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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

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 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)
+3 more

Trial Design

5Treatment groups
Active Control
Group I: Arm AActive Control6 Interventions
10/10 HLA Matched Related Donor or Unrelated Donor or 9/10 HLA with DQ mismatch Transplant
Group II: Arm BActive Control7 Interventions
9/10 or 8/10 HLA Match Related Donor or Unrelated Donor or Haploidentical Donor Transplant
Group III: Arm C (combined with Arm B per Amendment N)Active Control7 Interventions
Haploidentical Related Donor Transplant
Group IV: Arm D (Deleted this arm per amendment I)Active Control5 Interventions
Umbilical Cord Blood Transplant
Group V: Arm E (Deleted this arm per amendment O)Active Control1 Intervention
Donor

Find a Location

Who is running the clinical trial?

National Cancer Institute (NCI)Lead Sponsor
13,842 Previous Clinical Trials
41,002,856 Total Patients Enrolled
Dennis D Hickstein, M.D.Principal InvestigatorNational Cancer Institute (NCI)
5 Previous Clinical Trials
145 Total Patients Enrolled

Media Library

Allogeneic HSCT (Stem Cell Transplantation) Clinical Trial Eligibility Overview. Trial Name: NCT01861106 — Phase 2
Myelodysplastic Syndrome Clinical Trial 2023: Allogeneic HSCT Highlights & Side Effects. Trial Name: NCT01861106 — Phase 2
Allogeneic HSCT (Stem Cell Transplantation) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01861106 — Phase 2
Myelodysplastic Syndrome Research Study Groups: Arm A, Arm B, Arm C (combined with Arm B per Amendment N), Arm D (Deleted this arm per amendment I), Arm E (Deleted this arm per amendment O)
~3 spots leftby Dec 2024
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