Collecting stem cells safely in RUNX1 platelet disorder
Treatments studied
Part of Blood & lymphatic, Genetic & congenital 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 a low dose of sirolimus, a medicine that may help your bone marrow make more blood cells, especially platelets. It is for people with a specific genetic change (RUNX1) that affects blood cell production.
This study is for people with a specific genetic change (RUNX1 mutation) that increases the risk of blood cancers. It tests whether a stem cell transplant from a half-matched family member can help treat or cure these blood conditions.
This trial tests whether a low dose of sirolimus, a drug that calms the immune system, can help improve blood counts and reduce bleeding risk in people with a genetic condition called RUNX1 familial platelet disorder. If it works, it might lower the chance of developing serious blood problems later.
This study collects your own stem cells (the cells that make blood) so they can be used for future gene therapy. The stem cells are collected through a process called apheresis, after you receive two medications that move stem cells from your bone marrow into your blood.
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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