Clinical trials: Bone Marrow Failure Syndrome
Hereditary Diseases, Acquired Aplastic Anemia
Cord Blood Transplantation in Severe Aplastic Anemia
This is a Prospective Phase II Study to evaluate Cord Blood Transplantation in Inherited or Acquired Severe Aplastic Anemia Refractory or in Relapse after Immunosuppressive Therapy in the absence of an HLA identical donor;
- Conditions
- Hereditary Diseases, Acquired Aplastic Anemia, Relapse, Absence of an HLA Identical Donor
- Trial name
- APCORD (NCT01343953)
- Testing
- Cord Blood Transplantation
- Where
- France
- Volunteers
- 26 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Inherited Bone Marrow Failure Syndrome, Familial Platelet Disorder With Predisposition to Myeloid Malignancies
Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency
Background: Runt-related transcription factor 1 (RUNX1) gene regulates the formation of blood cells. People with mutations of this gene may bleed or bruise easily; they are also at higher risk of getting cancers of the blood…
- Trial name
- NCT06090669
- Testing
- TruSight Oncology, imatinib
- Where
- United States
- Volunteers
- 71 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Cancer
Donor Natural Killer Cell Infusion in Preventing Relapse or Graft Failure in Patients Who Have Undergone Donor Bone Marrow Transplant
RATIONALE: Giving donor natural killer cells to patients who have undergone donor bone marrow transplant may make the transplant work better and keep cancer cells from coming back.
- Trial name
- NCT00569283
- Testing
- therapeutic allogeneic lymphocytes
- Where
- 2 sites in South Korea
- Volunteers
- 18 people
- Sponsor
- Asan Medical Center
Metabolic Disorder; Carbohydrate, Absorption, Intestine
Determination of the Postprandial Responses to Two Cereal Foods Differing by Their SDS Content Consumed Alone.
This study aimed at determining the 4-hours postprandial blood glucose and insulin responses to the ingestion of cereal product differing by their SDS content.
- Trial name
- NCT02538653
- Testing
- Sandwich biscuit high in SDS, Co-extruded cereal low in SDS
- Where
- Australia
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Mondelēz International, Inc.
Fanconi Anemia
Whole Blood Biospecimen Collection for Subjects With Fanconi Anemia
The primary study objective is to collect biospecimen samples (e.g., blood) from participants diagnosed with Fanconi Anemia. The biospecimens will be used to create a biorepository that can be used to identify disease associated…
- Trial name
- NCT05910853
- Testing
- Specimen Donation
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Sanguine Biosciences
Asherman Syndrome, Atrophic Endometrium
Bone Marrow Derived Stem Cells Mobilization for Treatment of Abnormal Endometrium
This study will assess the use of autologous bone marrow stem cells mobilization using 1,1'-[1,4-phenylenebis-(methylene)]-bis-1,4,8,11-tetraazacyclotetradecane (PLERIXAFOR) as an effective medical therapy for the treatment of…
- Conditions
- Asherman Syndrome, Atrophic Endometrium, Recurrent Implantation Failure
- Trial name
- NCT05343572
- Testing
- Plerixafor
- Where
- United States
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Hugh Taylor
Aplastic Anemia, Myelodysplastic Syndromes
Posaconazole Prophylaxis During ATG Treatment for hMDS/AA Patients
To investigate the efficacy of posaconazole as prophylaxis antifungal agent in aplastic anemia / hypoplastic myelodysplastic syndrome (AA/hMDS) patients undergoing antithymocyte globulin (ATG) treatment
- Conditions
- Aplastic Anemia, Myelodysplastic Syndromes, Fungal Infection
- Trial name
- NCT03318159
- Testing
- Posaconazole
- Where
- South Korea
- Volunteers
- 20 people
- Sponsor
- Seoul National University Hospital
Fanconi Anemia
Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia
RATIONALE: A bone marrow or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy.
- Trial name
- NCT00258427
- Testing
- anti-thymocyte globulin, filgrastim, busulfan
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Severe Aplastic Anemia
Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia
The purpose of this study is to determine the feasibility of comparing outcomes of patients treated de novo with immunosuppressive therapy (IST) versus matched unrelated donor (MUD) hematopoietic stem cell transplant (HSCT) for…
- Trial name
- NCT02845596
- Testing
- cyclosporine, Matched Unrelated Donor Hematopoietic Stem Cell Transplant, horse anti-thymocyte globulin (ATG)
- Where
- 13 sites in United States
- Volunteers
- 40 people
- Sponsor
- Michael Pulsipher
Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies
This phase II trial studies how well reduced intensity donor stem cell transplant works in treating patients with hematologic malignancies.
- Conditions
- Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
- Trial name
- NCT01384513
- Testing
- Fludarabine, Busulfan, Total Body Irradiation (TBI)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Aplastic Anemia
Phase II Study of Bone Marrow Transplantation Using Related Donors in Patients With Aplastic Anemia
OBJECTIVES: I. Evaluate the efficacy of related, HLA-identical bone marrow transplantation following cyclophosphamide (CTX) and antithymocyte globulin in patients with aplastic anemia. II.
