Mercaptopurine for HLRCC and Kidney Cancer
This study is testing mercaptopurine to see if it's a safe and effective treatment for people with Hereditary Leiomyomatosis and Renal Cell Carcinoma (HLRCC), uterine/cutaneous leiomyomas (non-cancerous growths), and kidney cancer. HLRCC is a rare inherited condition that can lead to skin and uterine growths, and kidney cancer. Mercaptopurine works by stopping cancer cells from growing. The study aims to find the best dose of mercaptopurine and understand its side effects. It will also look at how well it treats kidney cancer and improves symptoms related to uterine growths. You may be able to join if you are 18 or older and have a specific genetic change (germline FH mutation) linked to HLRCC.
- Study design
- This is an interventional study with a planned enrollment of 18 participants. It is a phase I dose-escalation study followed by a phase II study.
- What's involved
- You would take mercaptopurine by mouth once daily. You would also undergo tumor biopsies, blood collection, CT scans, and MRI scans. Treatment could last up to 2 years for kidney cancer or up to 1 year for skin or uterine conditions.
- Compensation
- Not stated in the trial record.
- Follow-up
- The maximum tolerated dose will be measured up to cycle 2 (each cycle is 28 days).
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT07716735
Mercaptopurine for the Treatment of Hereditary Leiomyomatosis and Renal Cell Carcinoma (HLRCC), Uterine/Cutaneous Leiomyomas, and Kidney Cancer
Not Yet Recruiting
PHASE1Ages 18+InterventionalTreatmentJonsson Comprehensive Cancer CenterInvestigator-initiated
~18 participants
Updated 2026-07-21 on ClinicalTrials.gov
What's tested:Biopsy ProcedureBiospecimen CollectionComputed TomographyMagnetic Resonance ImagingMercaptopurineQuestionnaire Administration
At a glance
Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Maximum tolerated dose (MTD)
Measured over Up to cycle 2 (Cycles = 28 days)
Conditions
Where it's being run
1 sites across 1 statesCalifornia1
Study leadership
- Brian Shuch · PRINCIPAL_INVESTIGATOR · UCLA / Jonsson Comprehensive Cancer Center
Who to contact
Brian Shuch
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
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:
KIDNEY CANCER COHORT: Advanced, metastatic kidney cancer disease
KIDNEY CANCER COHORT: Measurable disease by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria
KIDNEY CANCER COHORT: ≥ 1 lines of systemic therapy (not considering adjuvant)
UTERINE FIBROIDS COHORT: Age ≥ 18
UTERINE FIBROIDS COHORT: Female biologic sex
UTERINE FIBROIDS COHORT: Any pre-menopausal patient
UTERINE FIBROIDS COHORT: Fibroid-associated symptoms including symptomatic menorrhagia and/or pelvic pain/pressure
UTERINE FIBROIDS COHORT: Estimated ≤ 15-week uterus by bimanual exam OR by radiographic parameters (≤ 10 cm max dimension of largest fibroid or estimated weight ≤ 400 g)
UTERINE FIBROIDS COHORT: Be willing to use non hormonal contraception if needed
CUTANEOUS LEIOMYOMAS COHORT: ≥ 5 cutaneous leiomyomas
CUTANEOUS LEIOMYOMAS COHORT: Leiomyoma-associated pain or paresthesia causing weekly pain ≥ 4/10 on a pain scale
Exclusion
Absolute contraindication to the use of contrast-enhanced imaging for efficacy assessment. If moderate allergy, patients could be allowed if pre-medication can be given to limit adverse reactions. (\*Not relevant for skin-only cohort)
Presence of untreated brain metastases. Treated brain metastases must be stable for 4 weeks after treatment, have no clinical symptoms, and not be on corticosteroids \> 10 mg/day of prednisone-equivalent \> 2 weeks prior to treatment. Patients with known leptomeningeal metastases are excluded
Any active or recent history of a known or suspected autoimmune disease or recent history of a syndrome that required systemic corticosteroids (\> 10 mg daily prednisone equivalent) or immunosuppressive medications within 14 days prior to first dose of study drug. An exception is allowed for syndromes which would not be expected to recur in the absence of an external trigger. Subjects with vitiligo or type I diabetes mellitus or residual hypothyroidism due to autoimmune thyroiditis only requiring hormone replacement are permitted to enroll. Inhaled steroids and adrenal replacement steroid doses \> 10 mg daily prednisone equivalents are permitted in the absence of active autoimmune disease
History of myocarditis or congestive heart failure (as defined by New York Heart Association Functional Classification III or IV), as well as unstable angina, serious uncontrolled cardiac arrhythmia, uncontrolled infection, or myocardial infarction 6 months prior to study entry
Known medical condition that, in the investigator's opinion, would increase the risk associated with study participation or interfere with the interpretation of safety results
Individuals who are pregnant; negative serum pregnancy tests in patients of childbearing potential and consent to an effective contraceptive method (if needed Centers for Disease Control and Prevention \[CDC\] guidelines provided) until a minimum of 30 days after cessation of therapy
Individuals who wish to continue actively breast-feeding must agree to not breastfeed during the study or for 180 days after the last dose of study treatment
An untreated non-renal malignancy with the following exceptions:
low risk prostate cancer on active surveillance (National Comprehensive Cancer Network \[NCCN\] very low/low risk)
non-melanoma skin cancer
Any prior treated, non-renal malignancy except for those meeting the following characteristics:
Treated stage I or II cancer from which the patient is currently in complete remission
Stage III cancer in remission for \> 2 years and is not receiving any current treatment
A hematologic malignancy from which the patient is considered to be in complete remission
UTERINE FIBROIDS COHORT: Hormonal management ≤ 2 months of starting treatment. Including gonadotrophin releasing hormone (GnRH) analog, progestins or estrogen (pills or intrauterine devices), or ulipristal acetate
UTERINE FIBROIDS COHORT: GnRH analog usage ≤ 12 months of starting treatment
UTERINE FIBROIDS COHORT: History of uterine artery embolization
UTERINE FIBROIDS COHORT: Prior radiofrequency ablation to a target lesion
UTERINE FIBROIDS COHORT: History of MR guided focused ultrasound
UTERINE FIBROIDS COHORT: Myomectomy ≤ 1 year of starting therapy
UTERINE FIBROIDS COHORT: Concern for gynecologic malignancy
UTERINE FIBROIDS COHORT: Any Federation of Gynecology and Obstetrics (FIGO) 1 or FIGO 2 myomas requiring immediate treatment
UTERINE FIBROIDS COHORT: Planning pregnancy in the next 6 months
UTERINE FIBROIDS COHORT: History of endometrial ablation
UTERINE FIBROIDS COHORT: Hormonal intrauterine device (IUD) in place
CUTANEOUS LEIOMYOMAS COHORT: Willingness/ability to have all cutaneous lesions completely removed
What this trial measures
- Maximum tolerated dose (MTD)Up to cycle 2 (Cycles = 28 days)
Will be determined using Common Terminology Criteria for Adverse Events version 5.0 grading.