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Results for “Anemia, Hypoplastic, Congenital”

138 trials

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Showing 20 of 138 results

Early research (Phase 1)Study completedNCT00243399
What this trial is testing

Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia

Who this might be right for
Fanconi Anemia
Children's Hospital Medical Center, Cincinnati 10
Not applicableLooking for participantsNCT00106015
What this trial is testing

Diamond Blackfan Anemia Registry (DBAR)

Who this might be right for
AnemiaBlood Disease
Northwell Health 900
Not applicableLooking for participantsNCT04781790
What this trial is testing

French National Registry of Bone Marrow Failures

Who this might be right for
Bone Marrow Failure Syndrome
Assistance Publique - Hôpitaux de Paris 5,000
Large-scale testing (Phase 3)Study completedNCT00235391
What this trial is testing

Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

Who this might be right for
ThalassemiaSickle Cell DiseaseDiamond Blackfan Anemia+1 more
Novartis Pharmaceuticals 1,683
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 completedNCT00479115
What this trial is testing

Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100

Who this might be right for
Fanconi Anemia
Children's Hospital Medical Center, Cincinnati 1
Testing effectiveness (Phase 2)Study completedNCT00229619
What this trial is testing

Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia

Who this might be right for
Anemia, AplasticRed-Cell Aplasia, PureAnemia, Diamond-Blackfan
National Heart, Lung, and Blood Institute (NHLBI) 11
Large-scale testing (Phase 3)Study completedNCT00171821
What this trial is testing

A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload

Who this might be right for
Transfusion-dependent Iron Overload
Novartis Pharmaceuticals 1,784
Not applicableStudy completedNCT03609814
What this trial is testing

Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)

Who this might be right for
Hematologic MalignanciesNonmalignant DiseasesImmunodeficiencies+5 more
University of California, San Francisco 30
Testing effectiveness (Phase 2)Study completedNCT01362595
What this trial is testing

Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia

Who this might be right for
Diamond Blackfan AnemiaBlackfan Diamond SyndromeDBA+2 more
Northwell Health 55
Testing effectiveness (Phase 2)Ended earlyNCT03966053
What this trial is testing

The Use of Trifluoperazine in Transfusion Dependent DBA

Who this might be right for
Diamond Blackfan AnemiaPure Red Cell Aplasia
Adrianna Vlachos, MD 2
Not applicableStudy completedNCT00499070
What this trial is testing

Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment

Who this might be right for
Dyskeratosis CongenitaFanconi AnemiaMyelodysplastic Syndromes+2 more
University Hospital Freiburg 119
Not applicableWithdrawnNCT06227429
What this trial is testing

A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients

Who this might be right for
Hereditary Tyrosinemia, Type I
Swedish Orphan Biovitrum
Testing effectiveness (Phase 2)Ended earlyNCT00001962
What this trial is testing

Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure

Who this might be right for
Aplastic AnemiaPure Red Cell AplasiaDiamond Blackfan Anemia
National Heart, Lung, and Blood Institute (NHLBI) 100
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
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)WithdrawnNCT01419704
What this trial is testing

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

Who this might be right for
Anemia, Sickle CellComplex and Transfusion-dependent HemoglobinopathiesThalassemia+4 more
Talaris Therapeutics Inc.
Not applicableActive Not RecruitingNCT04437771
What this trial is testing

Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy

Who this might be right for
Fanconi Anemia Complementation Group AFanconi Anemia
Rocket Pharmaceuticals Inc. 9
Testing effectiveness (Phase 2)Study completedNCT01044186
What this trial is testing

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

Who this might be right for
Transfusional Iron Overload
Novartis Pharmaceuticals 30
Not applicableAvailableNCT01995305
What this trial is testing

Use Massive Parallel Sequencing and Exome Capture Technology to Sequence the Exome of Fanconi Anemia Children and Their Patents

Who this might be right for
Fanconi AnemiaAutosomal or Sex Linked Recessive Genetic DiseaseBone Marrow Hematopoiesis Failure, Multiple Congenital Abnormalities, and Susceptibility to Neoplastic Diseases.+1 more
Xiaofan Zhu
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