201. アンジェルマン症候群 Angelman syndrome Clinical trials / Disease details
臨床試験数 : 24 / 薬物数 : 35 - (DrugBank : 9) / 標的遺伝子数 : 22 - 標的パスウェイ数 : 20
Showing 1 to 10 of 24 diseases
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05127226 (ClinicalTrials.gov) | December 22, 2021 | 9/11/2021 | HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Dos ... | HALOS: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients With Angelman Syndrome HALOS: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrat ... | Angelman Syndrome | Drug: ION582 | Ionis Pharmaceuticals, Inc. | NULL | Recruiting | 2 Years | 50 Years | All | 44 | Phase 1/Phase 2 | United States |
| 2 | NCT05011851 (ClinicalTrials.gov) | October 1, 2021 | 5/8/2021 | An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman S ... | An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman S ... | Angelman Syndrome | Drug: NNZ-2591 | Neuren Pharmaceuticals Limited | NULL | Not yet recruiting | 3 Years | 17 Years | All | 20 | Phase 2 | Australia |
| 3 | NCT04863794 (ClinicalTrials.gov) | April 29, 2021 | 26/4/2021 | A Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male Participants A Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathec ... | A Non-Randomized, Open-Label, Adaptive, Single Center, Positron Emission Tomography (Pet) Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male Participants A Non-Randomized, Open-Label, Adaptive, Single Center, Positron Emission Tomography (Pet) Study To A ... | Angelman Syndrome | Drug: RO7248824 | Hoffmann-La Roche | NULL | Recruiting | 25 Years | 55 Years | Male | 26 | Phase 1 | Netherlands |
| 4 | NCT04768803 (ClinicalTrials.gov) | March 15, 2021 | 16/2/2021 | Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and/or Overweight, and/or Obesity Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, an ... | Circulating Levels of Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and / or Overweight, and / or Obesity Circulating Levels of Ghrelin in Patients With a Rare Disease Associated With Intellectual Disabilit ... | Angelman Syndrome;Smith-Magenis Syndrome;X Fragile Syndrome;Epilepsy;Prader-Willi Syndrome | Biological: acylated and unacylated ghrelin dosages | University Hospital, Toulouse | NULL | Not yet recruiting | 3 Years | 50 Years | All | 300 | NULL | |
| 5 | EUCTR2019-003787-48-NL (EUCTR) | 22/09/2020 | 08/10/2020 | Angelman syndrome is a complex genetic disorder that primarily affects the nervous system. | AN OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF RO7248824 IN PARTICIPANTS WITH ANGELMAN SYNDROME AN OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARM ... | Angelman Syndrome MedDRA version: 20.0;Level: PT;Classification code 10049004;Term: Angelman's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Angelman Syndrome MedDRA version: 20.0;Level: PT;Classification code 10049004;Term: Angelman's syndr ... | Product Code: RO7248824/F02-01 INN or Proposed INN: Not available Other descriptive name: RO7248824 Product Code: RO7248824/F03-01 (Diluent for RO7248824/F02-01 INN or Proposed INN: Not available Other descriptive name: RO7248824 Product Code: RO7248824/F02-01 INN or Proposed INN: Not available Other descriptive name: RO7248824 Pro ... | F. Hoffmann-La Roche | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 1 | United States;Canada;Spain;Netherlands;United Kingdom;Italy | ||
| 6 | NCT04428281 (ClinicalTrials.gov) | August 19, 2020 | 9/6/2020 | A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 ... | An Open-Label, Multicenter Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome An Open-Label, Multicenter Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharm ... | Angelman Syndrome | Drug: RO7248824 | Hoffmann-La Roche | NULL | Recruiting | 1 Year | 12 Years | All | 140 | Phase 1 | United States;Italy;Netherlands;Spain;Canada;United Kingdom |
| 7 | EUCTR2019-003787-48-IT (EUCTR) | 03/07/2020 | 15/06/2021 | Angelman syndrome is a complex genetic disorder that primarily affects the nervous system. | AN OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF RO7248824 IN PARTICIPANTS WITH ANGELMAN SYNDROME - TANGELO AN OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARM ... | Angelman Syndrome MedDRA version: 20.0;Level: PT;Classification code 10049004;Term: Angelman's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Angelman Syndrome MedDRA version: 20.0;Level: PT;Classification code 10049004;Term: Angelman's syndr ... | Product Name: - Product Code: [RO7248824/F02-01] INN or Proposed INN: Non disponibile Other descriptive name: RO7248824 Product Name: - Product Code: [RO7248824/F02-01] INN or Proposed INN: Non disponibile Other descriptive ... | F. HOFFMANN - LA ROCHE LTD. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 1 | United States;Canada;Spain;Netherlands;United Kingdom;Italy | ||
| 8 | EUCTR2019-003787-48-GB (EUCTR) | 11/06/2020 | 16/03/2020 | Angelman syndrome is a complex genetic disorder that primarily affects the nervous system. | AN OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF RO7248824 IN PARTICIPANTS WITH ANGELMAN SYNDROME AN OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARM ... | Angelman Syndrome MedDRA version: 20.0;Level: PT;Classification code 10049004;Term: Angelman's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Angelman Syndrome MedDRA version: 20.0;Level: PT;Classification code 10049004;Term: Angelman's syndr ... | Product Code: RO7248824/F02-01 INN or Proposed INN: Not available Other descriptive name: RO7248824 Product Code: RO7248824/F03-01 (Diluent for RO7248824/F02-01 INN or Proposed INN: Not available Other descriptive name: RO7248824 Product Code: RO7248824/F02-01 INN or Proposed INN: Not available Other descriptive name: RO7248824 Pro ... | F. Hoffmann-La Roche | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 1 | United States;Canada;Spain;Netherlands;Italy;United Kingdom | ||
| 9 | EUCTR2019-004478-24-DE (EUCTR) | 05/06/2020 | 26/02/2020 | An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals with Angelman Syndrome (ELARA) An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individ ... | An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals with Angelman Syndrome (ELARA) - ELARA An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individ ... | Angelman Syndrome MedDRA version: 20.0;Level: PT;Classification code 10049004;Term: Angelman's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] Angelman Syndrome MedDRA version: 20.0;Level: PT;Classification code 10049004;Term: Angelman's syndr ... | Product Name: Gaboxadol monohydrate 0.5 mg (intended commercial formulation) Product Code: OV101 INN or Proposed INN: Gaboxadol Other descriptive name: GABOXADOL MONOHYDRATE Product Name: Gaboxadol monohydrate 2 mg (intended commercial formulation) Product Code: OV101 INN or Proposed INN: Gaboxadol Other descriptive name: GABOXADOL MONOHYDRATE Product Name: Gaboxadol monohydrate 5 mg (intended commercial formulation) Product Code: OV101 INN or Proposed INN: Gaboxadol Other descriptive name: GABOXADOL MONOHYDRATE Product Name: Gaboxadolmonohydrate0.5 mg (intended commercial formulation) Product Code: OV101 INN or ... | Ovid Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 170 | Phase 3 | United States;Australia;Israel;Netherlands;Germany | ||
| 10 | NCT04259281 (ClinicalTrials.gov) | February 24, 2020 | 31/1/2020 | A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome | A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS) A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability ... | Angelman Syndrome | Drug: GTX-102 | GeneTX Biotherapeutics, LLC | Ultragenyx Pharmaceutical Inc | Recruiting | 4 Years | 17 Years | All | 20 | Phase 1/Phase 2 | United States;Canada;United Kingdom |