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NCT05907746 · ClinicalTrials.gov registry record · Phase 2
Allogeneic Hematopoietic Stem Cell Transplantation With Briquilimab-Based Conditioning in Participants With GATA2 Deficiency
A Phase 2 study of Immunodeficiency and GATA2, sponsored by National Cancer Institute (NCI).
- Active
- Registry status
- Phase 2
- Development phase
- 7
- Enrollment target
- 1
- Study location
NCT05907746 is a Phase 2 study of Immunodeficiency and GATA2 that is active but no longer recruiting, run by National Cancer Institute (NCI). The registered enrollment target is 7 participants, below the 1,063-participant average among 5 other Immunodeficiency trials with a reported enrollment target (99% lower). The trial reports 1 study location across 1 state.
The verdict
NCT05907746, a Phase 2 study of Immunodeficiency and GATA2, is active but no longer recruiting, sponsored by National Cancer Institute (NCI).
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 7 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: People with GATA2 deficiency have a mutation on the GATA2 gene. This gene affects immune function. People with this disease are prone to serious infections; in time, they may develop blood cancers. A hematopoietic stem cell (HSC) transplant can cure GATA2 deficiency, but using stem cells donated by other people can cause serious side effects. Objective: To test a new drug (Briquilimab) to see if it can make HSC transplants safer. Eligibility: People aged 6 to 70 years who have GATA2 deficiency. Design: Participants will be screened. They will have a physical exam, with blood and urine tests. They will have tests of their heart and lung function. They may have a bone marrow biopsy: Their hip will be numbed; a large needle will be inserted to draw out tissue from inside the pelvis. Participants will have a central venous catheter placed in a vein of the neck or chest. This will be used to draw blood and administer drugs. Briquilimab will be given through the catheter about 11 days before the transplant. This is part of conditioning: preparing the body to receive the new stem cells. Conditioning also includes other medications and total body irradiation. Donor stem cells will be administered through the catheter. Participants will receive other approved drugs to help prevent side effects. Participants will stay in the hospital from the beginning of the conditioning until several weeks after the transplant. They will remain in the local area for 100 days after discharge; they will come to the clinic at least once a week during this time. Follow-up visits will continue for 3 years....
Conditions Studied
Interventions
- DRUG Mycophenolate Mofetil
- DRUG Tacrolimus
- RADIATION Total Body Irradiation
- PROCEDURE Hematopoietic Cell Transplant
- DRUG Post-Transplant Cyclophosphamide
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 7 participants |
| Start Date | 2023-11-29 |
| Est. Completion | 2028-07-31 |
| Phase | Phase 2 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05907746
The ClinicalTrials.gov registry entry for NCT05907746 describes a study currently listed as active not recruiting, categorized as Phase 2. The registered enrollment target is 7 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 1,063-participant average among 5 other Immunodeficiency trials with a reported enrollment target (99% lower). The listed sponsor is National Cancer Institute (NCI), which has 3,257 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with Immunodeficiency appearing as the primary indexed condition, and to 5 interventions - of which Mycophenolate Mofetil is the first listed.
NCT05907746 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.
Frequently Asked Questions
What is clinical trial NCT05907746 about?
NCT05907746 is a clinical study titled "Allogeneic Hematopoietic Stem Cell Transplantation With Briquilimab-Based Conditioning in Participants With GATA2 Deficiency". Background: People with GATA2 deficiency have a mutation on the GATA2 gene. This gene affects immune function. People with this disease are prone to serious infections; in time, they may develop blood cancers. A hematopoietic stem cell (HSC) transplant can cure GATA2 deficiency, but using stem cell...
What is the current status of trial NCT05907746?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 7 participants. The study started on 2023-11-29. Estimated completion is 2028-07-31.
What conditions does trial NCT05907746 study?
This clinical trial studies the following conditions: Immunodeficiency, GATA2.
What interventions are being tested in trial NCT05907746?
The interventions under investigation include: Mycophenolate Mofetil (DRUG), Tacrolimus (DRUG), Total Body Irradiation (RADIATION), Hematopoietic Cell Transplant (PROCEDURE), Post-Transplant Cyclophosphamide (DRUG).
Who is sponsoring clinical trial NCT05907746?
This trial is sponsored by National Cancer Institute (NCI), which has 3,257 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05907746 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Read our methodology - how this data is sourced, computed, and verified.
Related
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