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Research · Take part

HLRCC Clinical Trials and Studies

These are clinical trials and research studies that people with HLRCC or FH-deficient kidney cancer may be able to join. The list is checked against ClinicalTrials.gov, and the searches below always show the newest studies.

Reviewed by the HLRCC Foundation, Oct 2026. Trial status checked: Oct 29, 2026.

Listing a study is not a recommendation. Ask your care team whether a study fits you. Trial information changes often. Talk to your doctor before joining a trial. HLRCC Foundation does not run or endorse these studies.

What is a clinical trial?

A clinical trial is a research study with volunteers. Some test a new treatment; others follow people over time or collect samples to learn more about a condition.

  • Phase 1: a small group, to find a safe dose and learn about side effects.
  • Phase 2: more people, to see whether the treatment works for a certain cancer or condition.
  • Phase 3: a large group, to compare the new treatment with the usual treatment.
  • Not a drug trial (phase N/A): observational and research studies that do not test a treatment, such as natural history studies, registries and tissue studies.

Learn more: Learn about studies (ClinicalTrials.gov) (opens in a new tab) · How clinical trials work (NCI) (opens in a new tab)

The NIH HLRCC natural history study

The National Cancer Institute runs a long-running study of HLRCC at the NIH Clinical Center in Bethesda, Maryland (NCT00050752, NIH protocol 03-C-0066). People who have or may have HLRCC, and blood relatives of someone with HLRCC or an FH gene change, may be able to join. It is recruiting.

General contact (from the NIH protocol page): NCI Clinical Trials Referral Office, (888) 624-1937, ncimo_referrals@mail.nih.gov.

Learn about the NIH HLRCC studyNIH protocol page (opens in a new tab)ClinicalTrials.gov record NCT00050752 (opens in a new tab)

HLRCC-relevant studies now

7 studies: 6 recruiting and 1 not yet recruiting, grouped by topic. Studies that are no longer enrolling are not listed. Each card links to the full record on ClinicalTrials.gov, where the study team’s contacts are listed.

Kidney cancer (FH-deficient RCC) (2: 2 recruiting, 0 not yet)

  • RecruitingPhase 2

    Testing of Bevacizumab, Erlotinib, and Atezolizumab in Combination for Advanced-Stage Kidney Cancer

    NCT04981509 · Lead sponsor: National Cancer Institute (NCI)

    What it tests: Bevacizumab; Erlotinib; Atezolizumab; plus imaging/biopsy procedures

    Who can join

    Cohort 1 is for people with HLRCC whose kidney cancer is advanced (has grown or spread) and can be measured on scans. A second group is for people with papillary kidney cancer that is not linked to HLRCC.

    Where: United States: sites taking new people are in Atlanta, GA; Bethesda, MD (NCI Center for Cancer Research); Columbus, OH; and Houston, TX (13 US sites in total).

    • Some sites paused. Check with the study team which sites are taking new people.

    Last checked Oct 29, 2026

    View on ClinicalTrials.gov NCT04981509 (opens in a new tab)Contacts and locations for NCT04981509 (opens in a new tab)

  • RecruitingPhase 1

    [212Pb]VMT-Alpha-NET in Metastatic or Inoperable Somatostatin-Receptor Positive Gastrointestinal Neuroendocrine Tumors, Pheochromocytoma/Paragangliomas, Small Cell Lung, Renal Cell, and Head and Neck Cancers

    NCT06479811 · Lead sponsor: National Cancer Institute (NCI)

    What it tests: [212Pb]VMT-alpha-NET; [203Pb]VMT-alpha-NET; 68Ga-DOTATATE

    Who can join

    Adults with kidney cancer that has spread or cannot be removed may join if their tumor shows a certain marker (somatostatin receptor) on a scan. People with FH-deficient kidney cancer must have had at least one earlier treatment.

    Where: Bethesda, MD.

    Last checked Oct 29, 2026

    View on ClinicalTrials.gov NCT06479811 (opens in a new tab)Contacts and locations for NCT06479811 (opens in a new tab)

Skin and uterine leiomyomas (1: 0 recruiting, 1 not yet)

  • Not yet recruitingPhase 1

    Mercaptopurine for the Treatment of Hereditary Leiomyomatosis and Renal Cell Carcinoma (HLRCC), Uterine/Cutaneous Leiomyomas, and Kidney Cancer

    NCT07716735 · Lead sponsor: Jonsson Comprehensive Cancer Center

    What it tests: Mercaptopurine; plus imaging, biopsy and questionnaire procedures

    Who can join

    Adults (18+) with a disease-causing FH gene change can join if they have HLRCC signs such as skin leiomyomas, uterine fibroids or kidney cancer. People need certain blood test results and a gene test (TPMT/NUDT15) result before joining.

    Where: Los Angeles, CA.

    • Expected to open in December 2026.
    • The record lists Phase 1; its summary describes a Phase 1/2 study.

