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Stem Cell Transplantation

Stem Cell Transplant for Immune Deficiency Syndrome

Phase 2

Recruiting

Led By Dennis D Hickstein, M.D.

Research Sponsored by National Cancer Institute (NCI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients with evidence of MDS with specific blast percentages

Requirement for adult caregiver presence post-transplant

Must not have

Chronic active hepatitis B

Active infection refractory to antimicrobial therapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year and 2 years post-transplant

Awards & highlights

No Placebo-Only Group

Summary

This trial is for people with GATA2 deficiency to see if stem cell transplants are successful in treating the condition.

Who is the study for?

This trial is for people aged 8-70 with GATA2 deficiency, a genetic condition that can lead to leukemia. Participants need functioning kidneys, liver, heart, and lungs. They must have a matching stem cell donor and agree to use birth control. Excluded are those with active infections or malignancies, pregnant or breastfeeding women, HIV-positive individuals, and anyone allergic to the study drugs.

What is being tested?

The trial tests whether an allogeneic hematopoietic stem cell transplant (HSCT) from a matched donor can treat GATA2 mutations effectively. It involves chemotherapy or radiation before the transplant and requires hospitalization until stable post-transplant followed by regular monitoring.

What are the potential side effects?

Possible side effects include reactions to medications like Tacrolimus and Cyclophosphamide such as kidney damage or immune suppression; complications from Total Body Irradiation; graft-versus-host disease where donated cells attack the body; infection risk due to weakened immunity.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My blood disorder shows a certain level of immature cells.

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I will have an adult caregiver with me after my transplant.

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I have a GATA2 gene mutation or related health issues.

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I have GATA2 deficiency and had a serious infection.

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I am between 8 and 70 years old.

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I have a donor who is a close match to my tissue type.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have ongoing active hepatitis B.

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I have an infection that hasn't improved with treatment.

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I am HIV-positive.

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I have an active cancer in an organ other than my blood.

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I don't have a closely matched donor for a transplant.

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My cancer has spread to my brain or spinal cord.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year and 2 years post-transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year and 2 years post-transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year and 2 years post-transplant for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

To determine whether allogeneic HSCT approach results in engraftment and restores normal hematopoiesis by one year in patients with mutations GATA2.

Secondary study objectives

Overall survival, and disease-free survival.

To characterize the immune reconstitution in 10/10 matched related and unrelated donor transplant recipients and haploidentical related donor transplants who receive GVHD prophylaxis

To characterize the immune reconstitution inflammatory syndrome (IRIS)

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