74 terminated. 74 Phase 3, 19 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 74 | Terminated 74 |
| Phase 2 | 19 | Terminated 19 |
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 |
|---|---|---|---|---|---|---|
| ARD-101 for Treatment of PWS: The Hunger Elimination or Reduction Objective Trial Placebo, ARD-101 - Hyperphagia, Prader-Willi Syndrome | Phase 3 | Terminated | Aardvark Therapeutics, Inc. | 68 | 2024-12-20 | 2026-06-30 |
| Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) GTX-102, Sham-LP - Angelman Syndrome | Phase 3 | Terminated | Ultragenyx Pharmaceutical Inc | 129 | 2024-12-03 | 2026-11-03 |
| Study to Assess Efficacy, Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Obeticholic Acid (OCA) Compared to Placebo in Pediatric Participants With Biliary Atresia, Post-hepatoportoenterostomy OCA, Matching Placebo - Biliary Atresia | Phase 2 / Phase 3 | Terminated | Intercept Pharmaceuticals | 28 | 2024-09-02 | 2026-07-07 |
| Long-term Extension of GTX-102 in Angelman Syndrome GTX-102 - Angelman Syndrome | Phase 3 | Terminated | Ultragenyx Pharmaceutical Inc | 66 | 2024-07-31 | 2026-11-03 |
| The Hunger Elimination or Reduction Objective (HERO ) Open -Label Extension (OLE) Trial ARD-101 - Hyperphagia, Prader-Willi Syndrome | Phase 3 | Terminated | Aardvark Therapeutics, Inc. | 19 | 2024-07-23 | 2026-06-30 |
| LIFE-DSR-Biomarker Sub-study of Biomarkers in Down Syndrome Related Alzheimer's Disease (DS-AD) [18F]MK-6240 - Down Syndrome | Phase 3 | Terminated | LuMind IDSC Foundation | 5 | 2023-06-27 | 2025-03-06 |
| A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome Spesolimab - solution for infusion, Placebo matching to spesolimab - solution for infusion, Spesolimab - solution for injection - Netherton Syndrome | Phase 2 / Phase 3 | Terminated | Boehringer Ingelheim | 43 | 2023-06-12 | 2026-03-12 |
| A Study Evaluating the Efficacy and Safety of IV L-Citrulline for the Prevention of Clinical Sequelae of Acute Lung Injury Induced by Cardiopulmonary Bypass in Pediatric Patients Undergoing Surgery for Congenital Heart Defects L-citrulline, Plasmalyte A - Ventricular Septal Defect, Atrioventricular Septal Defect | Phase 3 | Terminated | Asklepion Pharmaceuticals, LLC | 64 | 2022-06-29 | 2024-09-19 |
| Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy Ganaxolone - Tuberous Sclerosis Complex | Phase 3 | Terminated | Marinus Pharmaceuticals | 117 | 2022-05-16 | 2025-10-01 |
| A Study to Assess RAD011 (Cannabidiol Oral Solution) for the Treatment of Participants With Prader-Willi Syndrome RAD011, Placebo - Prader-Willi Syndrome | Phase 2 / Phase 3 | Terminated | Radius Pharmaceuticals, Inc. | 4 | 2022-04-13 | 2023-10-19 |
| Roll-over Study to Assess Safety of Lixivaptan in Participants With ADPKD Who Completed Study PA-ADPKD-303 Lixivaptan - Polycystic Kidney Disease, Adult | Phase 3 | Terminated | Palladio Biosciences | 1 | 2022-02-10 | 2023-05-12 |
| Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste) QR-421a, Sham-procedure - Retinitis Pigmentosa, Usher Syndrome Type 2 | Phase 2 / Phase 3 | Terminated | Laboratoires Thea | 5 | 2021-12-15 | 2024-08-07 |
| Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius) Ultevursen, Sham-procedure - Retinitis Pigmentosa, Usher Syndrome Type 2 | Phase 2 / Phase 3 | Terminated | Laboratoires Thea | 7 | 2021-12-08 | 2024-07-12 |
