Stem Cell Collection for Familial Platelet Disorder
What You Need to Know Before You Apply
What is the purpose of this trial?
To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.
Will I have to stop taking my current medications?
The trial protocol does not specify whether you need to stop taking your current medications. However, if you have been treated with an investigational drug within 30 days of screening, you may not be eligible to participate.
Is stem cell mobilization safe for humans?
Stem cell mobilization using drugs like G-CSF and plerixafor has been generally well-tolerated in humans, with side effects similar to those of the individual drugs. Studies in normal volunteers and patients with various conditions have shown that these treatments are safe, although some patients may not respond well due to previous treatments or other factors.12345
How is the treatment for Familial Platelet Disorder using Autologous CD34+ Hematopoietic Stem Cells Mobilization different from other treatments?
This treatment is unique because it involves mobilizing the patient's own stem cells (autologous) using a combination of chemotherapy and growth factors like granulocyte colony-stimulating factor (G-CSF), which increases the number of stem cells available for collection. This approach is different from traditional methods that may rely solely on bone marrow or do not use the combination of chemotherapy and growth factors to enhance stem cell yield.56789
What data supports the effectiveness of the treatment Autologous CD34+ Hematopoietic Stem Cells Mobilization for Familial Platelet Disorder?
Research shows that using growth factors like G-CSF and drugs like plerixafor can significantly increase the number of stem cells collected, which is crucial for successful transplantation. This approach has been effective in patients with blood-related conditions, suggesting it may also be beneficial for Familial Platelet Disorder.710111213
Who Is on the Research Team?
Chitra Hosing
Principal Investigator
M.D. Anderson Cancer Center
Are You a Good Fit for This Trial?
This trial is for individuals with RUNX1 Familial Platelet Disorder, which affects blood clotting. Participants must be eligible to undergo procedures to collect stem cells from their own blood.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Mobilization and Apheresis
Participants receive G-CSF injections on Days 1-5, with optional plerixafor on Day 5 or 6 for stem cell mobilization and collection
Follow-up
Participants are monitored for safety and effectiveness after stem cell collection
What Are the Treatments Tested in This Trial?
Interventions
- Autologous CD34+ Hematopoietic Stem Cells Mobilization
Autologous CD34+ Hematopoietic Stem Cells Mobilization is already approved in United States, European Union for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
M.D. Anderson Cancer Center
Lead Sponsor
RUNX1 Foundation
Collaborator