EXG34217 for Bone Marrow Failure
Trial Summary
What is the purpose of this trial?
This trial tests the safety and tolerability of EXG34217 in patients with bone marrow failure due to telomere biology disorders. The treatment involves collecting, processing, and reinfusing the patient's own blood cells to help improve their bone marrow function.
Will I have to stop taking my current medications?
The trial requires that you stop taking danazol and androgens at least 60 days before starting. Other medications are not specifically mentioned, so it's best to discuss with the trial team.
What data supports the effectiveness of the drug EXG34217 for treating bone marrow failure?
Research on similar treatments, like recombinant human granulocyte-macrophage-colony-stimulating factor (rhGM-CSF), shows that they can increase certain blood cells in patients with conditions like aplastic anemia, which is a type of bone marrow failure. This suggests that EXG34217 might also help improve blood cell counts in bone marrow failure.12345
What safety data exists for EXG34217 (or similar treatments) in humans?
How is the drug EXG34217 different from other treatments for bone marrow failure?
EXG34217 is unique because it may involve the use of colony-stimulating factors (CSFs), which are proteins that help increase the production of blood cells by stimulating bone marrow. This approach is different from traditional treatments as it focuses on enhancing the body's natural ability to produce blood cells, potentially offering a novel way to address bone marrow failure.510111213
Eligibility Criteria
This trial is for adults over 18 with mild or moderate bone marrow failure due to telomere biology disorders. It's not for those with severe bone marrow failure, certain genetic abnormalities, uncontrolled infections, previous transplants, or who can't undergo specific treatments. Pregnant or breastfeeding women and patients on recent trials or cancer treatment are also excluded.Inclusion Criteria
Exclusion Criteria
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
Peripheral blood mononuclear cells (PBMNCs) collection
Mobilization and apheresis of PBMNCs
Ex vivo cell processing
Processing of collected cells ex vivo
Processed cell infusion and post-infusion safety monitoring
Infusion of processed cells and monitoring for safety
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- EXG34217 (Unknown)
EXG34217 is already approved in United States for the following indications:
- Telomere biology disorders with bone marrow failure
- Dyskeratosis congenita