253 completed. 253 Phase 3, 48 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 253 | Completed 253 |
| Phase 2 | 48 | Completed 48 |
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 |
|---|---|---|---|---|---|---|
| Prophylactic IV Paracetamol in Extremely Premature Infants Paracetamol (drug) - Patent Ductus Arteriosus in Preterm Infants, Patent Ductus Arteriosus After Premature Birth | Phase 2 / Phase 3 | Completed | Al-Quds University | 40 | 2025-01-01 | 2026-04-07 |
| Open-Label Extension Study of Setmelanotide Setmelanotide - Obesity Associated With Defects in Leptin-melanocortin Pathway | Phase 3 | Completed | Rhythm Pharmaceuticals, Inc. | 28 | 2024-10-21 | 2026-07-15 |
| Comparative Effectiveness of Gadopiclenol for Evaluation of Adult Congenital Heart Anatomy and Hemodynamics Gadopiclenol, Gadobenate dimeglumine - Congenital Heart Disease | Phase 3 | Completed | University of California, San Diego | 161 | 2024-08-05 | 2026-11-03 |
| Effects of Topical Lidocaine and Benzocaine on Pain Level and Injection Satisfaction Lidocaine Spray: Each ml contained 10 mg lidocaine and ethanol. Applied to the local site to provide anesthesia, Benzocaine spray, Placebo group (control arm) - Pain, Satisfaction | Phase 3 | Completed | TC Erciyes University | 60 | 2024-04-01 | 2025-12-01 |
| The Desensitising Effect of Nanosilver Fluoride in Molar-Incisor Hypomineralisation Low-level laser therapy, Nanosilver fluoride - Molar Incisor Hypomineralization, Hypersensitivity | Phase 2 / Phase 3 | Completed | Alexandria University | 120 | 2024-04-01 | 2025-01-30 |
| Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome Patidegib Topical Gel, Patidegib Topical Gel with no active patidegib - Gorlin Syndrome | Phase 3 | Completed | Sol-Gel Technologies, Ltd. | 113 | 2024-03-17 | 2026-09-18 |
| Augmented Reality for Orthognatic Surgery Patient Education Patient consultation - Malocclusion, Dentofacial Deformities | Phase 2 / Phase 3 | Completed | University Medical Center Groningen | 60 | 2023-10-01 | 2025-03-30 |
| Effects of Pazopanib on Hereditary Hemorrhagic Telangiectasia Related Epistaxis and Anemia (Paz) Pazopanib, Placebo oral capsule - Hereditary Hemorrhagic Telangiectasia, Epistaxis | Phase 2 / Phase 3 | Completed | Cure HHT | 71 | 2023-05-08 | 2026-10-27 |
| Open-Label Extension Study of DCCR in Patients With Prader-Willi Syndrome DCCR - Prader-Willi Syndrome | Phase 3 | Completed | Soleno Therapeutics, Inc. | 77 | 2023-01-31 | 2026-10-13 |
| A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS) TAK-625 - Alagille Syndrome (ALGS) | Phase 3 | Completed | Takeda | 7 | 2023-01-16 | 2026-01-29 |
| A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome BPN14770/ zatolmilast, Placebo - Fragile X Syndrome | Phase 3 | Completed | Tetra Discovery Partners | 171 | 2022-11-01 | 2026-07-10 |
| The Role of Dexmedetomidine as Myocardial Protector in Pediatric Cardiac Surgery Total Correction of Tetralogy of Fallot Dexmedetomidine Hcl 100 Mcg/mL Inj, Placebo - Congenital Heart Disease in Children, Cardiopulmonary Bypass | Phase 2 / Phase 3 | Completed | National Cardiovascular Center Harapan Kita Hospital Indonesia | 66 | 2022-10-10 | 2023-08-24 |
| Blood Flow and Blood Pressure Investigation in Down Syndrome Exercise - Down Syndrome | Phase 3 | Completed | University of Illinois at Chicago | 48 | 2022-09-01 | 2024-04-18 |
| Norepinephrine for Prevention of Intraoperative Hypotension in Infants Undergoing Kasai Portoenterostomy Norepinephrine Bitartrate, Placebo - Anesthesia, Pediatrics | Phase 3 | Completed | Kasr El Aini Hospital | 31 | 2022-08-28 | 2024-07-09 |
| A Study Testing the Use of a Perivascular Sirolimus Formulation (Sirogen) in ESRD Patients Undergoing AV Fistula Surgery Sirolimus - Complication of Renal Dialysis, End Stage Renal Disease | Phase 3 | Completed | Vascular Therapies, Inc. | 136 | 2022-08-26 | 2026-10-06 |
| The ASCEND Study: Evaluating TMB-001 in the Treatment of RXLI or ARCI Ichthyosis TMB-001, Matching Vehicle - Ichthyosis | Phase 3 | Completed | LEO Pharma | 153 | 2022-06-21 | 2026-05-22 |
| Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC NPC-12Y gel, NPC-12Y placebo gel - Tuberous Sclerosis Complex | Phase 3 | Completed | Nobelpharma | 43 | 2022-06-01 | 2024-11-22 |
| Comparison of Chronocort Versus Standard Hydrocortisone Replacement Therapy in Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasia Chronocort, Cortef, Placebo - Congenital Adrenal Hyperplasia | Phase 3 | Completed | Neurocrine UK Limited | 55 | 2022-05-24 | 2026-10-02 |
| Effect of Intranasal Oxytocin on Dysphagia in Children and Adolescents With Prader-Willi Syndrome Oxytocin nasal spray, Placebo - Prader-Willi Syndrome | Phase 2 / Phase 3 | Completed | University Hospital, Toulouse | 25 | 2022-04-05 | 2023-06-01 |
| Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy Ganaxolone, Placebo - Tuberous Sclerosis Complex | Phase 3 | Completed | Marinus Pharmaceuticals | 129 | 2022-04-01 | 2025-07-11 |
Showing 20 of 253. Sign in to see all of them.
