201. アンジェルマン症候群 Angelman syndrome Clinical trials / Disease details
臨床試験数 : 25 / 薬物数 : 40 - (DrugBank : 11) / 標的遺伝子数 : 22 - 標的パスウェイ数 : 20
| 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 | NCT05630066 (ClinicalTrials.gov) | June 1, 2023 | 9/11/2022 | Study to Investigate the Pharmacokinetics and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype | A Phase IIa Multicenter, Open-Label, 12-Week Study to Investigate the Pharmacokinetics and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype | Angelman Syndrome | Drug: 60 mg QD Alogabat;Drug: 40 mg QD Alogabat;Drug: 7 mg QD Alogabat;Drug: Part 2 Adult Alogabat High Dose (aged 15-17);Drug: Alogabat | Hoffmann-La Roche | NULL | Not yet recruiting | 5 Years | 17 Years | All | 56 | Phase 2 | NULL |
| 2 | NCT05011851 (ClinicalTrials.gov) | July 12, 2022 | 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 Syndrome | Angelman Syndrome | Drug: NNZ-2591 | Neuren Pharmaceuticals Limited | NULL | Recruiting | 3 Years | 17 Years | All | 20 | Phase 2 | Australia |
| 3 | 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 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients With Angelman Syndrome | Angelman Syndrome | Drug: ION582 | Ionis Pharmaceuticals, Inc. | Biogen | Recruiting | 2 Years | 50 Years | All | 44 | Phase 1/Phase 2 | United States;Australia;Israel;United Kingdom |
| 4 | 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 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 | Angelman Syndrome | Drug: RO7248824 | Hoffmann-La Roche | NULL | Completed | 25 Years | 55 Years | Male | 21 | Phase 1 | Netherlands |
| 5 | 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 | Circulating Levels of Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and / or Overweight, and / or Obesity | 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 | |
| 6 | 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 | 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] | 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 | 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 | ||
| 7 | 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 | An Open-Label, Multicenter Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome | Angelman Syndrome | Drug: RO7248824 | Hoffmann-La Roche | NULL | Recruiting | 1 Year | 12 Years | All | 74 | Phase 1 | United States;Italy;Netherlands;Spain;Canada;United Kingdom |
| 8 | 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 | 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] | Product Name: - Product Code: [RO7248824/F02-01] INN or Proposed INN: Non disponibile Other descriptive name: RO7248824 | 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 | ||
| 9 | 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 | 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] | 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 | 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 | ||
| 10 | 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 Individuals with Angelman Syndrome (ELARA) - ELARA | 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] | 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 | Ovid Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 170 | Phase 3 | United States;Australia;Israel;Netherlands;Germany | ||
| 11 | 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) | Angelman Syndrome | Drug: GTX-102 | Ultragenyx Pharmaceutical Inc | NULL | Recruiting | 4 Years | 17 Years | All | 83 | Phase 1/Phase 2 | United States;Canada;United Kingdom |
| 12 | EUCTR2019-002907-17-NL (EUCTR) | 19/02/2020 | 10/12/2019 | Clinical Trial to Evaluate the Efficacy and Safety of of OV101 in Pediatric Individuals With Angelman Syndrome | A MulticeNter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 3 Study to Evaluate the Efficacy and Safety of OV101 in PediaTric IndividUals With AngelmaN SyndromE (NEPTUNE) - NEPTUNE | 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] | Product Name: Gaboxadol monohydrate 0.5 mg capsule Product Code: OV101 INN or Proposed INN: Gaboxadol Other descriptive name: GABOXADOL MONOHYDRATE Product Name: Gaboxadol monohydrate 2 mg capsule Product Code: OV101 INN or Proposed INN: Gaboxadol Other descriptive name: GABOXADOL MONOHYDRATE | Ovid Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 95 | Phase 3 | United States;Australia;Israel;Germany;Netherlands;Italy | ||
| 13 | NCT04106557 (ClinicalTrials.gov) | September 9, 2019 | 25/9/2019 | A Study of OV101 in Individuals With Angelman Syndrome (AS) | A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 3 Study to Evaluate the Efficacy and Safety of OV101 in Pediatric Individuals With Angelman Syndrome | Primary Disease or Condition Being Studied: Angelman Syndrome (AS) | Drug: Gaboxadol;Drug: Placebo | Ovid Therapeutics Inc. | NULL | Completed | 2 Years | 12 Years | All | 104 | Phase 3 | United States;Australia;Germany;Israel;Netherlands |
| 14 | NCT03882918 (ClinicalTrials.gov) | February 25, 2019 | 18/3/2019 | An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome | An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome | Angelman Syndrome | Drug: OV101 | Ovid Therapeutics Inc. | NULL | Recruiting | 13 Years | 49 Years | All | 82 | Phase 2 | United States;Israel |
