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Results for “Rare Abnormalities”

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Showing 20 of 0 results

Not applicableStudy completedNCT00004805
What this trial is testing

Study of the Effect of Four Methods of Cardiopulmonary Resuscitation Instruction on Psychosocial Response of Parents With Infants at Risk of Sudden Death

Who this might be right for
Pulmonary HypertensionBronchopulmonary DysplasiaRespiratory Distress Syndrome
National Center for Research Resources (NCRR) 500
Not applicableEnded earlyNCT00006054
What this trial is testing

Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies

Who this might be right for
Immunologic Deficiency SyndromesChediak-Higashi SyndromeCommon Variable Immunodeficiency+11 more
Fairview University Medical Center
Not applicableUnknownNCT00004374
What this trial is testing

Study of Systemic Amyloidosis Presentation and Prognosis

Who this might be right for
Amyloidosis
National Center for Research Resources (NCRR) 100
Not applicableStudy completedNCT01880983
What this trial is testing

Mitoferrin-1 Expression in Erythropoietic Protoporphyria (Porphyria Rare Disease Clinical Research Consortium (RDCRC))

Who this might be right for
Erythropoietic Protoporphyria (EPP)
University of Alabama at Birmingham 150
Not applicableUnknownNCT00004341
What this trial is testing

Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders

Who this might be right for
X-Linked AgammaglobulinemiaX-Linked Hyper IgM SyndromeWiskott-Aldrich Syndrome+1 more
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Not applicableStudy completedNCT00004695
What this trial is testing

Randomized Study of Polyethylene-Glycol-Conjugated Interleukin 2 in Patients With Common Variable Immunodeficiency

Who this might be right for
Common Variable Immunodeficiency
Icahn School of Medicine at Mount Sinai 48
Not applicableStudy completedNCT05793385
What this trial is testing

The Uncommon Complications of Meckel's Diverticulum

Who this might be right for
To Report the Rare Occurrence of Meckel's Diverticulum
Haramaya Unversity 4
Not applicableStudy completedNCT00004350
What this trial is testing

Evaluation of Fanconi Syndrome and Cystinosis

Who this might be right for
CystinosisFanconi Syndrome
National Center for Research Resources (NCRR) 12
Large-scale testing (Phase 3)Study completedNCT00004785
What this trial is testing

Phase III Randomized, Double-Blind Study of Dexamethasone Vs Dexamethasone/Methylprednisolone Vs Placebo for Bronchopulmonary Dysplasia

Who this might be right for
Bronchopulmonary Dysplasia
National Center for Research Resources (NCRR) 54
Not applicableStudy completedNCT05712564
What this trial is testing

Fatigue, Depressive Disorders and Insomnia in Adult Patients with Marfan Syndrome and Ehlers-Danlos Syndrome: Survey

Who this might be right for
Rare DiseasesFatigueInsomnia+1 more
IRCCS Policlinico S. Donato 282
Not applicableLooking for participantsNCT06833489
What this trial is testing

Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases

Who this might be right for
Rare Genetic Muscle DiseasesMuscular Dystrophy, DuchenneMuscular Dystrophy, Becker+2 more
Assistance Publique Hopitaux De Marseille 50
Not applicableStudy completedNCT00004648
What this trial is testing

Studies of Hereditary Hemorrhagic Telangiectasia

Who this might be right for
Telangiectasia, Hereditary Hemorrhagic
National Center for Research Resources (NCRR) 65
Post-approval studies (Phase 4)Study completedNCT00004338
What this trial is testing

Study of Zinc for Wilson Disease

Who this might be right for
Wilson Disease
National Center for Research Resources (NCRR) 300
Not applicableStudy completedNCT02814747
What this trial is testing

Evaluate and Understand Preferences and Representations in Families of Patients With Regard to High-throughput Sequencing Technology for Diagnostic Purposes

Who this might be right for
Rare Diseases
Centre Hospitalier Universitaire Dijon 530
Not applicableStudy completedNCT05245123
What this trial is testing

Psychosocial Situation of Children With Rare Solid Abdominal Tumors and Their Families

Who this might be right for
NeuroblastomaNephroblastomaHepatoblastoma
Universitätsklinikum Hamburg-Eppendorf 100
Not applicableStudy completedNCT00005103
What this trial is testing

Study of the Pathogenesis of Porphyria Cutanea Tarda

Who this might be right for
Porphyria Cutanea Tarda
National Center for Research Resources (NCRR) 120
Testing effectiveness (Phase 2)Study completedNCT00004488
What this trial is testing

Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease

Who this might be right for
Gaucher's DiseaseOsteopenia
Children's Hospital Medical Center, Cincinnati 82
Testing effectiveness (Phase 2)Study completedNCT00004314
What this trial is testing

Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease

Who this might be right for
Lesch-Nyhan Syndrome
National Center for Research Resources (NCRR) 2
Not applicableLooking for participantsNCT05810181
What this trial is testing

Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)

Who this might be right for
Sickle Cell Disease
St. Jude Children's Research Hospital 145
Not applicableLooking for participantsNCT06250595
What this trial is testing

European Rare Blood Disorders Platform (ENROL)

Who this might be right for
AnemiaBone Marrow FailureBleeding Disorder+9 more
Hospital Universitari Vall d'Hebron Research Institute 37,090
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