- Trial name
- NCT00004323
- Testing
- anti-thymocyte globulin, cyclophosphamide, cyclosporine
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Center for Research Resources (NCRR)
Fanconi's Anemia
Phase I/II Study of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Cell Transplantation in Patients With Fanconi's Anemia
OBJECTIVES: I. Determine the probability of engraftment with total body irradiation, cyclophosphamide, fludarabine, and anti-thymocyte globulin followed by HLA nongenotypically identical donor, T-cell depleted hematopoietic cell…
- Trial name
- NCT00005898
- Testing
- anti-thymocyte globulin, cyclophosphamide, cyclosporine
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Minnesota
Hematological Malignancies
The Use of Intensive Care in Critically Ill Cancer Haematological Patients: "TRIAL-OH"
The purpose of this study is to determine: Condition of intensive care unit (ICU) admission in patients with haematological malignancies. ICU hospital and up to one year mortality.
- Trial name
- NCT01172132
- Where
- France
- Volunteers
- 1,011 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hematologic Malignancies, Inborn Errors of Metabolism Disorders
Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants
The objective of this study is to make T-cell depleted stem cells from a family member who is a half match (haplo-identical) available on an expanded access basis to patients receiving one or two unrelated cord blood transplants…
- Conditions
- Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
- Trial name
- NCT01881334
- Testing
- CliniMACS CD34 Reagent System
- Where
- United States
- Sponsor
- Joanne Kurtzberg, MD
Aplastic Anemia
Mesenchymal Stem Cells in the Treatment of Relapsed/Refractory Severe Acquired Aplastic Anemia
Mesenchymal stem cells have been tested in many autoimmune disorders with encouraging results and may be an alternative to the treatment of immune-mediated severe acquired aplastic anemia.
- Trial name
- NCT01297972
- Testing
- Intravenous bone marrow mesenchymal stem cells infusion
- Where
- Brazil
- Volunteers
- 9 people
- Sponsor
- University of Sao Paulo
Severe Aplastic Anemia (SAA), Transfusion-dependent Non-severe Aplastic Anemia
CsA+EPAG/HPAG+Romiplostim N01 in the Treatment of Newly-diagnosed SAA/TD-NSAA
This study aimed to explore the efficacy and safety of cyclosporine (CsA) combined with eltrombopag (EPAG)/hetrombopag (HPAG) and romiplostim N01 in the treatment of newly-diagnosed transfusion-dependent aplastic anemia (TD-NSAA)…
- Trial name
- NCT07523009
- Testing
- Cyclosporine (CsA), Thrombopoietin Receptor Agonist, Romiplostim N01
- Sponsor
- Peking Union Medical College Hospital
Aplastic Anemia
Efficacy and Safety in Transfusion Independent Non-severe Aplastic Anemia
Aplastic anemia (AA) is a rare bone marrow failure disease characterized by bone marrow hypocellularity and peripheral blood pancytopenia.
- Trial name
- NCT05399732
- Testing
- Luspatercept, Cyclosporine
- Where
- China
- Volunteers
- 58 people
- Sponsor
- Bing Han
Acute Leukemias, Aplastic Anemia
Safety Study of CD3/CD19 Depleted Haploidentical Stem Cells
Feasibility and toxicity of haploidentical transplantation of CD3/CD19 depleted stem cells in combination with a toxicity reduced conditioning regimen or with standard conditioning regimens according to underlying disease.
- Conditions
- Acute Leukemias, Aplastic Anemia, Childhood Solid Tumor
- Trial name
- NCT01919866
- Testing
- Transplantation of CD3/CD19 depleted stem cells
- Where
- 5 sites in Austria, Germany
- Volunteers
- 120 people
- Sponsor
- University Children's Hospital Tuebingen
Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia
Open-label Clinical Trial to Investigate the Safety and Tolerability of Allogeneic B-cell Concentrates for Immune Reconstitution After Allogeneic Stem Cell Transplantation Measured as Response to a Antedated Single Vaccination
The reconstitution of a functioning immune system after allogeneic stem cell transplantation takes months to years. Particularly memory B-lymphocytes reconstitute poorly with the current conditioning regimes.
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Myeloid Leukemia, Non Hodgkin's Lymphoma
- Trial name
- B-cell therapy (NCT02007811)
- Testing
- allogeneic donor derived B-lymphocytes
- Where
- Germany
- Volunteers
- 15 people
- Sponsor
- University of Erlangen-Nürnberg Medical School
Severe Aplastic Anemia
Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502)
This study is a prospective, multicenter phase II study with patients receiving haploidentical transplantation for Severe Aplastic Anemia (SAA).
- Trial name
- CHAMP (NCT02918292)
- Testing
- Antithymocyte Globulin (ATG), Fludarabine, Cyclophosphamide
- Where
- 28 sites in United States
- Volunteers
- 32 people
- Sponsor
- Medical College of Wisconsin