Hereditary thrombocytopenia and hematological cancer predisposition syndrome associated with RUNX1
MONDO:0100083This is an autosomal dominant disorder caused by mutations in the RUNX1 gene and is characterized by mild to moderate thrombocytopenia, platelet functional and/or ultrastructural defects and a predisposition to hematologic malignancies, most often AML and MDS, and less frequently T-ALL.
Also known as: FPD/AML syndrome, FPS/AML syndrome, Familial Platelet Disorder with Associated Myeloid Malignancy, familial platelet disorder with associated myeloid malignancy, familial platelet syndrome with predisposition to acute myelogenous leukaemia, familial platelet syndrome with predisposition to acute myelogenous leukemia, hereditary thrombocytopenia and hematological cancer predisposition syndrome associated with RUNX1, FPDMM
41 clinical trials for this condition and its sub-types.
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Could a cancer drug fix a bleeding disorder and prevent leukemia?
Disease control Recruiting nowThis early-stage trial tests imatinib, a drug already used for certain cancers, in adults with a harmful RUNX1 gene mutation. The mutation causes easy bleeding and a high risk of blood cancers. The study aims to find the best dose and see if imatinib can improve platelet function…
Phase: PHASE1 • Sponsor: National Cancer Institute (NCI) • Aim: Disease control
Last updated Aug 09, 2026 00:00 UTC
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Simple blood test aims to catch cancers early in genetically prone patients
Diagnosis Recruiting nowThis study is testing a blood test called GRAIL Galleri that looks for signs of many different cancers at once. It is being offered to 1000 people who have a high risk of cancer due to inherited genetic conditions. The goal is to see if the test can find cancers early, when they …
Phase: NA • Sponsor: Dana-Farber Cancer Institute • Aim: Diagnosis
Last updated Jun 27, 2026 08:12 UTC
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Scientists track rare gene variant to unlock blood disorder mysteries
Knowledge-focused Recruiting nowThis study follows people of all ages who have or may have a RUNX1 gene variant, which can cause bleeding problems and increase the risk of blood cancers. Researchers will collect medical history, blood samples, and bone marrow over many years to understand how the condition deve…
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Aug 18, 2026 05:00 UTC
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Scientists bank tumors and blood to unlock cancer secrets
Knowledge-focused Recruiting nowThis study collects blood, urine, saliva, and tissue samples from nearly 6,000 people who have or may have cancer, including rare inherited forms. The samples are stored and used for research to understand the genetic and molecular basis of cancer. No new treatment is being teste…
Sponsor: National Cancer Institute (NCI) • Aim: Knowledge-focused
Last updated Aug 18, 2026 05:00 UTC
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Scientists launch massive tumor repository to unlock cancer secrets
Knowledge-focused Recruiting nowThis study collects extra blood, urine, and tumor samples from children and adults with certain cancers to build a large tissue repository. Researchers will analyze these samples to learn more about the genetic and molecular makeup of these tumors. The goal is to support future s…
Sponsor: National Cancer Institute (NCI) • Aim: Knowledge-focused
Last updated Aug 06, 2026 00:00 UTC
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Scientists hunt for hidden genes behind hereditary cancers
Knowledge-focused Recruiting nowThis study collects tissue samples, medical data, and family histories from over 1,100 people being screened for colorectal or endometrial cancer. Researchers hope to discover new genes and mechanisms that increase cancer risk, especially in hereditary cancer syndromes. The goal …
Sponsor: Weill Medical College of Cornell University • Aim: Knowledge-focused
Last updated Aug 05, 2026 00:00 UTC
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Hunt for hidden cancer genes: families needed to unlock hereditary secrets
Knowledge-focused Recruiting nowThis study aims to discover new genes that may cause certain cancers to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had cancer, especially childhood cancers. The goal is to build a regist…
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC
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Can young adults at genetic cancer risk keep up with checkups after pediatric care ends?
Knowledge-focused Recruiting nowThis study follows 56 young adults who have a genetic condition that raises their cancer risk. After they graduate from pediatric care at St. Jude, researchers track whether they find adult doctors and continue regular cancer screenings over 8 years. Participants fill out surveys…
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC
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Can online tools boost genetic testing in families with hereditary cancer?
