Congenital, Hereditary, and Neonatal Diseases and Abnormalities: all 110 studies

Page 1 of 2. Open to participants first (20), then studies with results posted, then the rest; newest registration first within each.

• A Phase 3 Evaluation of Sutacimig in Glanzmann ThrombastheniaRecruitingNCT07935356Phase 3
• Fetal Embolization for Severe Vein of Galen Malformation - A Feasibility and Safety TrialNot yet recruitingNCT07927725Not applicable
• EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)Not yet recruitingNCT07894887Phase 1/2
• Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIBNot yet recruitingNCT07818759Phase 1/2
• Vision Baby : The Measurement of Vital Signs in Neonates by Lifelight® Software in Comparison to the Standard of CareRecruitingNCT07799870
• A Long-Term Observational Study of Patients With FucosidosisRecruitingNCT07615400
• Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)RecruitingNCT07608432Phase 3
• Study of Zodasiran in Adolescent Participants With Homozygous Familial HypercholesterolemiaRecruitingNCT07473843Phase 3
• Prenatal Transplantation for Fetuses With Fanconi AnemiaNot yet recruitingNCT07408583Phase 1/2
• Efficacy and Safety of SIL-8301 for Control of Hemolysis in a Uniform Sickle Cell Disease EndotypeRecruitingNCT07282210Phase 2
• Azithromycin and Ampicillin for Late PPROMRecruitingNCT07183462Phase 4
• Prospective Registry for Long-term Outcomes Following FETO in Severe Left and Right CDHRecruitingNCT07166172Not applicable
• Clinical Application of Artificial Intelligence in New Borns With Cleft Lip and PalateRecruitingNCT06970158Not applicable
• Multimodal Biocollection Linked to the French Register of Intracranial AneurysmsRecruitingNCT06689410Not applicable
• Vocal Pattern Assessment as a New Key to Identifying Rare SyndromesNot yet recruitingNCT05955794Not applicable
• Vaccinations and People With DisabilitiesRecruitingNCT05749419
• International Study of Cerebral Oxygenation and Electrical Activity During Major Neonatal SurgeryRecruitingNCT05673499
• Study of Late-foetal Human Organ DevelopmentRecruitingNCT04661059
• Measuring Adverse Pregnancy and Newborn Congenital OutcomesRecruitingNCT04405700
• Genome Medical Sequencing for Gene DiscoveryRecruitingNCT01087320
• Subcutaneous Hydrocortisone Children With Congenital Adrenal HyperplasiaCompletedNCT03718234Phase 1Results posted
• Low-dose Epinephrine Infusion Tests in Adolescent and Pediatric PatientsTerminatedNCT00548886Not applicableResults posted
• Pacemaker Therapy in Adults With Congenital Heart DefectsCompletedNCT00303511Results posted
• Spironolactone in Patients With Single Ventricle HeartCompletedNCT00211081Not applicableResults posted
• Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett SyndromeActive, not recruitingNCT07480564Phase 3
• Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical TrialActive, not recruitingNCT07226622
• Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)Active, not recruitingNCT06721871Phase 2/3
• Evaluation of Bili-ruler, a Low-cost, Plastic Icterometer for Community-based Screening of Jaundice in NewbornsCompletedNCT06687746
• Nasal High Frequency in Preterm NeonatesCompletedNCT06687083Not applicable
• Melatonin for Pulmonary Hypertension in Full Term NeonatesCompletedNCT06685679Phase 3
• Investigating How Childhood Tumours and Congenital Disease DevelopStatus unknownNCT06584877
• Learning Skills in Shoulder Dystocia Delivery Using Simulator ModelsCompletedNCT05921045
• TAAI Erasmus Research Initiative to Fight CF: Monitoring Inflammation in CF Lung Disease Into a New EraCompletedNCT05752019
• The Effect of Birth Ball Exercise on Labor Pain, Delivery Duration, Birth Comfort, and Birth SatisfactionCompletedNCT05711472Not applicable
• A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)Active, not recruitingNCT05606614Phase 3
• Learning Skills in Young Specialists and Residents in Operative Vaginal Delivery Using Simulator ModelsCompletedNCT05605626
• The Effect of Feng Shui Delivery Room Design on Maternal and Infant HealthCompletedNCT05468658Not applicable
• Flexible URS in Management of Renal Stones in Anomalous KidneyStatus unknownNCT05299996Not applicable
• Turkish Affordances in the Home Environment for Motor Development-Infant Scale (AHEMD-IS)CompletedNCT04556487
• A Review of Surgical Management of Congenital Pulmonary Airway Malformations (CPAM): A Decade of ExperienceCompletedNCT04449614
• Congenital Disability and Rehabilitation: the TOPS ProgramCompletedNCT04293705Not applicable
• Parametric Mapping in Paediatric Magnetic Resonance ImagingStatus unknownNCT04068987
• D-tecting Disease - From Exposure to Vitamin D During Critical Periods of LifeCompletedNCT03330301
• Impact of Percutaneous Laparoscopic Assisted Internal Ring Ligation During Lap OrchiopexyStatus unknownNCT03291678Not applicable
• HaemoDialysis Interventions to REduce Multi-Organ Dysfunction and Effect on Quality of Life Assessed by MRI ScanningCompletedNCT03280901Not applicable
• Supported Exercise Programme for Adults With Congenital Heart DiseaseCompletedNCT03262168Not applicable
• OPTI-Prem: Optimising Neonatal Service Provision for Preterm Babies Born Between 27 and 31 Weeks of Gestation in EnglandCompletedNCT02994849
• Kids Intestinal Dysfunction in Congenital Heart Disease: the Kind Heart StudyCompletedNCT02824705
• Tracheal Occlusion To Accelerate Lung Growth (TOTAL) Trial for Severe Pulmonary HypoplasiaCompletedNCT01240057Phase 2/3
• Pilot Study of Etanercept (Enbrel) in Children With Fanconi AnemiaCompletedNCT00965666Early Phase 1
• Biomarkers for Detection of Brain IschemiaCompletedNCT00490295
• Predictors for Pulmonary Valve Replacement - Anatomic and Hemodynamic Using MRICompletedNCT00489788
• Femoral Arterial CannulationTerminatedNCT00486096
• Review of Descending Aortic Flow Reversal in Total Anomalous Pulmonary Venous ConnectionTerminatedNCT00486070
• Daclizumab Experience in Pediatric Heart TransplantTerminatedNCT00486057
• Chlorhexidine Swabs Effectiveness in Reducing Blood Stream InfectionsTerminatedNCT00485992
• Repeat Sternotomy for PediatricsCompletedNCT00485745
• Racial Distribution of Heterotaxy SyndromeTerminatedNCT00485654
• Comparison of NIRS and Serum Lactate to Predict Poor Post-operative OutcomesCompletedNCT00485524
• Pulmonary Hypertension in Trisomy 21 PatientsCompletedNCT00478296
• A Retrospective Review - Anti-HLA AntibodiesWithdrawnNCT00460824
• Adult Congenital Heart Disease SurgeryCompletedNCT00446160
• Adult Pulmonary Valve Replacement: A Simple and Reproducible TechniqueTerminatedNCT00384163
• Exercise in Chronically Paced ChildrenCompletedNCT00374608
• Respiratory Motion Analysis in Children With MRITerminatedNCT00367354
• Patients With NMDA Biomarker Data Following Cardiac SurgeryTerminatedNCT00366886
• Computer Modeling of Congenital Heart DiseaseCompletedNCT00366847
• Retrospective Review of the Outcomes of Newborns With Genetic AbnormalitiesCompletedNCT00366821
• Neonates With Neurological ComplicationsCompletedNCT00366561
• Frequency of Accessing Central Lines for Blood SamplesTerminatedNCT00366314
• Re-operative Surgery in Children:A Technique for Sternal Re-EntryCompletedNCT00328146
• Hypoxemia in Infants After Palliative SurgeryCompletedNCT00327977
• Infant Weight Gain With Trisomy 21 and CAVCTerminatedNCT00327951
• Home Inotropic Therapy in ChildrenTerminatedNCT00327899
• Managing Cyanosis Following Bidirectional Glenn OperationTerminatedNCT00327886
• Palliative Care of Children With End-Stage Heart FailureTerminatedNCT00327782
• Effects of Beta-adrenergic in Adults w/Transposition of Great Arteries on Systemic Ventricular FunctionCompletedNCT00313352
• A Study to Evaluate Folate Levels in Women Taking Oral ContraceptivesWithdrawnNCT00301587Phase 3
• Growth in Children With HLHSTerminatedNCT00277992
• 3D Echocardiography Managing Infantile Pompe's DiseaseTerminatedNCT00277979
• Cath Lab Performance ImprovementWithdrawnNCT00277966
• MRI Review of Tracheal DimensionsTerminatedNCT00277953
• Aortic Arch ReconstructionTerminatedNCT00277940
• Mild Valvar Pulmonary StenosisTerminatedNCT00277927
• MRI Assessment of RV Function: Patients With TOF or Aortic CoarctationCompletedNCT00277901
• Resynchronization of Left Ventricular Contraction After Reimplantation of Anomalous Left Coronary ArteryTerminatedNCT00268112
• Optimal Timing for Repair of Right-to-Left Shunt LesionsTerminatedNCT00268099
• Constrictive Pericarditis in the Adult Congenital Cardiac PopulationTerminatedNCT00268086
• Incidence of Neo-Aortic InsufficiencyTerminatedNCT00268073
• Infant Medical Records: Case Report ProposalTerminatedNCT00268060
• Pulmonary Resistance in Fontan Who Have Undergone Heart TransplantationTerminatedNCT00268047
• Left Ventricular Aneurysms in ChildrenTerminatedNCT00268034
• Automated Cardioverter Defibrillator in ChildrenCompletedNCT00268021
• Children and Adolescents Receiving Mechanical and Prosthetic ValvesTerminatedNCT00268008
• Ross Aortic Valve Replacement PatientsTerminatedNCT00267995
• Radiofrequency Ablation of Ectopic Atrial TachycardiaTerminatedNCT00267982
• Hypertrophic Myopathy in ChildrenTerminatedNCT00267462
• Biventricular Pacing in the Pediatric PopulationTerminatedNCT00267423
• Prevalence and Clinical Spectrum of the 22q11 DeletionTerminatedNCT00267397
• Outcomes of Children With Congenital Single Ventricle HeartCompletedNCT00266968

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