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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Absorption, Distribution, Metabolism, and Excretion of 14C-VX-407 After a Single Oral DoseAbnormalities, Multiple, Ciliopathies, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Urogenital Diseases, autosomal dominant polycystic kidney disease, cystic kidney disease, hereditary disease, kidney disorder, polycystic kidney disease, urinary system disorderNot yet recruitingPhase 12026-10-09NCT07868926
Treatment of Low-flow Vascular Malformations With Bleomycin Electrosclerotherapy (BEST)Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, vascular malformationRecruitingPhase 22026-10-09NCT07579962
The Corrective Power of Subtalar Arthrodesis With Mini/Medial Bone Block (SAMBB) in Patients Affected by Progressive Collapsing Foot Deformity (PCFD): Analysis Using Weight-bearing Cone Beam CT.Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Foot Deformities, Foot Deformities, Acquired, Foot Deformities, Congenital, Limb Deformities, Congenital, Lower Extremity Deformities, Congenital, Musculoskeletal Abnormalities, clubfoot, flatfoot, musculoskeletal system disorderNot yet recruitingNot applicable2026-10-09NCT07515170
A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADSCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Sex Chromosome Disorders, X-linked disease, autism, autism spectrum disorder, chromosomal disorder, fragile X syndrome, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorder, neurodevelopmental disorder, pervasive developmental disorder, psychiatric disorderNot yet recruitingPhase 2 / Phase 32026-10-09NCT07439510
Clinical Genetics Branch Eligibility Screening SurveyAbnormalities, Multiple, Bone Marrow Failure Disorders, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Costello syndrome, Craniofacial Abnormalities, DNA repair disease, Fanconi anemia, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Legius syndrome, Li-Fraumeni syndrome, Musculoskeletal Abnormalities, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, Thoracic Neoplasms, anemia, aplastic anemia, bone marrow disorder, cardiofaciocutaneous syndrome, chordoma, hematologic disorder, hereditary disease, hereditary neoplastic syndrome, inherited aplastic anemia, lung neoplasm, melanocytic neoplasm, melanoma, metabolic disease, mixed neoplasm, musculoskeletal system disorder, neoplasm, neuroendocrine neoplasm, primitive neuroectodermal tumor, pulmonary blastoma, respiratory tract neoplasm, skin disorder, skin neoplasm, susceptibility to familial cutaneous melanomaNot yet recruitingNot stated2026-10-09NCT07005297
Effect of Sacubitril-Valsartan on Cardiac Structure and FunctionCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, congenital heart disease, heart disorderRecruitingPhase 32026-10-09NCT06693674
Learning by Heart: The Effectiveness of an EF Training Program for Pre-schoolers With Critical CHDCardiovascular Abnormalities, Cognition Disorders, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, cognitive disorder, congenital heart disease, heart disorder, psychiatric disorderActive, not recruitingNot applicable2026-10-09NCT06267430
A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway ImpairmentAbnormalities, Multiple, Body Weight, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Froelich syndrome, Imprinting Disorders, Male Urogenital Diseases, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Overweight, Parkes Weber syndrome, Pathological Conditions, Signs and Symptoms, Prader-Willi syndrome, Signs and Symptoms, Urogenital Abnormalities, Urogenital Diseases, chromosomal disorder, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, gonadal dysgenesis, hereditary disease, hypogonadism, intellectual disability, nervous system disorder, nutritional disorder, obesity disorder, overnutritionActive, not recruitingPhase 1 / Phase 22026-10-09NCT06239116
Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Lymphatic Abnormalities, Neoplasm, Lymphatic Tissue, Neoplasms by Histologic Type, cystic hygroma, lymphangioma, lymphatic system disorder, neoplasmRecruitingPhase 2 / Phase 32026-10-09NCT05948943
Merit WRAPSODY™ Endoprosthesis for Treatment of Stenosis or Occlusion (WRAP)Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fistula, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Vascular Fistula, arteriovenous hemangioma/malformation, cardiovascular disorder, congenital arteriovenous fistula, vascular disorder, vascular malformationActive, not recruitingNot stated2026-10-09NCT05062291
Echocardiographic Assessment of Valvular Dysfunction in Children With CHDCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, congenital heart disease, heart disorder, heart valve disorderNot yet recruitingNot stated2026-10-08NCT07866924
RVOT Stenting Versus Surgical RV-PA Conduit in Cyanotic AdultsCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Ventricular Outflow Obstruction, Ventricular Outflow Obstruction, Right, cardiovascular disorder, congenital heart disease, heart disorderNot yet recruitingNot stated2026-10-08NCT07866911
