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Results for “Rare Disorders”

979 trials

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Showing 20 of 979 results

Early research (Phase 1)Study completedNCT00004828
What this trial is testing

Liothyronine in Children With Single Ventricle Congenital Cardiac Malformations Undergoing the Fontan Procedure

Who this might be right for
Tricuspid AtresiaHeart Defects, Congenital
FDA Office of Orphan Products Development 28
Not applicableUnknownNCT00006128
What this trial is testing

Study of Neurobehavioral Outcome in Children or Adolescents With Closed Head Injuries

Who this might be right for
Head Injuries, Closed
National Center for Research Resources (NCRR) 374
Testing effectiveness (Phase 2)Study completedNCT00004658
What this trial is testing

Phase II Study of Ribose, Uridine, and Thymidine for a Complex Syndrome Involving Excessive 5'-Nucleotidase Activity

Who this might be right for
5'-Nucleotidase Syndrome
National Center for Research Resources (NCRR)
Not applicableNot Yet RecruitingNCT06412718
What this trial is testing

Validation of Human Drugs Target of Repurposed Drugs and Novel Therapies

Who this might be right for
Rare DiseasesAniridiaNeurotrophic Keratopathy+5 more
IRCCS Ospedale San Raffaele 274
Large-scale testing (Phase 3)Ended earlyNCT04612790
What this trial is testing

Investigate the Use of Benralizumab in Patients With Bullous Pemphigoid.

Who this might be right for
Bullous Pemphigoid
AstraZeneca 67
Not applicableUnknownNCT04152876
What this trial is testing

Functional Genomics of Rare Genetic Diseases: Realization of Innovative Tools With High Diagnostic Power

Who this might be right for
Rare Diseases
Neuromed IRCCS 300
Not applicableLooking for participantsNCT07072143
What this trial is testing

An International Study on Pediatric Patients With Rare Tumors.

Who this might be right for
Paraganglioma/ PhaeochromocytomaMelanoma and Other Malignant Neoplasms of SkinGastrointestinal Stromal Tumor (GIST)+11 more
Azienda Ospedaliera di Padova 6,250
Testing effectiveness (Phase 2)UnknownNCT04553887
What this trial is testing

Almonertinib as Upfront Treatment for Uncommon EGFR Mutation Harboring Non-Small-Cell Lung Cancer Patients: A Multicenter, Open-Label, Phase II Trial

Who this might be right for
Non Small Cell Lung Cancer
Tianjin Medical University Cancer Institute and Hospital 53
Not applicableUnknownNCT02588638
What this trial is testing

Next Generation Sequencing Diagnostics - On the Road to Rapid Diagnostics for Rare Diseases

Who this might be right for
Movement DisorderCognitive Decline
University Hospital Tuebingen 100
Not applicableStudy completedNCT00004825
What this trial is testing

Short Term Study of Recombinant Human Insulin-like Growth Factor I in Children With Hyperinsulinism

Who this might be right for
Hyperinsulinism
FDA Office of Orphan Products Development 10
Not applicableStudy completedNCT00010426
What this trial is testing

Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis

Who this might be right for
Cystinosis
FDA Office of Orphan Products Development 30
Large-scale testing (Phase 3)Study completedNCT00004297
What this trial is testing

Phase III Randomized Study of Diazepam Vs Lorazepam Vs Placebo for Prehospital Treatment of Status Epilepticus

Who this might be right for
Status Epilepticus
National Institute of Neurological Disorders and Stroke (NINDS) 210
Not applicableLooking for participantsNCT06796920
What this trial is testing

Epidural Spinal Cord Stimulation for Lower-limb Impairment in Adrenomyeloneuropathy

Who this might be right for
Adrenomyeloneuropathy Without Cerebral Involvement
Third Military Medical University 10
Not applicableStudy completedNCT03669380
What this trial is testing

Superior Mesenteric Artery Dissection

Who this might be right for
Vascular Diseases
Tongji Hospital 57
Large-scale testing (Phase 3)Study completedNCT00004646
What this trial is testing

Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
National Center for Research Resources (NCRR) 20
Not applicableUnknownNCT04137718
What this trial is testing

Multicenter Observational Study of Chinese Non-Small Cell Lung Cancer (NSCLC) Patients With Rare Driver Gene Mutation

Who this might be right for
ChineseNon-Small Cell Lung Cancer
Shanghai Pulmonary Hospital, Shanghai, China 50,000
Not applicableStudy completedNCT03067142
What this trial is testing

Proteomics of Primary Hyperoxaluria Type 1

Who this might be right for
Primary Hyperoxaluria Type 1
Ann & Robert H Lurie Children's Hospital of Chicago 93
Not applicableStudy completedNCT00004781
What this trial is testing

Oral Manifestations of Human Immunodeficiency Virus Infection in High Risk Groups

Who this might be right for
HIV InfectionsCandidiasis, Oral
National Institute of Dental and Craniofacial Research (NIDCR) 250
Not applicableUnknownNCT05955794
What this trial is testing

Vocal Pattern Assessment as a New Key to Identifying Rare Syndromes

Who this might be right for
Rare DiseasesCongenital Disorders
Fondazione Policlinico Universitario Agostino Gemelli IRCCS 500
Early research (Phase 1)UnknownNCT00004307
What this trial is testing

Study of Treatment and Metabolism in Patients With Urea Cycle Disorders

Who this might be right for
Amino Acid Metabolism, Inborn Errors
National Center for Research Resources (NCRR) 66
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