Clinical trials: Inherited Bone Marrow Failure Syndrome
Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria
Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF
This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell…
- Conditions
- Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Inherited Bone Marrow Failure Syndromes
- Trial name
- NCT03047746
- Testing
- CliniMACs
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Children's Hospital of Philadelphia
Fanconi Anemia, Anemia, Hypoplastic, Congenital
Prenatal Transplantation for Fetuses With Fanconi Anemia
The investigators aim to evaluate the safety and efficacy of in utero hematopoietic stem cell transplantation (IUHSCT) for the treatment of fetuses diagnosed with Fanconi anemia (FA) during pregnancy.
- Conditions
- Fanconi Anemia, Anemia, Hypoplastic, Congenital, Congenital Bone Marrow Failure Syndromes, Bone Marrow Failure Disorders
- Trial name
- NCT07408583
- Testing
- IUHSCT for FA-affected fetuses
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Agnieszka Czechowicz
Fanconi Anemia
Hematopoietic Stem Cell Transplant for Fanconi Anemia
The trial proposed is a single arm phase II treatment protocol designed to examine engraftment, toxicity, graft-versus-host disease, and ultimate disease-free survival following a novel cytoreductive regimen including busulfan…
- Trial name
- FA (NCT01071239)
- Testing
- CliniMACs device, Busulfan, Fludarabine
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Medical College of Wisconsin
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
Fanconi Anemia
Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102
- Trial name
- NCT07527975
- Testing
- RP-L102
- Where
- 3 sites in United States, Spain and 1 more
- Volunteers
- 14 people
- Sponsor
- Rocket Pharmaceuticals Inc.
MDS, Anemia, Aplastic
Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells
The main purpose of this investigational (not approved by the FDA) Phase I research is to test whether transplantation of umbilical cord blood cells can be safely supplemented with a transfusion of a portion of these cells that…
- Conditions
- MDS, Anemia, Aplastic, Inborn Errors of Metabolism, Congenital Marrow Failure
- Trial name
- NCT00692926
- Testing
- ALDHbr Umbilical Cord Blood Cells
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Joanne Kurtzberg, MD
Diamond-Blackfan Anemia, Kostmann's Neutropenia
Stem Cell Transplant for Bone Marrow Failure Syndromes
The researchers hypothesize that it will be possible to perform unrelated bone marrow or cord blood transplants in a safer manner by using less intensive therapy yet still achieve an acceptable level of donor cell engraftment for…
- Conditions
- Diamond-Blackfan Anemia, Kostmann's Neutropenia, Shwachman-Diamond Syndrome
- Trial name
- NCT00176878
- Testing
- Stem cell transplant, Fludarabine monophosphate, Total lymphoid irradiation
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Cancer
Antithymocyte Globulin, Clofarabine, and Rituximab in Treating Patients After an Unsuccessful Stem Cell Transplant
RATIONALE: Antithymocyte globulin, clofarabine, and rituximab may stop the patient's immune system from rejecting the donor's stem cells when they do not exactly match the patient's blood.
- Trial name
- NCT00617929
- Testing
- anti-thymocyte globulin, rituximab, clofarabine
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Transfusional Iron Overload
A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity
The purpose of this open-label, non-comparative, multi-center protocol was to further evaluate safety and to provide treatment with ICL670 to patients who had or were at risk of life threatening complications due to transfusional…
- Trial name
- NCT01044186
- Testing
- ICL670
- Where
- 18 sites in United States, Greece and 1 more
- Volunteers
- 30 people
- Sponsor
- Novartis Pharmaceuticals
Bone Marrow Diseases
Cytokine Gene Polymorphisms in Bone Marrow Failure
This study will examine whether cytokine gene polymorphisms affect the progression or response to therapy of bone marrow failure disorders.
- Trial name
- NCT00085670
- Where
- United States
- Volunteers
- 79 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Bone Marrow Failure Syndromes, Erythrocyte Disorder
Investigation of the Genetics of Hematologic Diseases
The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of blood diseases.
