Clinical trials: Severe Aplastic Anemia, active
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)
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
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)
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)
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
Severe Aplastic Anemia (SAA)
Peripheral Blood Versus Bone Marrow Plus Peripheral Blood Grafts for Haploidentical Transplantation in Severe Aplastic Anemia
Severe aplastic anemia is a life-threatening bone marrow failure disorder. Haploidentical hematopoietic stem cell transplantation has become an important curative treatment option for patients who do not have an HLA-matched…
- Trial name
- PB-Graft Haplo (NCT07629947)
- Testing
- G-CSF-Primed Peripheral Blood Stem Cell Graft, G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell Graft
- Where
- China
- Volunteers
- 180 people
- Sponsor
- Peking University People's Hospital
Acute Leukemia, Myelodysplastic Syndromes
Sirolimus+Ruxolitinib+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to CNI
Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled.
- Conditions
- Acute Leukemia, Myelodysplastic Syndromes, Severe Aplastic Anemia (SAA)
- Trial name
- NCT07796620
- Testing
- Sirolimus, Ruxolitinib, MMF
- Volunteers
- 40 people
- Sponsor
- Peking University People's Hospital
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Background: Severe aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause serious blood problems.
- Conditions
- Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS), Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Trial name
- NCT03520647
- Testing
- Cyclophosphamide, Peripheral Blood Stem Cells
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Bone Marrow Failure Syndromes, Aplastic Anemias
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
This is a multicenter, open-label, Phase 1/2 basket study to evaluate the safety and efficacy of Dibotatug (DR-01) in adults with Bone Marrow Failure syndromes.
- Conditions
- Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Relapse
- Trial name
- BMF (NCT07712562)
- Testing
- Dibotatug (DR-01)
- Where
- 10 sites in United States
- Volunteers
- 60 people
- Sponsor
- Dren Bio
Hematologic Malignancies, Inherited Disorders of Metabolism
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.
- Conditions
- Hematologic Malignancies, Inherited Disorders of Metabolism, Inherited Abnormalities of Platelets, Histiocytic Disorders
- Trial name
- NCT01351545
- Testing
- A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
- Where
- 142 sites in United States
- Volunteers
- 99,999 people
- Sponsor
- Center for International Blood and Marrow Transplant Research
Severe Aplastic Anemia, Aplastic Anaemia
A Novel Conditioning Regimen for Haplo-HSCT in Older Patients With SAA
The goal of this prospective, multicenter, single arm observational study is to evaluate the efficacy and safety of the BFCA regimen in ≥ 40 years old SAA patients undergoing haplo-HSCT.
- Conditions
- Severe Aplastic Anemia, Aplastic Anaemia, Hematopoietic Cell Transplant
- Trial name
- NCT07586735
- Testing
- BFCA conditiong regimen
- Where
- China
- Volunteers
- 64 people
- Sponsor
- Peking University People's Hospital
Hemoglobinopathy (Disorder), Severe Aplastic Anemia
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children
This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition.
- Conditions
- Hemoglobinopathy (Disorder), Severe Aplastic Anemia, Bone Marrow Failure Syndrome
- Trial name
- NCT04356469
- Testing
- Haploidentical Hematopoietic Cell Transplantation
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Johns Hopkins All Children's Hospital
Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Refractory
Phase I Clinical Study of Haplo-HSCT Combined With Hypoxic 3D-Cultured Umbilical Cord MSC for the Treatment of SAA
Efficacy and Safety of Sequential Infusion of Hypoxic 3D-Cultured Umbilical Cord Mesenchymal Stem Cells in Haploidentical Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia: A Multicenter, Randomized, Phase 1 Trial
- Conditions
- Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Refractory, Severe Aplastic Anemia
- Trial name
- NCT07299123
- Testing
- 3D hypoxia-preconditioned UC-MSC group, 2D UC-MSC group
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Chinese PLA General Hospital
Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia
MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.
A phase II trial of a reduced intensity conditioned (RIC) allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for idiopathic severe aplastic anemia (SAA), paroxysmal nocturnal…
- Conditions
- Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia, Acquired Pure Red Cell Aplasia, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT06412497
- Testing
- Rituximab, Rabbit ATG, Cyclophosphamide
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Severe Aplastic Anemia (SAA), Transfusion-dependent Non-severe Aplastic Anemia
CsA+EPAG/HPAG+Romiplostim N01 in Newly-diagnosed SAA/TD-NSAA
This study aimed to explore the efficacy and safety of cyclosporine (CsA) +eltrombopag (EPAG)/hetrombopag (HPAG)+romiplostim N01 in the treatment of newly-diagnosed transfusion-dependent aplastic anemia (TD-NSAA) and severe…
- Trial name
- NCT07558434
- Testing
- Cyclosporine (CsA), thrombopoietin receptor agonist (TPO-RA), Romiplostim N01
- Volunteers
- 43 people
- Sponsor
- Peking Union Medical College Hospital