Allogeneic Hematopoietic Stem Cell Transplantation With Briquilimab-Based Conditioning in Partici… (NCT05907746) | Clinical Trial Compass
Active — Not RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation With Briquilimab-Based Conditioning in Participants With GATA2 Deficiency
United States13 participantsStarted 2023-11-29
Plain-language summary
Background:
People with GATA-binding factor 2 (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....
Who can participate
Age range
6 Years – 70 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
* INCLUSION CRITERIA:
* Age \>= 6 and \<= 70 years old
* Germline mutation in the GATA binding protein 2 (GATA2) gene, predicted to be deleterious or previously reported in GATA2 deficiency as determined by targeted GATA2 sequencing performed at the National Institutes of Health (NIH)
* Clinical manifestation(s) consistent with a diagnosis of GATA2 deficiency, including any of the following (Note: only one clinical manifestation is required):
* History of severe, disfiguring, and/or recurrent infections
* Low monocyte (\< 190 cells/microL), B cell (\< 61 cells/microL) and/or Natural Killer (NK) cell (\< 126 cells/microL) counts
* Myelodysplastic syndrome by World Health Organization (WHO) criteria
* "Early stage" GATA2 deficiency defined as a hypocellular for age bone marrow with less than 5% blasts and normal cytogenetics or favorable cytogenetics (defined as "good" or "very good" cytogenetics risk groups plus trisomy 8)
* Availability of an 8/8 HLA-matched related or unrelated donor, a 7/8 HLA-matched unrelated donor or a haploidentical related donor
* Lansky (for participants \< 16 years of age) or Karnofsky (for participants \>=16 years of age) performance status of \>= 40%
* Left ventricular ejection fraction \> 40%, preferably by 2-D echocardiogram (echo) obtained within 90 days prior to treatment initiation
* Participants must have adequate organ function as defined below:
* Total bilirubin \<=2.5 x upper limit of normal (ULN)
* Alanine transaminase (ALT) a…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Percentage of Evaluable Participants With GATA-binding Factor 2 (GATA2) Deficiency With Sustained Donor Engraftment by 100 Days Post-transplant Reported Along With a Two-sided 90% Confidence Interval
Timeframe: 100 days
2
Percentage of Evaluable Participants With GATA-binding Factor 2 (GATA2) Deficiency With Sustained Donor Engraftment by 100 Days Post-transplant Reported Along With a Two-sided 95% Confidence Interval
Timeframe: 100 days
Trial details
NCT IDNCT05907746
SponsorNational Institutes of Health Clinical Center (CC)