Gene Therapy Stem Cell Collection for Blood Disorders
Treatments studied
Part of Blood & lymphatic clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a genetic bone marrow failure syndrome (a genetic condition affecting blood cell production) and are willing to donate your own stem cells for research
- You are between 18 and 25 years old
- Your blood counts meet minimum levels: hemoglobin above 8 g/dL, white blood cells above 500/mm³, and platelets above 30,000/mm³
- You had a bone marrow biopsy within the last 6 months
- You can undergo the cell collection procedure either with an existing central line, a temporary apheresis catheter, or without a catheter if medically appropriate
- You are in good overall health (Karnofsky score above 80, meaning you can carry out most normal activities) and have negative tests for syphilis, hepatitis B and C, HIV, and HTLV
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 new approach for people with non-cancerous blood and immune system diseases. It uses your own engineered immune cells (CAR-T cells) to prepare your body for a stem cell transplant from a relative.
This trial tests whether a stem cell transplant from a partially gene-matched family member can cure severe sickle cell disease in children and young adults. The transplant uses a new approach that may work even when a perfectly matched sibling isn't available, and it might help patients whose sickle cell is causing serious complications like strokes, organ damage, or frequent pain crises.
This trial tests a high-dose medicine to collect stem cells from young adults with sickle cell disease. It aims to prepare for a transplant that could potentially cure the condition.
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.
This trial studies whether an apheresis procedure (removing blood cells through a machine) can help people with specific inherited immune system conditions. Participants will also receive filgrastim to help move certain blood-forming cells for collection.
This Phase 2 study tests a specific chemotherapy combination (treosulfan, fludarabine, and thiotepa) before an unrelated or matched donor stem cell transplant. It may help people with certain non-cancer blood and immune disorders by trying to reset the immune system using donor stem cells.
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