26 recruiting. 26 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 4 | 26 | Recruiting 26 |
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 |
|---|---|---|---|---|---|---|
| Clinical Trial in Patients With Barth Syndrome- 4TAZPower Elamipretide, Placebo - Barth Syndrome | Phase 4 | Recruiting | Stealth BioTherapeutics Inc. | 48 | 2026-07-02 | 2026-09-17 |
| The Effect of Dapagliflozin on Exercise and Cardiac Functional Status of Patients After Fontan Procedure Dapagliflozin (5-10 mg daily) - SGLT2 Inhibitor Therapy - Fontan, Heart Failure | Phase 4 | Recruiting | The University of Hong Kong | 30 | 2026-07-01 | 2026-07-28 |
| Endotype DIrected Treatment for OSA in Down Syndrome ato-oxy, Oxygen - Obstructive Sleep Apnea (OSA), Down Syndrome | Phase 4 | Recruiting | University of Arizona | 200 | 2026-03-14 | 2026-09-03 |
| Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS) Osilodrostat (Isturisa) - Mild Autonomous Cortisol Secretion (MACS) | Phase 4 | Recruiting | Johns Hopkins University | 10 | 2026-02-02 | 2026-10-23 |
| De-implementing Inhaled Nitric Oxide for Congenital Diaphragmatic Hernia Inhaled Nitric Oxide (iNO) use, De-implementation of Inhaled Nitric Oxide (iNO) use - Congenital Diaphragmatic Hernia | Phase 4 | Recruiting | The University of Texas Health Science Center, Houston | 900 | 2025-11-01 | 2026-10-29 |
| GLP-1R Actions on Muscle and the Skeleton Tirzepatide, Lifestyle toolkit - Musculoskeletal Abnormalities, Obesity | Phase 4 | Recruiting | Pennington Biomedical Research Center | 50 | 2025-10-09 | 2026-08-27 |
| A Study of Sotatercept for Patients With Eisenmenger Syndrome or Unrepaired Shunt-Associated Pulmonary Arterial Hypertension Resistant to Vasodilator Therapy Sotatercept, vasodilator-based PAH therapy - Eisenmenger Syndrome, Pulmonary Arterial Hypertension of Congenital Heart Disease | Phase 4 | Recruiting | Kazuya Hosokawa | 36 | 2025-10-07 | 2026-01-21 |
| Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU) Livmarli Oral Product - Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis | Phase 4 | Recruiting | Mirum Pharmaceuticals, Inc. | 230 | 2025-09-25 | 2026-06-23 |
| Bleeding Patterns in Sequential and Continuous Progesterone Supplementation in Adolescents With Turner Syndrome Micronized progesterone 200 MG, Micronized Progesterone 100 MG - Turner Syndrome, Primary Ovarian Insufficiency (Poi) | Phase 4 | Recruiting | Children's Mercy Hospital Kansas City | 40 | 2025-07-31 | 2026-08-25 |
| Optimizing Perioperative Analgesia to Lower Pain Following Cleft Palate Surgery Methadone hydrochloride, Fentanyl/Hydromorphone - Cleft Palate, Pain | Phase 4 | Recruiting | Duke University | 60 | 2025-06-04 | 2026-04-14 |
| Sodium-Glucose Cotransporter 2 Inhibitors for Repaired Tetralogy of Fallot Patients for Enhancement of Cardio-Pulmonary Status Trial SGLT2 inhibitor (Dapagliflozin 10mg) - Congenital Heart Disease, Repaired Tetralogy of Fallot (rTOF) | Phase 4 | Recruiting | The University of Hong Kong | 106 | 2025-05-12 | 2026-10-28 |
| Tirzepatide in PWS, HO and GNSO Tirzepatide - Prader-Willi Syndrome, Hypothalamic Obesity | Phase 4 | Recruiting | Grace Kim | 36 | 2025-05-01 | 2025-09-16 |
| Klinefelter Syndrome and Testosterone Treatment in Puberty Testosterone gel, Placebo - Klinefelter Syndrome | Phase 4 | Recruiting | Lise Aksglæde | 32 | 2024-08-15 | 2025-02-19 |
| Sinus Tarsi Implant as an Adjuvant Procedure to Medial Displacement Calcaneal Osteotomy in the Treatment of Mobile Adult Acquired Flatfoot Deformity ProStop, sinus tarsi implant (STI), Conventional surgery - Acquired Adult Flatfoot Deformity, Flat Foot | Phase 4 | Recruiting | Region Skane | 130 | 2024-07-31 | 2025-12-02 |
| Nebulized Dexmedetomidine Combined With Ketamine Versus Nebulized Dexmedetomidine for Cleft Palate Nebulization of dexmedetomidine and ketamine, Nebulization of dexmedetomidine - Sedation, Emergence Delirium | Phase 4 | Recruiting | Assiut University | 60 | 2024-04-10 | 2025-10-03 |
