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Results for “RARE DISEASE”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Active Not RecruitingNCT06314490
What this trial is testing

Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A

Who this might be right for
Genetic Disease
University of California, San Diego 1
Testing effectiveness (Phase 2)Study completedNCT00004669
What this trial is testing

Phase II Pilot Study of Early Cortisol Replacement to Prevent Bronchopulmonary Dysplasia

Who this might be right for
Bronchopulmonary Dysplasia
National Center for Research Resources (NCRR) 40
Not applicableLooking for participantsNCT05990179
What this trial is testing

Genomic Uniformed-Screening Against Rare Disease In All Newborns

Who this might be right for
Early Onset Genetic Conditions With Near Complete Penetrance
Columbia University 100,000
Not applicableLooking for participantsNCT07102966
What this trial is testing

Genetic Study to Determine the Cause of Birth Defects in Newborns in Texas

Who this might be right for
Rare Diseases
Baylor College of Medicine 410
Not applicableStudy completedNCT00004753
What this trial is testing

Long-Term Study of Cerebral Glucose Metabolism in Huntington's Disease

Who this might be right for
Huntington's Disease
National Center for Research Resources (NCRR) 90
Very early researchEnded earlyNCT06860672
What this trial is testing

Clinical Trial of the Dual Vector Base Editor for the Treatment of the CHD3-R1025W Mutation

Who this might be right for
Developmental Delay DisorderIntellectual DisabilityRare Diseases
Yongguo Yu 1
Not applicableStudy completedNCT00004430
What this trial is testing

Randomized Study of Photodynamic Therapy Using Dihematoporphyrin in Patients With Corneal Neovascularization

Who this might be right for
Corneal Neovascularization
Medical College of Hampton Roads 150
Not applicableStudy completedNCT00005101
What this trial is testing

Study of Low Bone Mass in Premenopausal or Perimenopausal Women

Who this might be right for
Osteoporosis
National Center for Research Resources (NCRR)
Not applicableStudy completedNCT02740153
What this trial is testing

PCORI Urea Cycle Disorder Study

Who this might be right for
Urea Cycle Disorders
Children's National Research Institute 187
Not applicableNot Yet RecruitingNCT07799792
What this trial is testing

Implementation-effectiveness Trial of Mainstreaming of Clinical Genomic Sequencing for Rare Disease in Ontario, Canada

Who this might be right for
Evaluation of Mainstreamed Models of Genetic Service DeliveryGenetic DiseaseNeurodevelomental Disorders
The Hospital for Sick Children 100
Not applicableStudy completedNCT00004419
What this trial is testing

Study of Recombinant Human Insulin-Like Growth Factor I in Patients With Severe Insulin Resistance

Who this might be right for
Insulin ResistanceHyperglycemia
Beth Israel Deaconess Medical Center 18
Large-scale testing (Phase 3)Study completedNCT00634049
What this trial is testing

Isavuconazole in the Treatment of Renally Impaired Aspergillosis and Rare Fungi

Who this might be right for
AspergillosisInvasive Fungal Infections
Astellas Pharma Inc 149
Not applicableLooking for participantsNCT03161808
What this trial is testing

Rare CFTR Mutation Cell Collection Protocol (RARE)

Who this might be right for
Cystic Fibrosis
George Solomon 500
Not applicableUnknownNCT03347591
What this trial is testing

Rare Bleeding Disorders in the Netherlands

Who this might be right for
Rare Bleeding Disorders
Radboud University Medical Center 300
Not applicableStudy completedNCT00007033
What this trial is testing

Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver Disease

Who this might be right for
Alagille SyndromeCholestasisBiliary Atresia
National Center for Research Resources (NCRR) 25
Not applicableNot Yet RecruitingNCT06656247
What this trial is testing

Implementing Dynamic Consent for Rare Disease Patients

Who this might be right for
Rare Disorders
Istituto Ortopedico Rizzoli 50
Not applicableStudy completedNCT00004761
What this trial is testing

Establishment of the National Epidermolysis Bullosa Registry

Who this might be right for
Epidermolysis Bullosa
National Center for Research Resources (NCRR)
Large-scale testing (Phase 3)Looking for participantsNCT07668856
What this trial is testing

Cannabidiol as add-on Therapy for Children With Refractory Epilepsy (CBD-uN1que), a High-quality Individualized Approach: a Series of N-of-1 Trials

Who this might be right for
Refractory Epilepsy in ChildrenRefractory EpilepsyEpilepsy
UMC Utrecht 50
Not applicableStudy completedNCT02067754
What this trial is testing

Circulating Free DNA Analysis in Gastrointestinal Cancer, Genitourinary Cancer, Rare Cancer

Who this might be right for
Gastric Cancer
Samsung Medical Center 396
Not applicableActive Not RecruitingNCT05985122
What this trial is testing

New Analytic Tools for aHUS and C3G Diagnosis

Who this might be right for
Hemolytic-Uremic SyndromeMembranoproliferative GlomerulonephritisHealthy
Mario Negri Institute for Pharmacological Research 180
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