Heart Defects, Congenital (29) · Leukemia, Myeloid, Acute (22) · Polycystic Kidney, Autosomal Dominant (21) · Leukemia (18) · Leukemia, Erythroblastic, Acute (13) · Leukemia, Megakaryoblastic, Acute (13) · Leukemia, Monocytic, Acute (13) · Leukemia, Myelomonocytic, Acute (13) · Myelodysplastic Syndromes (13) · Myeloproliferative Disorders (12) · Ductus Arteriosus, Patent (11) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (11) · Tuberous Sclerosis (11) · Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative (10)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (253) · Genetic Diseases, Inborn (111) · Cardiovascular Diseases (101) · Cardiovascular Abnormalities (88) · Pathological Conditions, Signs and Symptoms (69) · Heart Diseases (66) · Female Urogenital Diseases and Pregnancy Complications (64) · Urogenital Diseases (64) · Female Urogenital Diseases (60) · Male Urogenital Diseases (60) · Nervous System Diseases (60) · Abnormalities, Multiple (59) · Neoplasms (46) · Heart Defects, Congenital (42)
Cytarabine (15) · Daunorubicin (12) · Human Growth Hormone (11) · Etoposide (7) · Sodium Chloride (7) · Tolvaptan (7) · Ibuprofen (6) · Saline Solution (6) · Sirolimus (6) · Fentanyl (5) · Gemtuzumab (5) · fludarabine phosphate (5) · Acetaminophen (4) · Busulfan (4) · Cyclophosphamide (4)
Novo Nordisk A/S (6) · Assistance Publique - Hôpitaux de Paris (5) · Otsuka Pharmaceutical Development & Commercialization, Inc. (5) · University Hospital, Toulouse (5) · Rhythm Pharmaceuticals, Inc. (4) · Bayer (3) · Children's Oncology Group (3) · Hospices Civils de Lyon (3) · National Cancer Institute (NCI) (3) · National Cardiovascular Center Harapan Kita Hospital Indonesia (3) · National Heart, Lung, and Blood Institute (NHLBI) (3) · Neurocrine UK Limited (3) · Nobelpharma (3) · Novartis Pharmaceuticals (3) · Palvella Therapeutics, Inc. (3)
United States (136) · France (52) · United Kingdom (43) · Canada (40) · Germany (35) · Netherlands (28) · Italy (27) · Spain (27) · Australia (24) · Belgium (24) · Israel (16) · Poland (15) · Japan (14) · Russia (14) · Brazil (12)
David Meeker, MD (4) · Alan K. Burnett, MD, FRCP (2) · Arlene Chapman, M.D. (2) · Brian McCrindle, MD (2) · David Hansen, MD (2) · Joyce Teng, MD (2) · Marva Moxey-Mims, M.D. (2) · Michael Portman, MD (2)
PMID 39356039, PMID 31375516, PMID 36184192, PMID 36191725, PMID 37250503, PMID 31014270, PMID 26091193, PMID 27578134, PMID 29023494, PMID 30053159, PMID 30936738, PMID 31171508, PMID 33159213, PMID 33527139, PMID 34957550, PMID 36567000, PMID 37267439, PMID 37349694, PMID 37460782, PMID 37919617, PMID 38671361, PMID 38825327, PMID 11430325, PMID 12622582, PMID 15784709, PMID 16203673, PMID 16221708, PMID 16484584, PMID 16647401, PMID 16757526, PMID 16765134, PMID 16769377, PMID 17188612, PMID 17210844, PMID 17307535, PMID 17315155, PMID 17606845, PMID 17868472, PMID 17911499, PMID 17962585, PMID 18242267, PMID 18245398, PMID 18341239, PMID 18946302, PMID 19081394, PMID 19117316, PMID 19117897, PMID 19291085, PMID 19525519, PMID 19568011
2015 (5), 2005 (4), 2008 (3), 2016 (3), P30CA068485 (3), U10CA031946 (3), 2004 (2), 2009 (2), 2017 (2), 2019 (2), U01DK062401 (2), U01DK082230 (2), U01HL068270 (2), U10CA021115 (2), U10CA098543 (2)