UltraTrac for Cardiac Output Monitoring in Congenital Heart DiseaseCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, congenital heart disease, heart disorderNot yet recruitingNot applicable2026-10-08NCT07866573
Migraine Headaches Elimination With Patent Foramen Ovale-Directed TherapyCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, atrial septal defect, brain disorder, cardiovascular disorder, central nervous system disorder, congenital heart disease, headache disorder, heart disorder, heart septal defect, hypnic headache, migraine disorder, nervous system disorder, patent foramen ovaleRecruitingPhase 32026-10-08NCT07629635
Observational Study of VYKAT™ XR in Patients With Prader-Willi SyndromeAbnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Overweight, Prader-Willi syndrome, chromosomal disorder, hereditary disease, intellectual disability, nervous system disorder, nutritional disorder, obesity disorder, overnutritionEnrolling by invitationNot stated2026-10-08NCT07450664
Pediatric Evaluation and Registry for Liver Cholestasis in CanadaAbnormalities, Multiple, Alagille syndrome, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, bile duct disorder, biliary tract disorder, cardiovascular disorder, cholestasis, congenital heart disease, hereditary disease, intrahepatic cholestasis, liver disorder, progressive familial intrahepatic cholestasis type 1RecruitingNot stated2026-10-08NCT07411716
A Study of ROC-101 in Patients With Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated With Interstitial Lung Disease (ILD-PH) (ROCSTAR STUDY)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, central nervous system malformation, hereditary disease, hereditary sensory and autonomic neuropathy, hypertensive disorder, inherited neurodegenerative disorder, interstitial lung disease, lung disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, polyneuropathy, pulmonary arterial hypertension, pulmonary hypertension, respiratory system disorder, vascular disorderRecruitingPhase 22026-10-08NCT07175038
ES Catheter vs Cryoablation After Pectus SurgeryBone Diseases, Developmental, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Musculoskeletal Abnormalities, bone disorder, musculoskeletal system disorder, pectus excavatumRecruitingNot applicable2026-10-08NCT06682208
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)Abnormalities, Multiple, Alagille syndrome, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, bile duct disorder, biliary tract disorder, cardiovascular disorder, cholestasis, congenital heart disease, hereditary disease, intrahepatic cholestasis, liver disorder, progressive familial intrahepatic cholestasis type 1RecruitingNot stated2026-10-08NCT06193928
Prospective Study for the Evaluation of the Cementless Anatomic Femoral Stem Minimax -LaunayCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hip Dislocation, Congenital, Musculoskeletal Abnormalities, Necrosis, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, arthritic joint disease, arthropathy, autoimmune disease, avascular necrosis, bone disorder, bone fracture, connective tissue disorder, developmental dysplasia of the hip, immune system disorder, injury, musculoskeletal system disorder, osteonecrosis, rheumatic disorder, rheumatoid arthritisActive, not recruitingNot stated2026-10-08NCT06012656
French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan SyndromeCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Musculoskeletal Abnormalities, Noonan syndrome, Skin and Connective Tissue Diseases, cardiovascular disorder, congenital heart disease, connective tissue disorder, heart disorder, musculoskeletal system disorderEnrolling by invitationNot stated2026-10-08NCT05308927
Comprehensive Study of Post-surgical Pain After Pectus or Spine SurgeryBone Diseases, Developmental, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Musculoskeletal Abnormalities, Neurologic Manifestations, Pain, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Postoperative Complications, Postoperative Pain, Signs and Symptoms, Spinal Curvatures, bone disorder, musculoskeletal system disorder, pectus excavatum, scoliosis, vertebral column disorderActive, not recruitingNot applicable2026-10-08NCT04031716
Postoperative and Chronic Pain Genetic Spine and Pectus Surgery StudyBone Diseases, Developmental, Chronic Pain, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Kyphosis, Musculoskeletal Abnormalities, Neurologic Manifestations, Pain, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Postoperative Complications, Postoperative Pain, Signs and Symptoms, Spinal Curvatures, bone disorder, musculoskeletal system disorder, pectus excavatum, scoliosis, vertebral column disorderActive, not recruitingNot stated2026-10-08NCT02998138
Pathogenesis and Genetics of Microphthalmia, Anophthalmia and Uveal Coloboma (MAC)Anophthalmos, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Abnormalities, Eye Diseases, Hereditary, coloboma, eye disorder, microphthalmiaRecruitingNot stated2026-10-08NCT01778543
MRI Study of Musculoskeletal FunctionCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, arthropathy, musculoskeletal system disorder, patellofemoral pain syndromeRecruitingNot stated2026-10-08NCT00051857