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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

No Placebo-Only Group

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 below

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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:

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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.

This trial's timeline: 3 weeks for screening, Varies for treatment, and through study completion; an average of 1 year. for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Safety and adverse events (AEs)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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