Collecting your own stem cells for gene therapy
Treatments studied
Part of Blood & lymphatic clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 18 and 25 years old.
- You need a bone marrow failure disorder that has a known genetic cause.
- Your blood counts should be within a certain range: hemoglobin above 8, white cell count above 500, and platelets above 30,000.
- You must have had a bone marrow test within the last 6 months.
- You must be able to have a central line (a special IV in a large vein) or be willing to get a temporary one for the collection procedure.
- You must be generally healthy with a good performance status (Karnofsky score > 80).
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 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 tests a drug that helps your body release stem cells so they can be collected for a future bone marrow transplant. It may help make the transplant process safer and more effective for people with sickle cell disease.
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.
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.
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.
Hear when a new Bone Marrow Failure Syndrome trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.