Knowledge-focused Recruiting nowThis study tests whether web-based tools can help people with hereditary cancer syndromes share risk information with their relatives and encourage them to get genetic testing. Researchers will enroll 625 participants (patients and their relatives) and track how many use the tool…
Phase: NA • Sponsor: University of Michigan Rogel Cancer Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC
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First step toward a cure? stem cell harvesting trial launches for rare platelet disorder
Knowledge-focused Recruiting nowThis phase 1 trial at M.D. Anderson Cancer Center is testing whether it is safe to collect stem cells from people with RUNX1 familial platelet disorder, a rare inherited condition that raises the risk of bleeding and leukemia. Four participants will receive drugs to mobilize stem…
Phase: PHASE1 • Sponsor: M.D. Anderson Cancer Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:06 UTC
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Massive study aims to decode cancer genes for better family counseling
Knowledge-focused Recruiting nowThis study looks at genetic changes in families to figure out which ones truly increase cancer risk. Researchers will analyze DNA from 11,000 people, including those with certain gene variants and their relatives. The goal is to improve genetic counseling and help doctors decide …
Phase: NA • Sponsor: Institut Curie • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:37 UTC
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Can better conversations prevent cancer? new study investigates
Knowledge-focused Recruiting nowThis study from St. Jude Children's Research Hospital looks at how parents and children (ages 10-24) talk about the child's genetic risk for cancer. The goal is to understand what helps or hinders these conversations, so doctors can create better education programs. The study wil…
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:23 UTC
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New study uses DNA tests to catch blood cancers before they start
Knowledge-focused Recruiting nowThis study is looking at 2,000 people who have early warning signs of blood cancers or bone marrow failure. Researchers use a special genetic test (next generation sequencing) to find changes in cancer-related genes. The goal is to better understand who is at risk and to catch th…
Phase: NA • Sponsor: Mayo Clinic • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:07 UTC
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Community clinics test smarter ways to spot cancer genes
Knowledge-focused Recruiting nowThis study compares two methods of offering cancer genetic testing to patients in community health centers. One method lets primary care doctors order tests directly, while the other uses a specialist model with extra support. Researchers will track how many patients get tested a…
Phase: NA • Sponsor: University of Illinois at Chicago • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:11 UTC
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Can a website replace the genetic counselor? new study tests online cancer risk testing
Knowledge-focused Recruiting nowThis study compares a self-guided online genetic testing program to standard in-person counseling for people at risk of hereditary cancer. About 1,000 adults who meet national guidelines for genetic testing will participate. The goal is to see if the online option works just as w…
Phase: NA • Sponsor: Abramson Cancer Center at Penn Medicine • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:11 UTC
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Can doctors help families unlock Life-Saving genetic secrets?
Knowledge-focused Recruiting nowThis study looks at a new way to help family members of cancer patients learn if they have inherited a higher risk for cancer. About 240 people with a known cancer gene change will be asked to share their test results with their close relatives, either on their own or with help f…
Phase: NA • Sponsor: City of Hope Medical Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:09 UTC
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Scientists hunt for hidden cancer genes in 1,500 patients
Knowledge-focused Recruiting nowThis study aims to discover new genes that increase the risk of developing cancer, especially in people with a strong family history or unusual cancer patterns. Researchers will analyze genetic data from 1,500 adults and children who have or may develop inherited cancer. The goal…
Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:04 UTC
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New study tracks kids prone to cancer to uncover early warning signs
Knowledge-focused Recruiting nowThis study follows children under 21 who have a genetic condition that raises their risk of developing cancer, along with their relatives. Researchers will collect health information and samples over time to better understand how these cancers develop and how to catch them early.…
Sponsor: Emory University • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC
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Scientists dig into genetic roots of rare bleeding disorders
Knowledge-focused Recruiting nowThis study looks at how blood cells interact and what goes wrong in inherited bleeding disorders like Glanzmann thrombasthenia. Researchers will analyze blood samples from up to 60 healthy volunteers and patients to find genetic defects. The goal is to better understand these con…
Sponsor: Rockefeller University • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC