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Results for “Genetic Rare Disease”

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Showing 20 of 0 results

Early research (Phase 1)Study completedNCT00014729
What this trial is testing

Phase I Study of Isotretinoin in Patients With Recessive Dystrophic Epidermolysis Bullosa

Who this might be right for
Epidermolysis Bullosa
University of North Carolina 20
Not applicableLooking for participantsNCT04746066
What this trial is testing

Observational Study for Patients With Hemoglobinopathies and Rare Inherited Anemia and Covid 19

Who this might be right for
Haemoglobinopathies
Società Italiana Talassemie ed Emoglobinopatie 10,000
Not applicableStudy completedNCT04217538
What this trial is testing

Observational Study of a Cohort of Patients With Hereditary Epidermolysis Bullosa

Who this might be right for
Epidermolysis Bullosa
Centre Hospitalier Universitaire de Nice 41
Testing effectiveness (Phase 2)Study completedNCT00006136
What this trial is testing

Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia

Who this might be right for
Beta-Thalassemia
Boston University 15
Not applicableStudy completedNCT00323167
What this trial is testing

Rare Genetic Disorders of the Breathing Airways

Who this might be right for
Kartagener SyndromeCystic FibrosisPseudohypoaldosteronism+1 more
University of North Carolina, Chapel Hill 367
Not applicableStudy completedNCT00004815
What this trial is testing

Descriptive Study of Recombinant Human Insulin-Like Growth Factor for Osteoporosis in Werner's Syndrome

Who this might be right for
Werner's Syndrome
National Center for Research Resources (NCRR) 1
Not applicableLooking for participantsNCT07391293
What this trial is testing

Genetic Aortic Diseases and Cardiovascular Complications. An Observational Survey of Risk Factors.

Who this might be right for
Thoracic Aortic Aneurysm (TAA)
IRCCS Policlinico S. Donato 208
Testing effectiveness (Phase 2)Ended earlyNCT00004418
What this trial is testing

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Who this might be right for
Adrenoleukodystrophy
Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 126
Not applicableLooking for participantsNCT07067827
What this trial is testing

Self-questionnaire in Osteoporosis

Who this might be right for
Osteoporosis
CHU de Quebec-Universite Laval 58
Testing effectiveness (Phase 2)Study completedNCT00004396
What this trial is testing

Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias

Who this might be right for
Porphyria
National Center for Research Resources (NCRR) 32
Not applicableLooking for participantsNCT05497518
What this trial is testing

The KidneyCARE (Community Access to Research Equity) Study

Who this might be right for
Chronic Kidney DiseaseDialysisKidney Transplant
Tufts Medical Center 50,000
Not applicableLooking for participantsNCT04046796
What this trial is testing

Genetic Causes of Discrepant Clinic in Monogenic Twins

Who this might be right for
Rare DiseasesGenetic Predisposition to Disease
University Hospital Tuebingen 50
Not applicableNot Yet RecruitingNCT07741747
What this trial is testing

Impact of Ultra-fast Genetic Diagnosis of Familial Lymphohistiocytosis on the Time to Bone Marrow Transplantation and Overall Survival

Who this might be right for
Familial LymphohistiocytosisLymphohistiocytosis
Assistance Publique Hopitaux De Marseille 240
Early research (Phase 1)Study completedNCT00004809
What this trial is testing

Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia

Who this might be right for
Familial Hypercholesterolemia
National Center for Research Resources (NCRR) 5
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 applicableStudy completedNCT02985710
What this trial is testing

Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan

Who this might be right for
Small Fiber NeuropathyFabry DiseaseEhlers Danlos Syndrome+1 more
Massachusetts General Hospital 102
Not applicableLooking for participantsNCT02705846
What this trial is testing

Analysing Outcomes After Prostate Cancer Diagnosis and Treatment in Carriers of Rare Germline Mutations

Who this might be right for
Prostate Cancer
Institute of Cancer Research, United Kingdom 4,260
Not applicableStudy completedNCT00007046
What this trial is testing

Genetic Study of Patients and Families With Diaphyseal Medullary Stenosis With Malignant Fibrous Histiocytoma of the Bone

Who this might be right for
StenosisHistiocytoma
National Center for Research Resources (NCRR)
Testing effectiveness (Phase 2)Study completedNCT00004359
What this trial is testing

Phase II Pilot Study of Extracorporeal Phototherapy for Epidermolysis Bullosa Acquisita

Who this might be right for
Epidermolysis Bullosa Acquisita
National Center for Research Resources (NCRR) 10
Not applicableStudy completedNCT00004361
What this trial is testing

Study of the Relationship Between Calcium Levels and Intact Parathyroid Hormone (iPTH) in Adults With Repaired or Palliated Conotruncal Cardiac Defects

Who this might be right for
HypoparathyroidismTetralogy of FallotPulmonary Valve Stenosis+3 more
National Center for Research Resources (NCRR) 150
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