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

  
No.TrialIDDate_
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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Secondary_
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Recruitment_
Status
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agemin
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PhaseCountries
1NCT05127226
(ClinicalTrials.gov)
December 22, 20219/11/2021HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman SyndromeHALOS: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients With Angelman SyndromeAngelman SyndromeDrug: ION582Ionis Pharmaceuticals, Inc.NULLRecruiting2 Years50 YearsAll44Phase 1/Phase 2United States
2NCT05011851
(ClinicalTrials.gov)
October 1, 20215/8/2021An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman SyndromeAn Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman SyndromeAngelman SyndromeDrug: NNZ-2591Neuren Pharmaceuticals LimitedNULLNot yet recruiting3 Years17 YearsAll20Phase 2Australia
3NCT04863794
(ClinicalTrials.gov)
April 29, 202126/4/2021A Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male ParticipantsA 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 ParticipantsAngelman SyndromeDrug: RO7248824Hoffmann-La RocheNULLRecruiting25 Years55 YearsMale26Phase 1Netherlands
4NCT04768803
(ClinicalTrials.gov)
March 15, 202116/2/2021Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and/or Overweight, and/or ObesityCirculating Levels of Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and / or Overweight, and / or ObesityAngelman Syndrome;Smith-Magenis Syndrome;X Fragile Syndrome;Epilepsy;Prader-Willi SyndromeBiological: acylated and unacylated ghrelin dosagesUniversity Hospital, ToulouseNULLNot yet recruiting3 Years50 YearsAll300NULL
5EUCTR2019-003787-48-NL
(EUCTR)
22/09/202008/10/2020Angelman 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 RocheNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
66Phase 1United States;Canada;Spain;Netherlands;United Kingdom;Italy
6NCT04428281
(ClinicalTrials.gov)
August 19, 20209/6/2020A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman SyndromeAn Open-Label, Multicenter Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman SyndromeAngelman SyndromeDrug: RO7248824Hoffmann-La RocheNULLRecruiting1 Year12 YearsAll140Phase 1United States;Italy;Netherlands;Spain;Canada;United Kingdom
7EUCTR2019-003787-48-IT
(EUCTR)
03/07/202015/06/2021Angelman 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
66Phase 1United States;Canada;Spain;Netherlands;United Kingdom;Italy
8EUCTR2019-003787-48-GB
(EUCTR)
11/06/202016/03/2020Angelman 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 RocheNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
66Phase 1United States;Canada;Spain;Netherlands;Italy;United Kingdom
9EUCTR2019-004478-24-DE
(EUCTR)
05/06/202026/02/2020An 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.NULLNot RecruitingFemale: yes
Male: yes
170Phase 3United States;Australia;Israel;Netherlands;Germany
10NCT04259281
(ClinicalTrials.gov)
February 24, 202031/1/2020A Study of the Safety and Tolerability of GTX-102 in Children With Angelman SyndromeA 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 SyndromeDrug: GTX-102GeneTX Biotherapeutics, LLCUltragenyx Pharmaceutical IncRecruiting4 Years17 YearsAll20Phase 1/Phase 2United States;Canada;United Kingdom
11EUCTR2019-002907-17-NL
(EUCTR)
19/02/202010/12/2019Clinical 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.NULLNot RecruitingFemale: yes
Male: yes
95Phase 3United States;Australia;Israel;Germany;Netherlands;Italy
12NCT04106557
(ClinicalTrials.gov)
September 9, 201925/9/2019A 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 SyndromePrimary Disease or Condition Being Studied: Angelman Syndrome (AS)Drug: Gaboxadol;Drug: PlaceboOvid Therapeutics Inc.NULLCompleted2 Years12 YearsAll104Phase 3United States;Australia;Germany;Israel;Netherlands
