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Results for “RPS19 Deficient Diamond-Blackfan Anemia”

23 trials

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Showing 20 of 23 results

Testing effectiveness (Phase 2)Ended earlyNCT02512679
What this trial is testing

Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells

Who this might be right for
Stem Cell TransplantationBone Marrow TransplantationPeripheral Blood Stem Cell Transplantation+10 more
Children's Hospital Los Angeles 20
Not applicableLooking for participantsNCT02720679
What this trial is testing

Investigation of the Genetics of Hematologic Diseases

Who this might be right for
Bone Marrow Failure SyndromesErythrocyte DisorderLeukocyte Disorder+10 more
St. Jude Children's Research Hospital 1,716
Not applicableLooking for participantsNCT03050268
What this trial is testing

Familial Investigations of Childhood Cancer Predisposition

Who this might be right for
Acute LeukemiaAdenomatous PolyposisAdrenocortical Carcinoma+43 more
St. Jude Children's Research Hospital 1,500
Testing effectiveness (Phase 2)Study completedNCT00001749
What this trial is testing

Medical Treatment for Diamond Blackfan Anemia

Who this might be right for
Fanconi's AnemiaHematologic Disease
National Heart, Lung, and Blood Institute (NHLBI) 25
Testing effectiveness (Phase 2)UnknownNCT02061800
What this trial is testing

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

Who this might be right for
Chronic Myeloid Leukemia (CML)Acute Myelogenous Leukemia (AML)Myelodysplastic Syndrome (MDS)+3 more
Diane George 14
Not applicableLooking for participantsNCT04528355
What this trial is testing

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+2 more
Paul Szabolcs 50
Testing effectiveness (Phase 2)Looking for participantsNCT01962415
What this trial is testing

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+4 more
Paul Szabolcs 100
Not applicableEnded earlyNCT00290628
What this trial is testing

Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer

Who this might be right for
Chronic Myeloproliferative DisordersDiamond-blackfan AnemiaFanconi Anemia+6 more
Masonic Cancer Center, University of Minnesota 43
Testing effectiveness (Phase 2)Active Not RecruitingNCT01966367
What this trial is testing

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

Who this might be right for
Bone Marrow Failure SyndromeSevere Aplastic AnemiaSevere Congenital Neutropenia+13 more
Diane George 37
Testing effectiveness (Phase 2)Study completedNCT00919503
What this trial is testing

Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders

Who this might be right for
Non-Neoplastic Hematologic and Lymphocytic Disorder
Fred Hutchinson Cancer Center 98
Early research (Phase 1)Study completedNCT01917708
What this trial is testing

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Who this might be right for
Hurler SyndromeFanconi AnemiaGlanzmann Thrombasthenia+12 more
Emory University 10
Testing effectiveness (Phase 2)Ended earlyNCT03733249
What this trial is testing

Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study

Who this might be right for
Acute Lymphoblastic LeukemiaLeukemia, Acute Myeloid (AML), ChildLymphoma, Non-Hodgkin+9 more
Bellicum Pharmaceuticals 187
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Early research (Phase 1)Looking for participantsNCT07476183
What this trial is testing

Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia

Who this might be right for
RPS19 Deficient Diamond-Blackfan Anemia
Apriligen, Inc. 4
Early research (Phase 1)Study completedNCT01586455
What this trial is testing

Human Placental-Derived Stem Cell Transplantation

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis VIAdrenoleukodystrophy+13 more
New York Medical College 43
Early research (Phase 1)Ended earlyNCT02231710
What this trial is testing

Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

Who this might be right for
Primary Immune Deficiency DisordersHemophagocytic LymphohistiocytosisInherited Bone Marrow Failure Syndrome+2 more
Bellicum Pharmaceuticals 1
Testing effectiveness (Phase 2)Study completedNCT03513328
What this trial is testing

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

Who this might be right for
Bone Marrow Failure SyndromeThalassemiaSickle Cell Disease+13 more
University of Florida 6
Testing effectiveness (Phase 2)Study completedNCT01529827
What this trial is testing

Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies

Who this might be right for
Accelerated Phase Chronic Myelogenous LeukemiaAdult Acute Lymphoblastic Leukemia in RemissionAdult Acute Myeloid Leukemia in Remission+75 more
Roswell Park Cancer Institute 94
Not applicableLooking for participantsNCT00027274
What this trial is testing

Cancer in Inherited Bone Marrow Failure Syndromes

Who this might be right for
Diamond Blackfan AnemiaDyskeratosis CongenitaFanconi Anemia+2 more
National Cancer Institute (NCI) 4,000
Not applicableEnded earlyNCT01319851
What this trial is testing

Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation

Who this might be right for
ThalassemiaSickle Cell DiseaseGlanzmann Thrombasthenia+10 more
Emory University 3
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