4093 completed, 1273 unknown, 1153 recruiting, 572 terminated. 2845 Not applicable, 1172 Phase 2, 645 Phase 1, 515 Phase 3, 274 Phase 4.
Follow Congenital Abnormalities: new trials by email Literature Review of Congenital Abnormalities trials Research Report on Congenital Abnormalities
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Parenting Program for Young Children With Spina Bifida Positive Parenting Program - Spina Bifida | Not applicable | Not yet recruiting | Phoenix Children's Hospital | 30 | 2028-01-01 | 2026-11-04 |
| Clinical Trial of a MEK Inhibitor for RASopathy-Associated Severe Infantile Hypertrophic Cardiomyopathy: Baby MERIT Trametinib - HCM, Noonan Syndrome and Other Rasopathy | Phase 3 | Not yet recruiting | Carelon Research | 25 | 2027-08-01 | 2026-09-14 |
| Epidermal Growth Factor Receptor Inhibition for Keratinopathies Erlotinib - Epidermolytic Ichthyosis, Palmoplantar Keratoderma | Phase 1 / Phase 2 | Not yet recruiting | Northwestern University | 44 | 2027-07-01 | 2026-08-03 |
| Continuous Passive Motion Device for Children With Arthrogryposis CPM device - Arthrogryposis | Not applicable | Not yet recruiting | Nemours Children's Clinic | 5 | 2027-06-01 | 2026-11-04 |
| Nano Silver Fluoride and Photobiomodulation Therapy for Hypersensitivity and Pain in MIH-Affected Molars Nano Silver Fluoride, Photobiomodulation Therapy, Pre-appointment analgesics - Molar-Incisor Hypomineralization (MIH) | Not applicable | Not yet recruiting | Ain Shams University | 45 | 2027-05-01 | 2026-09-17 |
| Indications and Evaluation of Microfat Grafting in Cleft Lip Nasal Deformities Microfat grafting - Cleft Lip and Nose | Not applicable | Not yet recruiting | Assiut University | 30 | 2027-05-01 | 2026-10-29 |
| Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A scAAV1.tMCK.NTF3 - Charcot-Marie-Tooth Neuropathy Type 1A | Phase 1 / Phase 2 | Withdrawn | Nationwide Children's Hospital | 2027-04-01 | 2026-07-29 | |
| TORCH-LATAM-003: Regional Advanced-Therapy Pathways for Congenital Heart Disease TORCH Congenital Advanced Therapies Pathway (TORCH-CATP) - Congenital Heart Disease, Advanced Heart Failure | Not applicable | Not yet recruiting | CardioVision International Tech, S.A.S. de C.V. | 360 | 2027-03-30 | 2026-10-13 |
| WSO BEAT STROKE Initiative Educational and implementation Neurocardiology tool, No educational or implementation tool will be provided - Stroke, PFO - Patent Foramen Ovale | Not applicable | Not yet recruiting | Luciano A. Sposato | 2400 | 2027-03-01 | 2026-09-18 |
| An Exercise and Lifestyle Programme for Adults With Vascular Ehlers-Danlos Syndrome: A Feasibility Study Co-designed exercise-based lifestyle intervention - Vascular Ehlers Danlos Syndrome | Not applicable | Not yet recruiting | Sheffield Hallam University | 40 | 2027-03-01 | 2026-07-13 |
| TORCH-LATAM: A Regional Pathway to Improve Transplant Evaluation for Congenital Heart Disease TORCH Integrated Transplant Access Pathway (TORCH-ITAP) - Congenital Heart Disease | Not applicable | Not yet recruiting | CardioVision International Tech, S.A.S. de C.V. | 600 | 2027-03-01 | 2026-10-13 |
| The Effect of Bitewing Radiography on Outcomes and Costs of Treating Caries in Permanent Teeth Visual-tactile caries examination, Visual-tactile caries examination and bilateral bitewing radiography - Dental Caries, Teeth Loss | Not applicable | Not yet recruiting | Jukka Leinonen | 250 | 2027-03-01 | 2026-10-20 |
| Topical TOR-582 Treatment of Epistaxis in HHT Topical sirolimus ointment - Hereditary Hemorrhagic Telangiectasia (HHT), Epistaxis | Phase 1 | Not yet recruiting | Columbia University | 27 | 2027-03-01 | 2026-11-06 |
| Fetal Embolization for Severe Vein of Galen Malformation - A Feasibility and Safety Trial Fetal embolization of severe Vein of Galen Malformation using a liquid embolic system - Vein of Galen Malformation | Not applicable | Not yet recruiting | Michael A Belfort | 10 | 2027-03-01 | 2026-11-10 |
| S-TiUltra Implant Study NobelReplace S-TiUltra, NobelReplace TiUnite - Missing Tooth/Teeth | Phase 4 | Not yet recruiting | University of Maryland, Baltimore | 30 | 2027-02-01 | 2026-11-02 |
| A Pilot Study to Examine the Efficacy and Tolerability of Sirolimus on Epidermal Nevi Lesions in Patients 6 Years Old and Above. sirolimus topical gel 0.2% - Epidermal Nevi | Early Phase 1 | Not yet recruiting | Northwestern University | 14 | 2027-02-01 | 2026-11-09 |
