Clinical trials: GATA2
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)
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/Myelodysplastic Syndrome, Multiple Myeloma (MM)
A Dose Escalation Phase I Study to Assess the Safety and Clinical Activity of Multiple Cancer Indications
THINK (THerapeutic Immunotherapy with NKR-2) is a multinational (EU/US) open-label Phase I study to assess the safety and clinical activity of multiple administrations of autologous NKR-2 cells in seven refractory cancers…
- Trial name
- THINK (NCT03018405)
- Testing
- NKR-2 cells
- Where
- 5 sites in United States, Belgium
- Volunteers
- 146 people
- Sponsor
- Celyad Oncology SA
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 Myeloid Leukemia, Chronic Myelomonocytic Leukemia
A Study to Evaluate S241656 Alone or in Combination in Participants With Selected Myeloid Malignancies
The objective of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of S241656 and to determine the recommended dose for expansion (RDE) of S241656 in participants with relapsed/refractory (R/R) acute…
- Conditions
- Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome Acute Myeloid Leukemia
- Trial name
- NCT07719348
- Testing
- S241656, Posaconazole
- Volunteers
- 64 people
- Sponsor
- Servier
Acute Myeloid Leukemia and Myelodysplastic Syndrome
A Study of QLF32101 in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndrome
This study aimed to evaluate the safety,tolerability and preliminary efficacy of QLF32101 administered intravenously and subcutaneously in patients with R/R, AML.
- Trial name
- NCT05703204
- Testing
- QLF32101
- Where
- China
- Volunteers
- 122 people
- Sponsor
- Qilu Pharmaceutical Co., Ltd.
Lymphedema Arm
Integrating Clinical, Protein Expression, and Genetic Markers to Predict Lymphedema in Breast Cancer
The goal of this observational ambidirectional cohort study is to develop a prediction model to identify the risk of lymphedema after axillary lymph node dissection (ALND) in patients with breast cancer.
- Trial name
- NCT07252843
- Where
- Indonesia
- Volunteers
- 133 people
- Sponsor
- Dharmais National Cancer Center Hospital
LAD-1, DOCK8
Apheresis of Patients With Immunodeficiency
Background: Gene therapy is being investigated as a possible treatment for individuals with immunodeficiency diseases or other conditions that make it difficult to fight off infection.
- Conditions
- LAD-1, DOCK8, GATA2 Deficancy
- Trial name
- NCT01212055
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- National Cancer Institute (NCI)
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
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)
Acute Myeloid Leukemia, Myelodysplastic Syndrome
A Phase I, Open-Label, Dose Escalation and Cohort Expansion Study of BS HH 002.SA in Patients With AML and MDS
Indication:Relapsed or refractory AML in patients for whom no established treatment options are available (this indication will heretofore be referred to as the protocol AML indication), or adult patients with MDS who are…
- Trial name
- NCT04743115
- Testing
- BS HH 002.SA
- Volunteers
- 66 people
- Sponsor
- Shanghai Bensen Pharmaceutical Co., Ltd.
Chronic Myelomonocytic Leukemia, Chronic Myelomonocytic Leukemia-1
Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia
This randomized phase II/III trial studies how well azacitidine works with or without lenalidomide or vorinostat in treating patients with higher-risk myelodysplastic syndromes or chronic myelomonocytic leukemia.
- Conditions
- Chronic Myelomonocytic Leukemia, Chronic Myelomonocytic Leukemia-1, Chronic Myelomonocytic Leukemia-2, Myelodysplastic Syndrome
- Trial name
- NCT01522976
- Testing
- Azacitidine, Laboratory Biomarker Analysis, Lenalidomide
- Where
- 294 sites in United States, Canada
- Volunteers
- 282 people
- Sponsor
- National Cancer Institute (NCI)
Acute Myeloid Leukemia Refractory, Acute Myeloid Leukemia, in Relapse
AB8939 in Patients With Relapsed/Refractory Acute Myeloid Leukemia
The primary objective is to define the safety and tolerability of AB8939 in patients with AML by determining the dose-limiting toxicities, the maximum tolerated dose, and the recommended dose for dose expansion study.
- Conditions
- Acute Myeloid Leukemia Refractory, Acute Myeloid Leukemia, in Relapse, Myelodysplastic Syndrome Acute Myeloid Leukemia
- Trial name
- NCT05211570
- Testing
- AB8939, Venetoclax
- Where
- 10 sites in United States, France and 2 more
- Volunteers
- 78 people
- Sponsor
- AB Science
Acute Myeloid Leukemia Refractory, Myelodysplastic Syndrome Acute Myeloid Leukemia
A Study to Investigate APL-4098 Alone and in Combination in Adults With AML or MDS
This is an open-label, Phase 1 study to determine the safety, tolerability, and efficacy of APL-4098 alone, and in combination with azacitidine, and in combination with azacitidine plus venetoclax for the treatment of acute…
- Conditions
- Acute Myeloid Leukemia Refractory, Myelodysplastic Syndrome Acute Myeloid Leukemia, Myelodysplastic Syndrome With Excess Blasts, Acute Myeloid Leukemia, in Relapse
- Trial name
- NCT06372717
- Testing
- APL-4098, Azacitidine and APL-4098, Azacitidine and Venetoclax and APL-4098
- Where
- 9 sites in Australia, United Kingdom
- Volunteers
- 100 people
- Sponsor
- Apollo Therapeutics Ltd
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
Acute Myeloid Leukemia, Myelodysplastic Syndrome/Acute Myeloid Leukemia
Iadademstat in Combination With Azacitidine and Venetoclax in Treating Newly Diagnosed Acute Myeloid Leukemia
This phase I trial tests the safety, side effects, and best dose of iadademstat when given together with azacitidine and venetoclax in treating patients with newly diagnosed acute myeloid leukemia (AML).
- Trial name
- NCT06357182
- Testing
- Azacitidine, Biospecimen Collection, Bone Marrow Biopsy
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- OHSU Knight Cancer Institute
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
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
Nivolumab for High-Risk MDS/AML Patients After Allogeneic Stem Cell Transplant With Post-Transplant Cyclophosphamide
There are no strategies developed post-stem cell transplant (SCT) for patients who receive allogenic SCT with a significant amount of blasts prior SCT.
- Conditions
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Myelodysplastic Syndrome Acute Myeloid Leukemia
- Trial name
- NCT04361058
- Testing
- Nivolumab
- Where
- United States
- Sponsor
- SCRI Development Innovations, LLC
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
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