Clinical trials: Aplastic Anemias, active
Myelodysplastic Syndromes
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Background: Myelodysplastic syndrome (MDS) and myelodysplastic/myeloproliferative neoplasm (MDS/MPN) are blood disorders that can cause serious complications in children and adults.
- Trial name
- NCT06303193
- Testing
- pacritinib
- Where
- United States
- Volunteers
- 160 people
- Sponsor
- National Cancer Institute (NCI)
Acute Leukemia, Severe Aplastic Anemia
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion.
- Conditions
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma
- Trial name
- NCT04099966
- Testing
- alpha beta depletion
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Mitchell Cairo
Severe Aplastic Anemia, Telomere Biology Disorders
Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
Background: Bone marrow failure diseases are rare. Much is known about the diseases at the time of diagnosis, but long-term data about the effects of the diseases and treatments are lacking.
- Conditions
- Severe Aplastic Anemia, Telomere Biology Disorders, Inherited Bone Marrow Failure Syndromes
- Trial name
- NCT05012111
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Hematopoietic/Lymphoid Cancer, Accelerated Phase Chronic Myelogenous Leukemia
Infection Prophylaxis and Management in Treating Cytomegalovirus (CMV) Infection in Patients With Hematologic Malignancies Previously Treated With Donor Stem Cell Transplant
RATIONALE: Infection prophylaxis and management may help prevent cytomegalovirus (CMV) infection caused by a stem cell transplant.
- Conditions
- Hematopoietic/Lymphoid Cancer, Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
- Trial name
- NCT01199562
- Testing
- infection prophylaxis and management, laboratory biomarker analysis, flow cytometry
- Where
- United States
- Volunteers
- 153 people
- Sponsor
- City of Hope Medical Center
Aplastic Anaemia
Conditioning Regimen Containing Low-Dose ATG for the Treatment of AA Receiving sUCBT
To evaluate the safety and efficacy of Single Umbilical Cord blood transplantation (sUCBT) containing low dose ATG based conditioning regimen in the treatment of Aplastic Anemia (AA)
- Trial name
- NCT07885982
- Testing
- Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®)
- Where
- China
- Volunteers
- 59 people
- Sponsor
- Anhui Provincial Hospital
Aplastic Anemia
CK0801 for Treatment of Aplastic Anemia
This Phase 2, multicenter, open-label study is evaluating CK0801 for the treatment of aplastic anemia in adults with persistent transfusion dependence after at least one prior line of therapy or intolerance to standard-of-care…
- Trial name
- NCT07499102
- Testing
- CK0801
- Volunteers
- 12 people
- Sponsor
- Cellenkos, Inc.
Diamond Blackfan Anemia, Dyskeratosis Congenita
Cancer in Inherited Bone Marrow Failure Syndromes
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.
- Conditions
- Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome
- Trial name
- NCT00027274
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Background: Stem cell transplants from related donors (allogenic stem cell transplants) can be used to treat individuals with certain kinds of severe blood diseases or cancers, such as severe anemia.
- Trial name
- NCT01174108
- Testing
- Miltenyi CD34 Reagent System, Donor derived G-CSF mobilized PBC
- Where
- 2 sites in United States
- Volunteers
- 120 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Inherited Bone Marrow Failure Syndrome, Fanconi Anemia
Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data
A non-profit, translational, prospective and retrospective study. Patients will be enrolled according to the procedures described in the protocol, and samples will be processed and analyzed as specified in the protocol.
- Conditions
- Inherited Bone Marrow Failure Syndrome, Fanconi Anemia, Dyskeratosis Congenita, Telomere Biology Disorder
- Trial name
- 2023-12377081 (NCT07877727)
- Where
- 4 sites in Italy
- Volunteers
- 250 people
- Sponsor
- Istituto Giannina Gaslini
Bone Marrow Failure Disorders, VEXAS Syndrome
Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study
STUDY DESCRIPTION: Bone marrow failure can result from immune mediated attack on stem cells or from inherited genetic defects such as telomere biology disorders or Fanconi anemia.
- Conditions
- Bone Marrow Failure Disorders, VEXAS Syndrome, Hemoglobinurea, Paroxysmal, Myelodysplastic Syndromes
- Trial name
- NCT07102849
- Where
- United States
- Volunteers
- 1,400 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Severe Aplastic Anemia
Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
Severe aplastic anemia is a rare and serious form of bone marrow failure related to an immune-mediated mechanism that results in severe pancytopenia and high risk for infections and bleeding.
