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Stem Cell Therapy
Stem Cell Collection for Familial Platelet Disorder
Phase < 1
Recruiting
Led By Chitra Hosing
Research Sponsored by M.D. Anderson Cancer Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Participants aged ≥ 18 to 75 years
Participants with a confirmed diagnosis of RUNX1 FPD, verified by a CLIA-certified genetic sequencing report
Must not have
Participants with severe splenomegaly (≥ 20 cm)
Participants with a diagnosis of MDS or hematologic malignancies
Timeline
Screening 3 weeks
Treatment Varies
Follow Up through study completion; an average of 1 year.
Awards & highlights
Summary
This trial will test if it is safe and possible to collect certain stem cells from individuals with a specific genetic condition called RUNX1-FPD.
Who is the study for?
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.
What is being tested?
The study tests the safety of using drugs Plerixafor and G-CSF (filgrastim or biosimilar) to mobilize stem cells in the bloodstream and then collecting these cells through a process called Apheresis.
What are the potential side effects?
Possible side effects include reactions at the injection site, bone pain, headache, nausea, vomiting, tiredness and potential complications related to Apheresis such as bleeding or infection.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am between 18 and 75 years old.
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My genetic test confirms I have RUNX1 FPD.
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I am mostly active and can care for myself.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
My spleen is significantly enlarged.
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I have been diagnosed with MDS or a blood cancer.
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I haven't taken any experimental drugs recently.
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I am not pregnant, breastfeeding, and I use effective birth control.
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I do not have serious mental health issues or unstable medical conditions.
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I have sickle cell disease.
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I do not have any serious or active infections.
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My heart's pumping ability is below normal.
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My kidney function is reduced, with a filtration rate below 60 mL/min.
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I have a history of blood clotting, immune system, or bone marrow disorders.
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My liver disease is in an advanced stage.
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I have had a stem cell transplant or gene therapy in the past.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ through study completion; an average of 1 year.
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~through study completion; an average of 1 year.
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Safety and adverse events (AEs)
Trial Design
1Treatment groups
Experimental Treatment
Group I: Autologous CD34+ Hematopoietic Stem Cells Mobilization and ApheresisExperimental Treatment3 Interventions
On Days 1-5, participants will receive a Granulocyte colony-stimulating factor (G-CSF), such as filgrastim, as an injection or by vein over about 5 minutes.
If the study doctor thinks it is needed, participants will also receive plerixafor as an injection under the skin on Day 5 (and 6, if you have 2 days of apheresis).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Plerixafor
2011
Completed Phase 3
~710
Apheresis
2003
Completed Phase 2
~660
Find a Location
Who is running the clinical trial?
RUNX1 FoundationUNKNOWN
M.D. Anderson Cancer CenterLead Sponsor
3,039 Previous Clinical Trials
1,799,771 Total Patients Enrolled
Chitra HosingPrincipal InvestigatorM.D. Anderson Cancer Center
3 Previous Clinical Trials
36 Total Patients Enrolled
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