Collecting stem cells safely in RUNX1 platelet disorder
Treatments studied
Part of Blood & lymphatic clinical trials.
This study tests the safety of collecting your own blood stem cells (called CD34+ cells) using a process called apheresis. It is for people with RUNX1 familial platelet disorder who may need a stem cell transplant in the future.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 18 and 75 years old (or 12 and older after safety review).
- You need a genetic test confirming RUNX1 familial platelet disorder.
- Your platelet count must be at least 50,000 before the procedure (or you can get a platelet transfusion).
- You cannot have active blood cancer, a prior stem cell transplant, or serious uncontrolled bleeding.
- You must be generally healthy enough for stem cell collection and have no major organ problems.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This trial tests whether a stem cell transplant from a half-matched donor can treat blood cancers in people with a RUNX1 gene mutation. The goal is to replace the faulty bone marrow with healthy cells that can fight the cancer.
This trial collects your own blood-forming stem cells (special cells that make blood) to advance gene therapy treatments for genetic bone marrow failure syndromes. Researchers will use growth factors to mobilize these cells into your bloodstream, then collect them through a process called apheresis—similar to donating blood plasma.
This study follows people who have a known or suspected RUNX1 gene variant linked to FPDMM-like features. It may help improve understanding of the gene, and participants may contribute samples that researchers use for genetic testing and research.
This Phase 3 study tests using filgrastim (a medicine that helps stem cells leave the bone marrow) to collect stem cells from unrelated donors for transplant. It focuses on donor safety and whether this collection method works well enough for transplantation.
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This Phase 2 study tests a specific type of donor stem cell transplant for severe bone marrow failure conditions, aiming to improve cure rates while reducing transplant risks. It includes people with diseases like severe aplastic anemia and certain bone marrow disorders, especially when standard transplant plans feel too risky.
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