- Conditions
- Bone Marrow Failure Syndromes, Erythrocyte Disorder, Leukocyte Disorder, Hemostasis
- Trial name
- NCT02720679
- Where
- United States
- Volunteers
- 1,716 people
- Sponsor
- St. Jude Children's Research Hospital
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant
This protocol provides expanded access to bone marrow transplants for children who lack a histocompatible (tissue matched) stem cell or bone marrow donor when an alternative donor (unrelated donor or half-matched related donor)…
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT01200017
- Testing
- CD34+ enriched, T Cell Depleted donor stem cell product
- Where
- United States
- Sponsor
- Christopher Dvorak
Fanconi's Anemia
Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia
OBJECTIVES: I. Evaluate the toxicity of amifostine in patients with bone marrow failure related to Fanconi's anemia. II. Determine the efficacy of this treatment regimen in this patient population. III.
- Trial name
- NCT00006127
- Testing
- amifostine
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Dana-Farber Cancer Institute
Leukemia, Lymphoma
CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant
CD34+ stem cell selection in children, adolescents and young adults receiving partially matched family donor or matched unrelated adult donor allogeneic bone marrow or peripheral blood stem cell transplant will be safe and well…
- Conditions
- Leukemia, Lymphoma, Bone Marrow Failure, Immunodeficiencies
- Trial name
- NCT01049854
- Testing
- Full Intensity with TBI, Full Intensity, Reduced Intensity
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- New York Medical College
Fanconi Anemia
Blood Cell Collection for Future Use in Individuals With Fanconi Anemia
Fanconi anemia (FA) is a disease that affects an individual's bone marrow. It is caused by a defective gene in the CD34+ cells, which are responsible for producing various types of blood cells.
- Trial name
- NCT00271089
- Testing
- PBSC Collection, Bone Marrow Harvest, CliniMacs Cell Selection System
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Inherited BMF Syndrome, Inherited Platelet Disorder
Diagnosis and Phenotype Characterisation Using Genomics in Patients With Inherited Bone Marrow Failure (IBMDx Study)
This project seeks to perform whole genome sequence (WGS) and whole transcriptome sequence (WTS) analysis on 350 patients with suspected inherited bone marrow failure syndromes and related disorder (IBMFS-RD) in order to increase…
- Conditions
- Inherited BMF Syndrome, Inherited Platelet Disorder, Hematologic Diseases
- Trial name
- IBMDx (NCT05196789)
- Testing
- whole genome and transcriptome sequencing
- Where
- Australia
- Volunteers
- 350 people
- Sponsor
- Peter MacCallum Cancer Centre, Australia
Fanconi Anemia
Gene Therapy for Fanconi Anemia
This clinical trial will access the toxicity and efficacy of infusion of gene modified cells for patients with Fanconi anemia (FA).
- Trial name
- NCT01331018
- Testing
- Bone Marrow Aspiration, Filgrastim, Genetically Engineered Hematopoietic Stem Progenitor Cells
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Fred Hutchinson Cancer Center
Fanconi Anemia
Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A
This is an open, Phase I / II clinical trial to evaluate the safety and efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene for patients with Fanconi…
- Trial name
- FANCOLEN-1 (NCT03157804)
- Testing
- IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs), Genetically Engineered Hematopoietic Stem/Progenitor Cells, Laboratory Biomarker Analysis
- Where
- 2 sites in Spain
- Volunteers
- 9 people
- Sponsor
- Hospital Infantil Universitario Niño Jesús, Madrid, Spain
Bone Marrow Failure, Hemoglobinopathies
Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases
This phase II trial investigates how well a naive T cell depleted graft work for the reduction of graft versus host disease in patients with non-malignant diseases requiring hematopoietic cell transplantation.
- Conditions
- Bone Marrow Failure, Hemoglobinopathies, Primary Immune Deficiency (PID), Autoimmune Cytopenia
- Trial name
- NCT07660783
- Testing
- ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs
- Where
- 2 sites in United States
- Volunteers
- 40 people
- Sponsor
- Fred Hutchinson Cancer Center
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