutaneous Leiomyomas, and Kidney Cancer
United States18 participantsStarted 2026-12-01
Plain-language summary
This phase I/II trial studies the side effects and best dose of mercaptopurine, and to see how well it works in treating patients with hereditary leiomyomatosis and renal cell carcinoma (HLRCC). HLRCC is a rare inherited disorder that increases the risk of developing benign (not cancer) tumors of the skin and the uterus (leiomyomas) and malignant (cancer) tumors of the uterus (leiomyosarcoma) and the kidney. Mercaptopurine is in a class of medications called purine antagonists. It works by stopping the growth of cancer cells.
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:
* Age ≥ 18
* Disease-causing, germline FH mutation including variants considered either:
* a) Pathogenic/likely pathogenic OR
* b) variants of unknown significance (VUS) with immunohistochemical staining showing loss of FH or high 2-SC expression in tumor tissue
* Eastern Cooperative Oncology Group (ECOG) performance status (PS) ≤ 1
* Thiopurine S-methyltransferase (TPMT) and NUDT15 homozygous, wild-type genotype
* TPMT\*1/TPMT\*1 and NUDT15\*1/ NUDT15\*1
* Calculated creatinine clearance ≥ 30 milliliters per minute (mL/min) per the Cockcroft and Gault formula OR serum creatinine \< 1.5 x upper limit of normal (ULN)
* Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \< 3 x ULN (\< 5 x ULN if liver metastases are present)
* Total bilirubin \< 1.5 x ULN (except subjects with Gilbert Syndrome, who can have total bilirubin up to 3.0 mg/dL)
* Albumin ≥ 2.2 mg/dL
* White blood cells (WBC) \> 2,000/mm\^3
* Hemoglobin (Hb) ≥ 9
* Neutrophils \> 1,500/mm\^3
* Platelets \> 100,000/mm\^3
* Inclusion into ≥ 1 of the following symptomatic, disease states listed below:
* Inclusion allows entry into that cohort for efficacy assessments. Patients can be in ≥ 1 cohort if they have more than one disease manifestation fitting the below criteria. If a patient fits inclusion/exclusion into one cohort and has disease manifestations that are excluded from another cohort, they can still participate in the trial but will not be assessed for efficacy fo…
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
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.