    Last checked Oct 29, 2026

    View on ClinicalTrials.gov NCT07716735 (opens in a new tab)Contacts and locations for NCT07716735 (opens in a new tab)

Natural history, registries, tissue studies (4: 4 recruiting, 0 not yet)

  • RecruitingNot a drug trial (phase N/A)

    Hereditary Leiomyomatosis Renal Cell Cancer - Study of the Genetic Cause and the Predisposition to Renal Cancer

    NCT00050752 · Lead sponsor: National Cancer Institute (NCI)

    What it involves: No study treatment (observational study)

    Who can join

    People who have or may have HLRCC can join, such as people with skin leiomyomas plus kidney cancer or uterine fibroids, or with a kidney tumor type seen in HLRCC. Blood relatives of someone with HLRCC or an FH gene change may also be able to join.

    Where: Bethesda, MD.

    Last checked Oct 29, 2026

    View on ClinicalTrials.gov NCT00050752 (opens in a new tab)Contacts and locations for NCT00050752 (opens in a new tab)

  • RecruitingNot a drug trial (phase N/A)

    Von Hippel-Lindau (VHL): Clinical Manifestations, Diagnosis, Management and Molecular Bases of Inherited Renal and Other Urologic Malignant Disorders

    NCT00001238 · Lead sponsor: National Cancer Institute (NCI)

    What it involves: No study treatment (observational study)

    Who can join

    People and their family members with a known or suspected inherited kidney or urologic cancer can join, including families with skin or uterine leiomyomas. The study description names HLRCC as one of the inherited conditions it covers.

    Where: Bethesda, MD.

    Last checked Oct 29, 2026

    View on ClinicalTrials.gov NCT00001238 (opens in a new tab)Contacts and locations for NCT00001238 (opens in a new tab)

  • RecruitingPhase N/A

    Cohort Evaluation of Body Fluids Early Detection of Cancer in High-risk Individuals

    NCT06907095 · Lead sponsor: Gustave Roussy, Cancer Campus, Grand Paris

    What it involves: biological samples

    Who can join

    Adults at high risk of cancer can join, including people aged 50 or older who carry a harmful FH gene change. The study tests body fluid samples for early cancer detection.

    Where: Villejuif, France.

    Last checked Oct 29, 2026

    View on ClinicalTrials.gov NCT06907095 (opens in a new tab)Contacts and locations for NCT06907095 (opens in a new tab)

  • RecruitingPhase N/A

    An Investigation of Kidney and Urothelial Tumor Metabolism in Patients Undergoing Surgical Resection and/or Biopsy

    NCT04623502 · Lead sponsor: University of Texas Southwestern Medical Center

    What it involves: 13C-Glucose; 13C-Acetate; 13C-Lactate; 13C-Glutamine; 13C-Fructose

    Who can join

    Adults with a kidney or urinary tract tumor who need surgery or a biopsy can join. HLRCC and FH deficiency are listed among the conditions studied.

    Where: Dallas, TX.

    Last checked Oct 29, 2026

    View on ClinicalTrials.gov NCT04623502 (opens in a new tab)Contacts and locations for NCT04623502 (opens in a new tab)

How to talk to your doctor about trials

What to bring

  • The NCT number of any study you are interested in.
  • Your genetic test report showing the FH gene change, if you have one.
  • Your pathology report, including whether the tumor was tested for FH (for example, “FH-deficient”).
  • Ask whether a referral or your scans and records need to be sent to the study team.

Questions to ask the study team

  • The study: What is being tested, and what are the possible benefits and risks?
  • Costs: What does the study pay for, and what will my insurance be billed?
  • Daily life and travel: How many visits are there, where, and is travel help available?
  • Your rights: Can I leave the study at any time, and what happens to my care if I do?

Based on NCI guidance: What to expect (NCI) (opens in a new tab) · Who pays (NCI) (opens in a new tab)

Past HLRCC trials

Show 8 past trials that are no longer open

Stay up to date

We share trial news when it happens. Follow our News.

Find an HLRCC specialistTalk to a genetic counselor (opens in a new tab)Contact us

FAQ

Are there clinical trials for HLRCC?

Yes. As of Oct 29, 2026, this page lists 7 studies that include people with HLRCC or FH-deficient kidney cancer: 6 are recruiting and 1 has not started recruiting yet. 2 are kidney cancer studies, 1 is about skin and uterine leiomyomas, and 4 are natural history, registry or tissue studies.

What is the NIH HLRCC natural history study?

It is a long-running National Cancer Institute study at the NIH Clinical Center in Bethesda, Maryland, for people who have or may have HLRCC and their relatives. It studies the condition over time; it is not a drug trial.

How do I search for HLRCC trials near me?

Use the ready-made ClinicalTrials.gov searches on this page, then use the location filter on ClinicalTrials.gov. Each study record lists its sites and how to contact the study team.

Do I need a doctor’s referral to join a trial?

It depends on the study. Some study teams take calls from patients directly, and others ask for a referral or medical records from your doctor. The study team can tell you.

Will a trial cost me anything?

Ask the study team what the study pays for and what your insurance is expected to cover, including travel. The National Cancer Institute explains who pays for what.

Learn more from trusted sources

Study details come from ClinicalTrials.gov, last checked Oct 29, 2026. See our disclaimer.

This is a staging enviroment