12 unknown. 12 Phase 3, 3 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 12 | Unknown 12 |
| Phase 2 | 3 | Unknown 3 |
Follow Abnormalities, Multiple: new trials by email Literature Review of Abnormalities, Multiple trials Research Report on Abnormalities, Multiple
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 |
|---|---|---|---|---|---|---|
| Long-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial Follow-up study of the treated cohort, Follow-up study of the untreated cohort - Prader-Willi Syndrome | Phase 3 | Unknown | University Hospital, Toulouse | 80 | 2021-09-07 | 2024-04-30 |
| Metformin vs Tolvaptan for Treatment of Autosomal Dominant Polycystic Kidney Disease Metformin, Tolvaptan - ADPKD | Phase 3 | Unknown | Azienda Ospedaliero-Universitaria Consorziale Policlinico di Bari | 150 | 2019-01-30 | 2018-12-07 |
| A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome Recombinant Human Growth Hormone (rhGH) Injection - Prader-Willi Syndrome | Phase 3 | Unknown | Changchun GeneScience Pharmaceutical Co., Ltd. | 30 | 2018-04-14 | 2018-06-12 |
| Indicated Prevention With Long-chain Polyunsaturated Omega-3 Fatty Acids in Patients With 22q11 Microdeletion Syndrome. omega-3 PUFAs, Standard care, placebo - 22q11 Deletion Syndrome | Phase 2 / Phase 3 | Unknown | Bambino Gesù Hospital and Research Institute | 80 | 2014-06-01 | 2014-02-25 |
| Triptolide-Containing Formulation as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD) Triptolide-Containing Formulation, Placebo - Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Phase 3 | Unknown | Shanghai Changzheng Hospital | 100 | 2014-04-01 | 2014-04-17 |
| Pulsed Oral Sirolimus in Autosomal Dominant Polycystic Kidney Disease Sirolimus, Placebo - Polycystic Kidney, Type 1 Autosomal Dominant Disease, Polycystic Kidney, Type 2 Autosomal Dominant Disease | Phase 3 | Unknown | Medical University of Vienna | 68 | 2014-04-01 | 2018-10-31 |
| Study of Lanreotide to Treat Polycystic Kidney Disease Lanreotide - Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Phase 3 | Unknown | University Medical Center Groningen | 300 | 2012-06-01 | 2017-05-23 |
| Sirolimus for Massive Polycystic Liver Sirolimus - Polycystic Kidney Diseases | Phase 2 / Phase 3 | Unknown | Seoul National University Hospital | 44 | 2011-09-01 | 2012-09-07 |
| Atenolol Versus Losartan in the Prevention of Progressive Dilation of the Aorta in Marfan Syndrome Losartan, Atenolol - Marfan Syndrome | Phase 3 | Unknown | Forteza, Albert, M.D. | 140 | 2008-10-01 | 2010-08-12 |
| Nebivolol Versus Losartan Versus Nebivolol+Losartan Against Aortic Root Dilation in Genotyped Marfan Patients Losartan and nebivolol, Losartan, Nebivolol - Marfan Syndrome | Phase 3 | Unknown | Fondazione IRCCS Policlinico San Matteo di Pavia | 291 | 2008-07-01 | 2011-07-22 |
| Open-Label Extension of LOCKCYST Trial lanreotide - Polycystic Liver Disease, Hepatomegaly | Phase 2 / Phase 3 | Unknown | Radboud University Medical Center | 54 | 2008-04-01 | 2008-11-13 |
| Vitamin E in Aging Persons With Down Syndrome Vitamin E, multivitamin, Placebo - Down Syndrome, Alzheimer Disease | Phase 3 | Unknown | New York State Institute for Basic Research | 350 | 2002-04-01 | 2012-05-04 |
Polycystic Kidney Diseases (4) · Polycystic Kidney, Autosomal Dominant (4) · Marfan Syndrome (2) · Polycystic liver disease (2) · Prader-Willi Syndrome (2) · 22q11 Deletion Syndrome (1) · Alzheimer Disease (1) · Camurati-Engelmann Syndrome (1) · Down Syndrome (1) · Hepatomegaly (1) · Hereditary Sensory and Autonomic Neuropathies (1) · Liver Diseases (1) · Multiple Pterygium Syndrome, Autosomal Dominant (1) · Psychotic Disorders (1)
Congenital Abnormalities (12) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (12) · Genetic Diseases, Inborn (12) · Ciliopathies (6) · Female Urogenital Diseases (6) · Female Urogenital Diseases and Pregnancy Complications (6) · Kidney Diseases (6) · Kidney Diseases, Cystic (6) · Male Urogenital Diseases (6) · Urogenital Diseases (6) · Urologic Diseases (6) · Chromosome Disorders (4) · Nervous System Diseases (4) · Cardiovascular Abnormalities (3)
Losartan (2) · Sirolimus (2) · lanreotide (2) · Atenolol (1) · Fish Oils (1) · Geritol (1) · Growth Hormone (1) · Metformin (1) · Nebivolol (1) · Standard of Care (1) · Tocopherols (1) · Tolvaptan (1) · Vitamin E (1)
Azienda Ospedaliero-Universitaria Consorziale Policlinico di Bari (1) · Bambino Gesù Hospital and Research Institute (1) · Changchun GeneScience Pharmaceutical Co., Ltd. (1) · Fondazione IRCCS Policlinico San Matteo di Pavia (1) · Forteza, Albert, M.D. (1) · Medical University of Vienna (1) · New York State Institute for Basic Research (1) · Radboud University Medical Center (1) · Seoul National University Hospital (1) · Shanghai Changzheng Hospital (1) · University Hospital, Toulouse (1) · University Medical Center Groningen (1)
China (2) · Italy (2) · Netherlands (2) · Australia (1) · Austria (1) · Belgium (1) · Canada (1) · France (1) · Holy See (1) · South Korea (1) · Spain (1) · United Kingdom (1) · United States (1)
Alberto Forteza, Dr (1) · Arthur J Dalton, PhD (1) · Arturo Evangelista, Dr (1) · Changlin Mei, MD (1) · Curie Ahn, MD, PhD (1) · Eloisa Arbustini, MD,FESC,FACC (1) · Feihong Luo, Doctor (1) · Frederik Nevens, MD, PhD (1) · Gere Sunder-Plassmann, MD (1) · Joost PH Drenth, PhD (1) · Loes van Keimpema, MSc (1) · Loreto Gesualdo (1) · Luigi Tavazzi, MD,FESC,FACC (1) · Marco Armando, MD, PhD (1)
PMID 39356039, PMID 16601194, PMID 17188619, PMID 17911499, PMID 19430350, PMID 25899445, PMID 27995519, PMID 30422235, PMID 31022403, PMID 33779943, PMID 38837240
R01AG016381 (1)