| Effect of Dexmedetomidine Supplementation to General Anaesthesia in Paediatric Transcatheter Closure of Atrial Septal Defect Dexmedetomidine, Saline (NaCl 0,9 %) (placebo) - Atrial Septal Defect (ASD), Dexmedetomidine | Phase 4 | Recruiting | Alexandria University | 30 | 2024-04-03 | 2024-10-15 |
| Safety and Efficacy of 18 mm Short Vitrectomy Probe for Pediatric Vitreoretinal Surgeries Pars plana vitrectomy with a 18 mm short vitrector - Retinopathy of Prematurity Both Eyes, Retinal Disease | Phase 4 | Recruiting | Chang Gung Memorial Hospital | 60 | 2024-03-05 | 2024-07-25 |
| Multifidus Cervicis Plane Block Vs. Sham Block For Posterior Cervical Spine Fusion Surgery Bupivacaine Hcl 0.25% Inj, Normal saline - Cervical Spine Fusion | Phase 4 | Recruiting | Hartford Hospital | 38 | 2023-12-12 | 2025-09-05 |
| Real-World Effects of MC4R Agonist Therapy in BBS and Severe Genetic Obesity Setmelanotide - Bardet Biedl Syndrome (BBS), Bardet Biedl Syndrome | Phase 4 | Recruiting | Tom Hühne | 200 | 2023-01-01 | 2026-06-29 |
| Long Term Effects of Oral Versus Transdermal Estrogen Replacement Therapy in Turner Syndrome 17-beta estradiol - Turner Syndrome, Hypogonadism; Ovarian | Phase 4 | Recruiting | Aarhus University Hospital | 50 | 2021-11-29 | 2024-08-26 |
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Heart Defects, Congenital (3) · Turner Syndrome (3) · Adrenal Hyperplasia, Congenital (2) · Agnosia (2) · Cleft Lip (2) · Cleft Palate (2) · Emergence Delirium (2) · Hypogonadism (2) · Obesity (2) · ACTH Syndrome, Ectopic (1) · Alagille Syndrome (1) · Alstrom Syndrome (1) · Bardet-Biedl Syndrome (1) · Barth Syndrome (1) · Cardiovascular Diseases (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (26) · Cardiovascular Diseases (13) · Genetic Diseases, Inborn (13) · Cardiovascular Abnormalities (11) · Heart Diseases (10) · Pathological Conditions, Signs and Symptoms (9) · Endocrine System Diseases (8) · Heart Defects, Congenital (8) · Nervous System Diseases (8) · Signs and Symptoms (8) · Chromosome Disorders (7) · Disorders of Sex Development (7) · Female Urogenital Diseases (7) · Female Urogenital Diseases and Pregnancy Complications (7)
Dexamethasone (2) · Estradiol (2) · Tirzepatide (2) · dapagliflozin (2) · estradiol, estriol drug combination (2) · ACE-011 (1) · Atomoxetine Hydrochloride (1) · Bupivacaine (1) · Cortisone (1) · Dexmedetomidine (1) · Epinephrine (1) · Fentanyl (1) · Fluoxetine (1) · Hydromorphone (1) · Inosine (1)
The University of Texas Health Science Center, Houston (3) · Aarhus University Hospital (2) · The University of Hong Kong (2) · Alexandria University (1) · Assiut University (1) · Chang Gung Memorial Hospital (1) · Children's Mercy Hospital Kansas City (1) · Duke University (1) · Federico II University (1) · Grace Kim (1) · Hartford Hospital (1) · Johns Hopkins University (1) · Kazuya Hosokawa (1) · Lise Aksglæde (1) · Mirum Pharmaceuticals, Inc. (1)
United States (12) · Denmark (3) · Egypt (2) · Germany (2) · Hong Kong (2) · Italy (2) · Belgium (1) · Canada (1) · France (1) · Greece (1) · Israel (1) · Japan (1) · Netherlands (1) · Portugal (1) · Spain (1)
Claus H Gravholt, Professor (2) · Amir Hamrahian, MD (1) · Chun-Ka Dr Wong, Clinical Assistant Professor (1) · Daniel Combs, MD (1) · Ida Osbeck, MD (1) · Kevin Engelhardt, MD (1) · Lisa M. Einhorn, M.D. (1) · Lise Aksglaede, MD (1) · Matthew Greives, MD (1) · Matthew Harting, MD, MS, FACS (1) · Matthew R Grieves, MD,MS,FACS (1) · Metin Cetiner, PD Dr. med. (1) · Pranjali Kainkaryam, MD (1) · Prof Yiu Fai Cheung (1) · Rekha Sathyanarayana (1)
PMID 17572925, PMID 20970202, PMID 26114442, PMID 27454103, PMID 28887710, PMID 29222139, PMID 29318534, PMID 29510982, PMID 29663436, PMID 30817193, PMID 31206616, PMID 31351553, PMID 31535829, PMID 31980166, PMID 33197559, PMID 34405863, PMID 34656466, PMID 35220871, PMID 3536461, PMID 35487320, PMID 35817047, PMID 36171554, PMID 36605072, PMID 36952249, PMID 37318239, PMID 37435053, PMID 37615988, PMID 37622666, PMID 37801655, PMID 38162132, PMID 38430085, PMID 39062179, PMID 39086899, PMID 40090688, PMID 40903014, PMID 41819579, PMID 6135282
2023 (2)