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Condition: Multiple Congenital Anomaly Synced from ClinicalTrials.gov · July 29, 2026

Recruiting studies: Multiple Congenital Anomaly

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

DFT383 in Pediatric Participants With Nephropathic Cystinosis
Phase 1/Phase 2 · 4 location(s) · Novartis Pharmaceuticals · NCT06910813Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MY
Phase 1/Phase 2 · 10 location(s) · Tenaya Therapeutics · NCT05836259Autosomal Dominant Polycystic Kidney Disease (ADPKD) Study
Phase N/A · 4 location(s) · Children's Hospital of Philadelphia · NCT04338048Natural History of Photoreceptor Degeneration in USH1B: Clinical Parameters and Validation of Functional Vision Tests in
Phase N/A · 1 location(s) · Centre Hospitalier National d'Ophtalmologie des Quinze-Vingt · NCT07278843Natural History Study of Parathyroid Disorders
Phase N/A · 1 location(s) · National Institute of Diabetes and Digestive and Kidney Dise · NCT04969926The Effect of the HEP Approach in Infants With Down Syndrome
NA · 1 location(s) · Izgi Miray Demirbag · NCT07163780Balloon Inflation Time for Esophageal Strictures (BITES): A Randomized Multi-Center Study
NA · 2 location(s) · Boston Children's Hospital · NCT07100379Animal Fun Program and Kinesthetic Development in Down Syndrome
NA · 1 location(s) · Riphah International University · NCT07579559Adapting RDAD for DS
NA · 1 location(s) · University of Kansas Medical Center · NCT07425769Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor
Phase 2 · 84 location(s) · Merck Sharp & Dohme LLC · NCT04924075Effects of the Otago Exercise Program on Balance, Endurance, and Motor Coordination in Children With Down Syndrome
NA · 1 location(s) · Riphah International University · NCT07484464Long-term Safety and Effectiveness of Growth Hormone With GHD, TS, CRF, SGA , ISS and PWS in Children
Phase N/A · 1 location(s) · LG Chem · NCT01604395Osteogenesis Imperfecta Trial of AGA2115 for ADUlts With COL1A1 and/or COL1A2 GeNetic Variations (IDUN)
Phase 2 · 26 location(s) · Angitia Incorporated Limited · NCT07062588International Pachyonychia Congenita Research Registry
Phase N/A · 1 location(s) · Pachyonychia Congenita Project · NCT02321423ARPKD Database Study
Phase N/A · 6 location(s) · Children's Hospital of Philadelphia · NCT01401998Propranolol and Von Hippel-Lindau Disease
NA · 1 location(s) · Assistance Publique - Hôpitaux de Paris · NCT05424016The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2
Phase 3 · 7 location(s) · Lupin Ltd. · NCT06523400A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Phase 2/Phase 3 · 5 location(s) · AO GENERIUM · NCT05208281A Study of Pitolisant in Patients With Prader-Willi Syndrome
Phase 3 · 54 location(s) · Harmony Biosciences Management, Inc. · NCT06366464A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants Wi
Phase 2 · 23 location(s) · ReCode Therapeutics · NCT06237335Post-market Surveillance Study of the BD® WavelinQ™ EndoAVF System
NA · 17 location(s) · C. R. Bard · NCT04634916Effects of Exogenous Ketosis on Proteinuria and Renal Function
NA · 1 location(s) · Gødstrup Hospital · NCT06867471A Single and Multiple Ascending Dose Trial of LT-002-158 Tablets in Healthy Adult Volunteers
Phase 1 · 1 location(s) · Leadingtac Pharmaceutical (Shaoxing) Co., Ltd. · NCT06082323A Study to Evaluate the Safety and Tolerability of Multiple Dose of IBI3002 in Patients With Atopic Dermatitis
Phase 1 · 1 location(s) · The First Affiliated Hospital of Soochow University · NCT07015762Prospective Research Rare Kidney Stones (ProRKS)
Phase N/A · 11 location(s) · Mayo Clinic · NCT02780297Screening for Endolymphatic Sac Tumours (ELSTs) in Von Hippel-Lindau (vHL) Patients
Phase N/A · 1 location(s) · Marie Luise Bisgaard, MD · NCT02420067NYSCF Scientific Discovery Biobank
Phase N/A · 1 location(s) · New York Stem Cell Foundation Research Institute · NCT06203106Effects of Jump Rope on Navicular Drop in Down Syndrome
NA · 1 location(s) · Riphah International University · NCT07428863Aerobic Exercise With and Without Low Calorie Diet on Adiposity and BMI of Down Syndrome
NA · 1 location(s) · Riphah International University · NCT06943170Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Phase 2 · 11 location(s) · Darcy Krueger · NCT05104983
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.