Clinical trials: GATA2
Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder
Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)
This is a research protocol that will examine Hematopoietic Stem Cell Transplantation (HSCT) using a reduced conditioning regimen (RIC) with total body Irradiation (TBI) in those diagnosed with Common Variable Immunodeficiency…
- Conditions
- Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder, Immune Dysregulation, DiGeorge Syndrome
- Trial name
- CVID/PIRD (NCT07284641)
- Testing
- Hematopoietic stem cell transplant (HSCT)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Paul Szabolcs
Severe Congenital Neutropenia, GATA2 Deficiency
Total Lymphoid Irradiation Pre-HSCT in Severe Congenital Neutropenia
Severe congenital neutropenia (SCN) is a group of primary immunodeficiencies caused by distinct gene mutations and characterized by neutrophil maturation impairment, which leads to neutropenia, predisposition to severe bacterial…
- Trial name
- NCT04844177
- Testing
- conditioning with TLI
- Where
- Russia
- Volunteers
- 10 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Neutropenia
A Study of Mavorixafor in Participants With Congenital Neutropenia and Chronic Idiopathic Neutropenia Disorders
This is a 2-part study of mavorixafor in participants diagnosed with chronic neutropenia. The main goal of Part 1 (Phase 1b) is to help researchers learn more about how the investigational medicine, mavorixafor, impacts people…
- Trial name
- NCT04154488
- Testing
- Mavorixafor
- Where
- 7 sites in United States
- Volunteers
- 32 people
- Sponsor
- X4 Pharmaceuticals
GATA2, Immunodeficiency
Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations
Background: GATA2 deficiency is a disease caused by mutations in the GATA2 gene. It can cause different types of leukemia and other diseases. Researchers want to see if a stem cell transplant can be used to treat this condition.
- Conditions
- GATA2, Immunodeficiency, MDS
- Trial name
- NCT01861106
- Testing
- Allogeneic HSCT, Busulfan Test dose, Fludarabine (Fludara, Berlex Laboratories)
- Where
- United States
- Volunteers
- 144 people
- Sponsor
- National Cancer Institute (NCI)
Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations
Reduced-Intensity Conditioning Before Donor Stem Cell Transplant in With High-Risk Hematologic Malignancies
This phase II trial studies reduced-intensity conditioning before donor stem cell transplant in treating patients with high-risk hematologic malignancies.
- Conditions
- Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, Acute Myeloid Leukemia With Inv(3) (q21.3;q26.2); GATA2, MECOM, Acute Myeloid Leukemia With t(6;9) (p23;q34.1); DEK-NUP214
- Trial name
- NCT01760655
- Testing
- Fludarabine phosphate, Busulfan, Total-Body Irradiation
- Where
- United States
- Volunteers
- 62 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
DOK 8, STAT1
Genetic Analysis of Immune Disorders
The purposes of this study are to 1) identify the genes responsible for certain immune disorders, 2) learn about the medical problems they cause, and 3) learn how to predict who is likely to develop these disorders and what the…
- Conditions
- DOK 8, STAT1, GATA2, Immunodeficiency
- Trial name
- NCT00001467
- Where
- United States
- Volunteers
- 5,000 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
MDS, Immunodeficiency
Pilot and Feasibility Study of Reduced-Intensity Hematopoietic Stem Cell Transplant for MonoMAC
Background: Stem cells are immature blood cells that grow in the bone marrow and produce all of the cells needed for normal blood and immunity.
- Conditions
- MDS, Immunodeficiency, GATA2
- Trial name
- NCT00923364
- Testing
- Cyclophosphamide (CTX, Cytoxan), Fludarabine(Fludara,Berlex Laboratories), Total Body Irradiation (TBI)
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- National Cancer Institute (NCI)
GATA2 Gene Mutation
Study for Characterisation of Predictive Parameters of Clonal Evolution in Subjects With GATA2 Germline Mutation
This trial is a translational, open-label, multi-site, retrospective and prospective cohort study of 250 patients aiming to characterize predictive parameters of clonal evolution in a population of subjects carrying the germline…
- Trial name
- GATA-2 (NCT05983991)
- Testing
- Biological samples (blood and bone marrow samples).
- Where
- 24 sites in France
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Institut Claudius Regaud
Primary Immunodeficiencies, APECED
Participation in a Research Registry for Immune Disorders
Background: People with primary immune deficiency diseases (PIDD) have weak immune systems. This makes it hard for their bodies to fight infection.
- Conditions
- Primary Immunodeficiencies, APECED, CGD, Wiskott-Aldrich Syndrome
- Trial name
- NCT01953016
- Where
- United States
- Volunteers
- 716 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Acute Leukemia, Adenomatous Polyposis
Familial Investigations of Childhood Cancer Predisposition
NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll.
- Conditions
- Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML
- Trial name
- SJFAMILY (NCT03050268)
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- St. Jude Children's Research Hospital
GATA2 Deficiency
Natural History Study of GATA2 Deficiency and Related Disorders
Background: GATA2 deficiency is a genetic disorder that can cause problems with a person s immune system and other body systems. Some people who have this disorder develop few problems from it.
- Trial name
- NCT01905826
- Where
- United States
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Endocrine Disease, Infertility
Evaluation of Women With Endocrine and Reproductive-Related Conditions
This study was designed to allow inpatient and outpatient evaluation of women with a variety of reproductive and endocrine-related disorders for purposes of research and physician education.
- Conditions
- Endocrine Disease, Infertility, Leiomyoma, Endometriosis
- Trial name
- NCT00001850
- Where
- United States
- Volunteers
- 833 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia
Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)
This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with…
- Conditions
- Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Hereditary Sideroblastic Anemia
- Trial name
- NCT04965597
- Testing
- Treosulfan, Fludarabine Phosphate, Tacrolimus
- Where
- 24 sites in United States
- Volunteers
- 40 people
- Sponsor
- Fred Hutchinson Cancer Center
GATA2, Immunodeficiency
Allogeneic Hematopoietic Stem Cell Transplantation With Briquilimab-Based Conditioning in Participants With GATA2 Deficiency
Background: People with GATA-binding factor 2 (GATA2) deficiency have a mutation on the GATA2 gene. This gene affects immune function.
- Trial name
- NCT05907746
- Testing
- Mycophenolate Mofetil, Tacrolimus, Post-Transplant Cyclophosphamide
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- National Cancer Institute (NCI)
Acute Biphenotypic Leukemia, Acute Leukemia
Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults With Blood Cancers Undergoing Donor Stem Cell Transplant
This phase II trial studies how well naive T-cell depletion works in preventing chronic graft-versus-host disease in children and young adults with blood cancers undergoing donor stem cell transplant.
- Conditions
- Acute Biphenotypic Leukemia, Acute Leukemia, Acute Leukemia of Ambiguous Lineage, Acute Lymphoblastic Leukemia
- Trial name
- NCT03779854
- Testing
- Total-Body Irradiation, Thiotepa, Fludarabine
- Where
- 9 sites in United States
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Fred Hutchinson Cancer Center