- Trial name
- NCT02828592
- Testing
- Fludarabine, Cyclophosphamide, Total Body Irradiation
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Northside Hospital, Inc.
Severe Aplastic Anemia, Single Lineage Cytopenias, T-LGL
JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure
Background: Immune bone marrow failure is a condition that occurs when a person s immune system attacks the cells of the bone marrow. This can lead to diseases including different types of anemias and blood cancers.
- Conditions
- Severe Aplastic Anemia, Single Lineage Cytopenias, T-LGL, Hypoplastic MDS
- Trial name
- NCT05998408
- Testing
- Ruxolitinib
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion
Patients with medical conditions requiring allogeneic hematopoietic cell transplantation (allo-HCT) are at risk of developing a condition called graft versus host disease (GvHD) which carries a high morbidity and mortality.
- Conditions
- Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Myelodysplastic Syndromes, Chronic Myeloid Leukemia
- Trial name
- HAPLOTAB (NCT05236764)
- Testing
- CliniMACS
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Baylor College of Medicine
Severe Aplastic Anemia
Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy
Background: People with severe aplastic anemia (SAA) do not make enough red and white blood cells, and/or platelets. Their body's immune system stops the bone marrow from making these cells.
- Trial name
- NCT02979873
- Testing
- Sirolimus
- Where
- United States
- Volunteers
- 84 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Hematologic Malignancy, Bone Marrow Transplant Rejection
Darzalex Faspro (Daratumumab and Hyaluronidase-fihj) Before Standard Desensitization and Allogeneic Bone Marrow or Peripheral Blood Stem Cell Transplantation in Adult Patients at High-risk for Primary Graft Failure Secondary to Donor Specific Antibodies
This research is being done to investigate the safety and effectiveness of Darzalex Faspro (daratumumab and hyaluronidase-fihj) (a monoclonal antibody that targets plasma cells that make antibodies) and whether it can lower donor…
- Conditions
- Hematologic Malignancy, Bone Marrow Transplant Rejection, Acute Myeloid Leukemia (AML), Myelodysplastic Syndromes (MDS)
- Trial name
- NCT06398457
- Testing
- Darzalex Faspro (Daratumumab and hyaluronidase-fihj), JH-DSA Semi-Quant Screen and Response Score
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Severe Aplastic Anemia, Hypo-Plastic MDS
Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome
Background: Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant.
- Conditions
- Severe Aplastic Anemia, Hypo-Plastic MDS, Myelodysplastic Syndrome (MDS)
- Trial name
- NCT03173937
- Testing
- Omidubicel (former CordIn), Miltenyi CliniMACS(R) CD34 Reagent System
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Aplastic Anemia
Phase I/II Study of Anti-CD38 Monoclonal Antibody in Refractory Severe Aplastic Anemia
This is a phase I/II clinical study in adult patients with refractory severe aplastic anemia (SAA). Eligible patients must meet the criteria for refractory SAA and have a platelet count (PLT) <30 × 10^9/L and/or hemoglobin (HGB)…
- Trial name
- NCT07714512
- Testing
- Isatuximab
- Where
- China
- Volunteers
- 32 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Severe Aplastic Anemia, Hematopoietic Cell Transplant
Thiotepa-Based Conditioning for Haploidentical Hematopoietic Stem Cell Transplantation in Older Patients With Severe Aplastic Anemia
This prospective, single-center observational cohort study will follow participants aged 50 years or older with severe or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with a…
- Trial name
- NCT07819812
- Testing
- Thiotepa
- Volunteers
- 20 people
- Sponsor
- Peking University People's Hospital
Aplastic Anemia, Myelodysplastic Syndromes
Brain Cognitive Network Abnormalities in Anemia Patients Using fNIRS
Anemia is a global public health concern and is closely linked to dysfunction of brain cognitive neural networks, a key mechanism underlying cognitive impairment.
- Conditions
- Aplastic Anemia, Myelodysplastic Syndromes, Autoimmune Hemolytic Anemia, Functional Near-Infrared Spectroscopy
- Trial name
- AN-CNA (NCT07578675)
- Where
- China
- Volunteers
- 323 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Aplastic Anemia, Bone Marrow Failure Syndrome
Haploidentical HCT for Severe Aplastic Anemia
This study is a prospective, single center phase II clinical trial in which patients with Severe Aplastic Anemia (SAA) ) will receive a haploidentical transplantation.
- Trial name
- NCT04558736
- Testing
- Anti-Thymocyte Globulin (Rabbit), Fludarabine, Cyclophosphamide
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- St. Jude Children's Research Hospital