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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year and 2 years post-transplant
Awards & highlights

Study 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.Check my eligibility
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.See study design
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.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
To determine whether allogeneic HSCT approach results in engraftment and restores normal hematopoiesis by one year in patients with mutations GATA2.
Secondary outcome measures
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
13,688 Previous Clinical Trials
40,930,262 Total Patients Enrolled
Dennis D Hickstein, M.D.Principal InvestigatorNational Cancer Institute (NCI)
5 Previous Clinical Trials
128 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)

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Will this research be enrolling any participants who are over 50 years old?

"The age requirements for this trial state that the youngest a participant can be is 8 years old, while the oldest a patient can be and still participate is 70."

Answered by AI

Are there any unfilled vacancies for participants in this research project?

"Yes, this trial is still open to recruitment and information on clinicaltrials.gov supports this claim. The listing was originally posted on July 24th, 2013 with the most recent update being made on October 31st, 2020."

Answered by AI

How can I become a subject in this clinical trial?

"This clinical trial is looking for 144 participants, within the ages of 8 and 70 who have immunodeficiencies. It is required that patients also meet the following criteria:-Creatinine: Adult patients: less than or equal to 2.0 mg/dl and creatinine clearance greater than or equal to 30 ml/min; Pediatric patients (<18 years old): creatinine <1.5 mg/dL and a creatinine clearance , using the Schwartz Formula, > 30 mL/min/1.73m(2).-INCLUSION CRITERIA- Recipient,"

Answered by AI

Are the risks of Arm B outweighed by its benefits?

"Arm B's safety is rated a 2 because there is only evidence of its safety and no efficacy data."

Answered by AI

What is the standard care that Arm B is used to compare against in this study?

"While Arm B is the most popular treatment for leukemia, it can also be used to effectively manage osteoporosis caused by glucocorticoid, lung cancers, and dermatitis, atopic."

Answered by AI

How many people are included in the data set for this research?

"This is accurate. The website provides evidence that this study, which was originally posted on July 24th 2013 and last updated on October 31st 2020, is currently looking for 144 participants from 2 locations."

Answered by AI

Is Arm B a new experimental treatment?

"At the moment, there are 1077 ongoing studies that focus on Arm B. Out of those 189 trials are currently in Phase 3. The majority of clinical trials for Arm B are located in Philadelphia, however, there 29790 different locations running studies for Arm B."

Answered by AI
~7 spots leftby Dec 2024