Mercaptopurine for Hereditary Leiomyomatosis and Renal Cell Carcinoma

No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

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 Is on the Research Team?

BS

Brian Shuch

Principal Investigator

UCLA / Jonsson Comprehensive Cancer Center

Are You a Good Fit for This Trial?

This trial is for adults (18+) with hereditary leiomyomatosis and renal cell carcinoma (HLRCC), kidney cancer, or multiple skin or uterine tumors. Participants need good organ function, specific genetic markers, and must not be pregnant. Certain recent treatments or serious health conditions may exclude people.

Inclusion Criteria

White blood cells > 2,000/mm³
Hemoglobin ≥ 9
Neutrophils > 1,500/mm³
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Exclusion Criteria

I had another cancer that is in complete remission or has been treated and inactive for over 2 years.
Pregnant individuals; negative serum pregnancy test required for childbearing potential and consent to effective contraception until 30 days post-therapy
Absolute contraindication to contrast-enhanced imaging for efficacy assessment (not relevant for skin-only cohort)
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Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 6-MP orally once daily on days 1-28 of each cycle. Cycles repeat every 28 days for up to 26 cycles/2 years for kidney cohort or up to 13 cycles/1 year for skin or uterine cohorts.

1-2 years
Monthly visits for drug administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment completion, with follow-up within 45 days and then every 12 months for 2 years.

2 years
Annual visits

What Are the Treatments Tested in This Trial?

Interventions

  • Mercaptopurine

Trial Overview

The study tests different doses of mercaptopurine to find the safest and most effective amount for treating HLRCC-related cancers and tumors. It also uses scans, biopsies, and questionnaires to monitor effects. This is a phase I/II trial.

How Is the Trial Designed?

1

Treatment groups

Experimental Treatment

Group I: Treatment (6-MP)Experimental Treatment7 Interventions

Find a Clinic Near You

Who Is Running the Clinical Trial?

Jonsson Comprehensive Cancer Center

Lead Sponsor

Trials
373
Recruited
35,200+

The V Foundation

Collaborator

Trials
10
Recruited
320+

Phase One Foundation

Collaborator

Trials
5
Recruited
180+