| A Study to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy of S247240 in Adult Male Participants With Fragile X Syndrome S247240 Low dose, S247240 Middle dose, S247240 High dose - Fragile X Syndrome (FXS) | Phase 2 | Not yet recruiting | Institut de Recherches Internationales Servier (I.R.I.S.) | 100 | 2027-01-25 | 2026-11-12 |
| Regenerative Medicine for Joint Hypermobility and Instability Dextrose 20% Prolotherapy, Autologous Platelet-Rich Plasma, Doxycycline Hyclate Injectable (Investigational) - Ehlers-Danlos Syndrome Hypermobility Type (hEDS), Ehlers-Danlos Syndrome (EDS) | Phase 1 / Phase 2 | Not yet recruiting | Manhattan Pain Medicine, PLLC | 100 | 2027-01-15 | 2026-10-06 |
| Mirdametinib to Reduce the Size of Large/Giant Congenital Melanocytic Nevi in Children, Adolescents and Young Adults Mirdametinib - LGCMN, Large and Giant Congenital Melanocytic Nevi | Phase 2 | Not yet recruiting | National Cancer Institute (NCI) | 15 | 2027-01-04 | 2026-10-29 |
| Bioengineered Penile Tissue Constructs for Irreversibly Damaged Penile Corpora Autologous tissue engineered corpora - Urologic Diseases, Male Urogenital Diseases | Phase 1 | Not yet recruiting | Wake Forest University Health Sciences | 10 | 2027-01-01 | 2026-10-23 |
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Heart Defects, Congenital (991) · Down Syndrome (433) · Leukemia, Myeloid, Acute (375) · Leukemia (249) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (231) · Arteriovenous Fistula (224) · Polycystic Kidney, Autosomal Dominant (184) · Hereditary Sensory and Autonomic Neuropathies (175) · Leukemia, Lymphocytic, Chronic, B-Cell (173) · Flatfoot (167) · Myeloproliferative Disorders (165) · Leukemia, Myeloid, Accelerated Phase (163) · Myelodysplastic Syndromes (161) · Lymphoma, Follicular (160)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8213) · Cardiovascular Diseases (3074) · Cardiovascular Abnormalities (2626) · Genetic Diseases, Inborn (2612) · Nervous System Diseases (2193) · Pathological Conditions, Signs and Symptoms (2113) · Heart Diseases (2071) · Musculoskeletal Diseases (1470) · Abnormalities, Multiple (1428) · Urogenital Diseases (1257) · Female Urogenital Diseases and Pregnancy Complications (1205) · Musculoskeletal Abnormalities (1177) · Neurologic Manifestations (1165) · Pathologic Processes (1163)
Cytarabine (95) · fludarabine phosphate (95) · Cyclophosphamide (84) · Blood Specimen Collection (82) · Peripheral Blood Stem Cell Transplantation (79) · Busulfan (69) · Tacrolimus (61) · Mycophenolic Acid (59) · Cyclosporine (56) · Sirolimus (55) · Surveys and Questionnaires (55) · Saline Solution (52) · Whole-Body Irradiation (50) · Sodium Chloride (49) · Antilymphocyte Serum (47)
Assiut University (128) · Cairo University (125) · Assistance Publique - Hôpitaux de Paris (116) · Boston Children's Hospital (83) · National Cancer Institute (NCI) (79) · Riphah International University (67) · Hospices Civils de Lyon (65) · Mayo Clinic (60) · Children's Hospital Medical Center, Cincinnati (58) · University of Colorado, Denver (53) · Fred Hutchinson Cancer Center (52) · Stanford University (50) · University Hospital, Montpellier (50) · The Hospital for Sick Children (49) · Emory University (43)
United States (3120) · France (893) · Canada (482) · China (452) · Italy (436) · United Kingdom (434) · Turkey (Türkiye) (397) · Germany (383) · Egypt (360) · Spain (326) · Netherlands (242) · Belgium (221) · Australia (177) · Denmark (161) · Brazil (151)
David Meeker, MD (11) · Forbes D Porter, M.D. (7) · Judith Karp (7) · Amy Paller, MD (6)
PMID 39356039, PMID 41385299, PMID 22587997, PMID 37250503, PMID 24383844, PMID 25527568, PMID 31375516, PMID 18035553, PMID 18424759, PMID 18557726, PMID 19066739, PMID 20236866, PMID 32499241, PMID 33112424, PMID 36184192, PMID 40938675, PMID 41348481, PMID 17284531, PMID 21306277, PMID 30936738, PMID 33159213, PMID 35029841, PMID 35080267, PMID 36184076, PMID 36191725, PMID 36978128, PMID 37349694, PMID 37979797, PMID 38837240, PMID 12084585, PMID 18000608, PMID 22065264, PMID 24586400, PMID 26554876, PMID 29023494, PMID 29718281, PMID 30935731, PMID 31014270, PMID 32034419, PMID 33395098, PMID 33527139, PMID 34783716, PMID 36214650, PMID 36519620, PMID 37460782, PMID 38671361, PMID 41491634, PMID 42002216, PMID 12401454, PMID 15100156
2023 (28), P30CA015704 (26), 2025 (22), 2017 (20), 2018 (18), P30CA006973 (17), 2015 (16), 2016 (16), 2019 (16), P30CA043703 (16), U10CA032102 (16), P30CA033572 (14), U01CA070095 (12), P30CA072720 (11), 2008 (9)