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Results for “Rare Abnormalities”

425 trials

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Showing 20 of 425 results

Not applicableLooking for participantsNCT04151342
What this trial is testing

CAnadian CAncers With Rare Molecular Alterations (CARMA) - Basket Real-world Observational Study (BROS)

Who this might be right for
CancerMalignancies MultipleMalignant Solid Tumor+7 more
University Health Network, Toronto 5,500
Large-scale testing (Phase 3)Study completedNCT03936777
What this trial is testing

Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome

Who this might be right for
Dravet SyndromeLennox Gastaut SyndromeEpileptic Encephalopathy
Zogenix, Inc. 412
Not applicableStudy completedNCT01914172
What this trial is testing

Health Needs of Patients With Kallmann Syndrome

Who this might be right for
Kallmann SyndromeCongenital Hypogonadotropic HypogonadismIdiopathic Hypogonadotropic Hypogonadism
Centre Hospitalier Universitaire Vaudois 249
Early research (Phase 1)Study completedNCT00004806
What this trial is testing

Phase I Study of Liposome-Mediated Gene Transfer in Patients With Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
University of Alabama at Birmingham 9
Not applicableStudy completedNCT00004773
What this trial is testing

Study of Cardiac and Paroxysmal Abnormalities in Rett Syndrome

Who this might be right for
Rett Syndrome
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 95
Not applicableStudy completedNCT02793063
What this trial is testing

Osteogenesis Imperfecta (OI) Quality of Life Survey Pilot Project 2

Who this might be right for
Osteogenesis ImperfectaBrittle Bone Disorders
University of South Florida 300
Early research (Phase 1)Study completedNCT00004471
What this trial is testing

Phase I Pilot Study of Gene Therapy for Cystic Fibrosis Using Cationic Liposome Mediated Gene Transfer

Who this might be right for
Cystic Fibrosis
University of Alabama at Birmingham 9
Not applicableLooking for participantsNCT06969209
What this trial is testing

Brain Aging in Phenylketonuria

Who this might be right for
Phenylketonuria (PKU)
Insel Gruppe AG, University Hospital Bern 90
Not applicableEnrolling By InvitationNCT07417267
What this trial is testing

Clean Air for Rare MUcociliary Clearance dIsorders

Who this might be right for
Primary Ciliary Dyskinesia (PCD)Cystic Fibrosis (CF)
University of Cyprus 62
Not applicableEnrolling By InvitationNCT03836300
What this trial is testing

Parent and Infant Inter(X)Action Intervention (PIXI)

Who this might be right for
Fragile X SyndromeAngelman SyndromePrader-Willi Syndrome+11 more
RTI International 120
Not applicableLooking for participantsNCT07206095
What this trial is testing

Integrative Diagnosis for SCD and Other RADs

Who this might be right for
Sickle Cell DiseaseThalassaemiaCongenital Dyserythropoietic Anemia (CDA)+6 more
Hospital Universitari Vall d'Hebron Research Institute 200
Not applicableLooking for participantsNCT05865119
What this trial is testing

OPTImal Treatment of Sinus VENOSUS Defect

Who this might be right for
Sinus Venosus DefectCongenital Heart DiseaseSinus Venosus Atrial Septum Defect
Centre Chirurgical Marie Lannelongue 60
Not applicableStudy completedNCT00004696
What this trial is testing

Study of Morphine in Postoperative Infants to Allow Normal Ventilation

Who this might be right for
Infant, Newborn, DiseasesPain
Seattle Children's Hospital 100
Not applicableStudy completedNCT05917236
What this trial is testing

Biliary Atresia With Rare Associations, a Case Report

Who this might be right for
BA - Biliary Atresia
Sarah Magdy Abdelmohsen 1
Not applicableStudy completedNCT02116764
What this trial is testing

Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995

Who this might be right for
Chronic Granulomatous Disease
National Institute of Allergy and Infectious Diseases (NIAID) 137
Testing effectiveness (Phase 2)Looking for participantsNCT06525636
What this trial is testing

A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia

Who this might be right for
X-linked Hypophosphatemia
Kyowa Kirin Co., Ltd. 39
Large-scale testing (Phase 3)Study completedNCT01000961
What this trial is testing

Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis

Who this might be right for
Cystinosis
Amgen 43
Not applicableUnknownNCT01954953
What this trial is testing

Clinical and Genetic Examination of Usher Syndrome Patients' Cohort in Europe

Who this might be right for
Usher Syndrome
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts 100
Not applicableStudy completedNCT05199402
What this trial is testing

Clinical Trial Data Set Re-use With Statistical Methodologies Tailored for Clinical Trials in Rare Diseases

Who this might be right for
Tuberous SclerosisEpilepsy
Imagine Institute 10
Not applicableLooking for participantsNCT06677866
What this trial is testing

Group CBT in Adolescents With Fragile X Syndrome and in Adolescents With Autism Spectrum Disorder

Who this might be right for
Fragile X Syndrome (FXS)Autism Spectrum Disorder
Bambino Gesù Hospital and Research Institute 20
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