| 15 | NCT03644693 (ClinicalTrials.gov) | November 1, 2018 | 13/8/2018 | Nutritional Formulation for Angelman Syndrome | Evaluation of the Safety and Tolerability of a Nutritional Formulation in Angelman Syndrome | Angelman Syndrome | Dietary Supplement: Nutritional Formulation Containing Exogenous Ketones;Dietary Supplement: Placebo Formulation | University of Colorado, Denver | NULL | Completed | 4 Years | 11 Years | All | 15 | N/A | United States |
| 16 | NCT03109756 (ClinicalTrials.gov) | April 3, 2017 | 6/4/2017 | Single Dose Pharmacokinetic (PK) Study | A Phase 1 Single Dose PK Study in Adolescent Subjects With Fragile X Syndrome or Angelman Syndrome | Angelman Syndrome;Fragile X Syndrome | Drug: OV101 | Ovid Therapeutics Inc. | NULL | Completed | 13 Years | 17 Years | All | 12 | Phase 1 | United States |
| 17 | NCT02996305 (ClinicalTrials.gov) | January 2016 | 2/12/2016 | A Study in Adults and Adolescents With Angelman Syndrome | A Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of Gaboxadol | Angelman Syndrome | Drug: OV101 Regimen 1;Drug: OV101 regimen 2;Other: Placebo | Ovid Therapeutics Inc. | NULL | Completed | 13 Years | 49 Years | All | 88 | Phase 2 | United States;Israel |
| 18 | NCT02056665 (ClinicalTrials.gov) | January 2014 | 5/2/2014 | Study to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome | Randomized Clinical Trial, Placebo Compared to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome | Angelman Syndrome | Drug: MINOCYCLINE;Drug: PLACEBO (for Minocycline) | Puerta de Hierro University Hospital | NULL | Completed | 6 Years | 30 Years | Both | 32 | Phase 2 | Spain |
| 19 | EUCTR2013-002154-67-ES (EUCTR) | 26/12/2013 | 16/09/2013 | STUDY TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME (A-MANECE STUDY) | RANDOMIZED CLINICAL TRIAL, PLACEBO COMPARED TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME (A-MANECE STUDY) - A-MANECE | Angelman Syndrome MedDRA version: 16.0;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] | Trade Name: Aknemin 50 INN or Proposed INN: MINOCYCLINE Other descriptive name: MINOCYCLINE HYDROCHLORIDE | Dra. Cristina Avendaño Solá | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Spain | ||||
| 20 | NCT03235037 (ClinicalTrials.gov) | November 26, 2013 | 28/7/2017 | Clinical Trial of Levodopa/Carbidopa ( Sinemet) Therapy in Angel Man Syndrome | Clinical Trial of Levodopa/Carbidopa ( Sinemet) Therapy in Angel Man Syndrome | Angelman Syndrome | Drug: Levodopa | Bennett Lavenstein | NULL | Completed | 18 Months | 16 Years | All | 10 | N/A | NULL |
| 21 | NCT01531582 (ClinicalTrials.gov) | April 2012 | 5/2/2012 | Minocycline in the Treatment of Angelman Syndrome | The Efficacy of Minocycline in the Treatment of Angelman Syndrome | Angelman Syndrome | Drug: minocycline | University of South Florida | NULL | Active, not recruiting | 4 Years | 12 Years | Both | 25 | N/A | United States |
| 22 | NCT01281475 (ClinicalTrials.gov) | January 2011 | 20/1/2011 | A Trial of Levodopa in Angelman Syndrome | A Phase 2 Randomized Placebo-Controlled Trial of Levodopa in Angelman Syndrome | Angelman Syndrome | Drug: Levodopa;Drug: Placebo Oral Capsule | Wen-Hann Tan | Rady Children's Hospital, San Diego;University of California, San Francisco;Baylor College of Medicine;Vanderbilt University Medical Center;Greenwood Genetic Center;Children's Hospital Medical Center, Cincinnati;Angelman Syndrome Foundation, Inc. | Completed | 4 Years | 12 Years | All | 67 | Phase 2/Phase 3 | United States |
| 23 | NCT00829439 (ClinicalTrials.gov) | January 2009 | 26/1/2009 | Study on Tolerability of Levodopa/Carbidopa in Children With Angelman Syndrome | A Dose-escalation Tolerability Study of Levodopa/Carbidopa in Angelman Syndrome | Angelman Syndrome | Drug: Levodopa/Carbidopa (4:1) | Boston Children’s Hospital | NULL | Completed | 4 Years | 12 Years | All | 16 | Phase 1 | United States |
| 24 | NCT00348933 (ClinicalTrials.gov) | July 2006 | 3/7/2006 | Dietary Supplements for the Treatment of Angelman Syndrome | Efficacy of a Therapeutic Treatment Trial in Angelman Syndrome | Angelman Syndrome;Nervous System Diseases | Drug: Betaine;Drug: Creatine;Drug: Metafolin;Drug: Vitamin B12 | University of California, San Diego | Baylor College of Medicine;Rady Children's Hospital, San Diego;Boston Children’s Hospital;Greenwood Genetic Center;Rare Diseases Clinical Research Network | Completed | N/A | 5 Years | All | 90 | N/A | United States |
| 25 | EUCTR2019-004478-24-NL (EUCTR) | 18/03/2020 | 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 Individuals with Angelman Syndrome (ELARA) - ELARA | 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] | Product Name: Gaboxadol monohydrate 0.5 mg Product Code: OV101 INN or Proposed INN: Gaboxadol Other descriptive name: GABOXADOL MONOHYDRATE Product Name: Gaboxadol monohydrate 2 mg Product Code: OV101 INN or Proposed INN: Gaboxadol Other descriptive name: GABOXADOL MONOHYDRATE Product Name: Gaboxadol monohydrate 5 mg Product Code: OV101 INN or Proposed INN: Gaboxadol Other descriptive name: GABOXADOL MONOHYDRATE | Ovid Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | United States;Australia;Israel;Germany;Netherlands |