| Efficacy and Safety of Lixivaptan in the Treatment of Autosomal Dominant Polycystic Kidney Disease Lixivaptan, Placebo - Autosomal Dominant Polycystic Kidney, ADPKD | Phase 3 | Terminated | Palladio Biosciences | 12 | 2021-10-28 | 2023-02-06 |
| Opioid Reduction Initiative During Outpatient Pediatric Urologic Procedures Using Exparel Exparel 133 miligrams per 10 milliliter injection, Bupivacaine Hydrochloride - Urologic Diseases, Hypospadias | Phase 3 | Terminated | Johns Hopkins University | 104 | 2021-10-01 | 2023-12-26 |
| Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures GWP42003-P - Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome | Phase 3 | Terminated | Jazz Pharmaceuticals | 3 | 2021-05-19 | 2025-10-31 |
| Specialized Pacing for Patients With Congenital Heart Disease Closed loop stimulation, Standard rate response - Congenital Heart Disease, Bradycardia Sinus | Phase 2 / Phase 3 | Terminated | University of California, Los Angeles | 4 | 2021-05-09 | 2023-01-20 |
| Safety of Lixivaptan in Subjects Previously Treated With Tolvaptan for Autosomal Dominant Polycystic Kidney Disease Lixivaptan - Polycystic Kidney Disease, Adult, ADPKD | Phase 3 | Terminated | Palladio Biosciences | 7 | 2020-09-02 | 2023-04-10 |
| A Pilot Study of the Efficacy and Safety of Dupilumab Versus Placebo in Patients With Netherton Syndrome Dupilumab Prefilled Syringe, Placebo Prefilled Syringe - Netherton Syndrome | Phase 2 / Phase 3 | Terminated | University Hospital, Toulouse | 10 | 2020-07-23 | 2026-09-21 |
| Propranolol for Epistaxis in Hereditary Hemorrhagic Telangiectasia Patients Propranolol treatment, Placebo - Hereditary Hemorrhagic Telangiectasia, Osler Weber Rendu Disease | Phase 3 | Terminated | University Hospital, Bordeaux | 15 | 2020-06-23 | 2026-06-23 |
Showing 20 of 74. Sign in to see all of them.
Polycystic Kidney, Autosomal Dominant (9) · Prader-Willi Syndrome (8) · Hereditary Sensory and Autonomic Neuropathies (6) · Hyperphagia (5) · Down Syndrome (4) · Osteoarthritis (4) · Angelman Syndrome (3) · Breast Neoplasms (3) · Ductus Arteriosus, Patent (3) · Foramen Ovale, Patent (3) · Arthritis (2) · Basal Cell Nevus Syndrome (2) · Carcinoma, Basal Cell (2) · Cognitive Dysfunction (2) · Collagen Diseases (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (74) · Genetic Diseases, Inborn (45) · Abnormalities, Multiple (33) · Nervous System Diseases (30) · Pathological Conditions, Signs and Symptoms (23) · Cardiovascular Diseases (21) · Cardiovascular Abnormalities (20) · Chromosome Disorders (18) · Neurologic Manifestations (18) · Heart Defects, Congenital (16) · Heart Diseases (16) · Intellectual Disability (14) · Neurobehavioral Manifestations (14) · Female Urogenital Diseases and Pregnancy Complications (13)
lixivaptan (3) · Donepezil (2) · Fulvestrant (2) · Indomethacin (2) · Intravitreal Injections (2) · Sirolimus (2) · bardoxolone methyl (2) · macitentan (2) · tanezumab (2) · Acetaminophen (1) · Alpelisib (1) · Anti-Inflammatory Agents, Non-Steroidal (1) · Aspirin (1) · Atenolol (1) · Bevacizumab (1)
Novartis Pharmaceuticals (3) · Palladio Biosciences (3) · Aardvark Therapeutics, Inc. (2) · Actelion (2) · Assistance Publique - Hôpitaux de Paris (2) · Biogen (2) · Cardiox Corporation (2) · Competence Network for Congenital Heart Defects (2) · DePuy International (2) · Eisai Inc. (2) · Laboratoires Thea (2) · Pfizer (2) · The Hospital for Sick Children (2) · Ultragenyx Pharmaceutical Inc (2) · A. Manzoni Hospital (1)
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