Clinical trials: Bone Marrow Failure
Diamond Blackfan Anemia
Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS)
Gene therapy is a new possible treatment for the anemia of DBAS. Gene therapy will soon be available for patients with RPS19-mutated DBAS.
- Trial name
- NCT07186179
- Testing
- Mobilization Regimen
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Northwell Health
Chronic Myocardial Ischemia
Bone Marrow-derived Mesenchymal Stem Cells (MSC) Administration in Weaning From Left Ventricular Assist Device
Left Ventricular Assist Devices are commonly used to assist failing hearts as a bridge to transplantation or for long-term support.
- Trial name
- MESAD (NCT02460770)
- Testing
- Autologous mesenchymal stem cells
- Where
- France
- Sponsor
- University Hospital, Toulouse
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
Aplastic Anemia
Romiplostim N01 in Chemoradiotherapy-induced Aplastic Anemia
This study aimed to explore the efficacy and safety of romiplostim N01 in the treatment of chemoradiotherapy-induced aplastic anemia (AA)
- Trial name
- NCT07572084
- Testing
- Romiplostim N01
- Volunteers
- 43 people
- Sponsor
- Peking Union Medical College Hospital
Refractory Idiopathic Aplastic Anemia, Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide in Patients With Acquired Refractory Aplastic Anemia or in Relapse After Immunosuppression
Outcomes for patients with severe aplastic anemia (SAA) who are refractory to first-line immunosuppressive therapy (IST) and who lack a matched unrelated donor (MUD) remain poor.
- Trial name
- HAPLO-EMPTY (NCT05126849)
- Testing
- Allogenic transplantation
- Where
- 35 sites in France
- Volunteers
- 31 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Kidney Transplantation, Kidney Failure, Chronic
Combined Kidney and Bone Marrow Transplantation to Prevent Kidney Transplant Rejection
In small initial studies, combined kidney and bone marrow transplants from the same donor have permitted some individuals to stop taking anti-rejection medicines without rejecting their transplant.
- Trial name
- NCT00801632
- Testing
- Kidney Transplantation, Bone marrow transplantation, MEDI-507
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Myelodysplastic Syndrome
Addition of Vorinostat to Azacitidine in Higher Risk MDS a Phase II add-on Study in Patients With Azacitidine Failure
Azacytidine (AZA) is the current standard of care for frontline patient treated with high-risk MDS and is clinically active in all type of MDS, however, 50% of the patients will never respond.
- Trial name
- GFM-AZA-VOR (NCT01748240)
- Testing
- Azacitidine and oral vorinostat
- Where
- 24 sites in France
- Volunteers
- 21 people
- Sponsor
- Groupe Francophone des Myelodysplasies
Childhood Acute Erythroleukemia (M6), Childhood Acute Megakaryocytic Leukemia (M7)
Identification of de Novo Fanconi Anemia in Younger Patients With Newly Diagnosed Acute Myeloid Leukemia
This research study is studying identification of de novo Fanconi anemia in younger patients with newly diagnosed acute myeloid leukemia.
- Conditions
- Childhood Acute Erythroleukemia (M6), Childhood Acute Megakaryocytic Leukemia (M7), Childhood Acute Minimally Differentiated Myeloid Leukemia (M0), Childhood Acute Monoblastic Leukemia (M5a)
- Trial name
- NCT01146210
- Testing
- laboratory biomarker analysis
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Children's Oncology Group
Graft Versus Host Disease, Leukemia
Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Other Hematologic or Metabolic Diseases
RATIONALE: Umbilical cord blood transplantation may be able to replace cells destroyed by chemotherapy or radiation therapy. PURPOSE: Phase II trial to study the effectiveness of umbilical cord blood transplantation in treating…
- Conditions
- Graft Versus Host Disease, Leukemia, Myelodysplastic Syndromes, Thymic Carcinoma
- Trial name
- NCT00003662
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- 15 sites in United States
- Volunteers
- 90 people
- Sponsor
- Roswell Park Cancer Institute
Anemia, Diamond-Blackfan
Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
Background: Diamond-Blackfan anemia (DBA) is treated with steroids. But some people cannot take steroids, or steroids don t work.
- Trial name
- NCT04269889
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Fanconi Anemia, Severe Marrow Failure
HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA).
- Conditions
- Fanconi Anemia, Severe Marrow Failure, Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML)
- Trial name
- RAFA (NCT02143830)
- Testing
- Busulfan, Cyclophosphamide, Fludarabine
- Where
- 3 sites in United States
- Volunteers
- 70 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Malignant Diseases (ie, Leukemia, MDS, Lymphoma), Non-malignant Diseases (ie, Bone Marrow Failure Syndromes)
Peripheral Blood Stem Cell Transplantation Using the CliniMACS Device
T cell depletion utilizing the CliniMACS device will allow more precise, specific and controlled graft engineering of peripheral blood stem cells from unrelated and partially matched related donors without an increase in relapse…
- Trial name
- NCT01071226
- Testing
- CliniMACS (CD+3, CD+19 depletion), CliniMACS (CD 34+ positive selection), HSCT
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Medical College of Wisconsin
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Bone Marrow Transplantation in Treating Patients With Severe Aplastic Anemia or Rejection of Previous Bone Marrow Transplant
RATIONALE: Bone marrow from donors may be able to treat patients with severe aplastic anemia and patients whose bodies have rejected previous bone marrow transplantation.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00005852
- Testing
- anti-thymocyte globulin, filgrastim, muromonab-CD3
- Sponsor
- H. Lee Moffitt Cancer Center and Research Institute