13NCT03882918
(ClinicalTrials.gov)
February 25, 201918/3/2019An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman SyndromeAn Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman SyndromeAngelman SyndromeDrug: OV101Ovid Therapeutics Inc.NULLRecruiting13 Years49 YearsAll82Phase 2United States;Israel
14NCT03644693
(ClinicalTrials.gov)
November 1, 201813/8/2018Nutritional Formulation for Angelman SyndromeEvaluation of the Safety and Tolerability of a Nutritional Formulation in Angelman SyndromeAngelman SyndromeDietary Supplement: Nutritional Formulation Containing Exogenous Ketones;Dietary Supplement: Placebo FormulationUniversity of Colorado, DenverNULLCompleted4 Years11 YearsAll15N/AUnited States
15NCT03109756
(ClinicalTrials.gov)
April 3, 20176/4/2017Single Dose Pharmacokinetic (PK) StudyA Phase 1 Single Dose PK Study in Adolescent Subjects With Fragile X Syndrome or Angelman SyndromeAngelman Syndrome;Fragile X SyndromeDrug: OV101Ovid Therapeutics Inc.NULLCompleted13 Years17 YearsAll12Phase 1United States
16NCT02996305
(ClinicalTrials.gov)
January 20162/12/2016A Study in Adults and Adolescents With Angelman SyndromeA Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of GaboxadolAngelman SyndromeDrug: OV101 Regimen 1;Drug: OV101 regimen 2;Other: PlaceboOvid Therapeutics Inc.NULLCompleted13 Years49 YearsAll88Phase 2United States;Israel
17NCT02056665
(ClinicalTrials.gov)
January 20145/2/2014Study to Evaluate the Efficacy and Safety of Minocycline in Angelman SyndromeRandomized Clinical Trial, Placebo Compared to Evaluate the Efficacy and Safety of Minocycline in Angelman SyndromeAngelman SyndromeDrug: MINOCYCLINE;Drug: PLACEBO (for Minocycline)Puerta de Hierro University HospitalNULLCompleted6 Years30 YearsBoth32Phase 2Spain
18EUCTR2013-002154-67-ES
(EUCTR)
26/12/201316/09/2013STUDY 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 SoláNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Spain
19NCT03235037
(ClinicalTrials.gov)
November 26, 201328/7/2017Clinical Trial of Levodopa/Carbidopa ( Sinemet) Therapy in Angel Man SyndromeClinical Trial of Levodopa/Carbidopa ( Sinemet) Therapy in Angel Man SyndromeAngelman SyndromeDrug: LevodopaBennett LavensteinNULLCompleted18 Months16 YearsAll10N/ANULL
20NCT01531582
(ClinicalTrials.gov)
April 20125/2/2012Minocycline in the Treatment of Angelman SyndromeThe Efficacy of Minocycline in the Treatment of Angelman SyndromeAngelman SyndromeDrug: minocyclineUniversity of South FloridaNULLActive, not recruiting4 Years12 YearsBoth25N/AUnited States
21NCT01281475
(ClinicalTrials.gov)
January 201120/1/2011A Trial of Levodopa in Angelman SyndromeA Phase 2 Randomized Placebo-Controlled Trial of Levodopa in Angelman SyndromeAngelman SyndromeDrug: Levodopa;Drug: Placebo Oral CapsuleWen-Hann TanRady 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.Completed4 Years12 YearsAll67Phase 2/Phase 3United States
22NCT00829439
(ClinicalTrials.gov)
January 200926/1/2009Study on Tolerability of Levodopa/Carbidopa in Children With Angelman SyndromeA Dose-escalation Tolerability Study of Levodopa/Carbidopa in Angelman SyndromeAngelman SyndromeDrug: Levodopa/Carbidopa (4:1)Boston Children’s HospitalNULLCompleted4 Years12 YearsAll16Phase 1United States
23NCT00348933
(ClinicalTrials.gov)
July 20063/7/2006Dietary Supplements for the Treatment of Angelman SyndromeEfficacy of a Therapeutic Treatment Trial in Angelman SyndromeAngelman Syndrome;Nervous System DiseasesDrug: Betaine;Drug: Creatine;Drug: Metafolin;Drug: Vitamin B12University of California, San DiegoBaylor College of Medicine;Rady Children's Hospital, San Diego;Boston Children’s Hospital;Greenwood Genetic Center;Rare Diseases Clinical Research NetworkCompletedN/A5 YearsAll90N/AUnited States
24EUCTR2019-004478-24-NL
(EUCTR)
18/03/2020An 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.NULLNot RecruitingFemale: yes
Male: yes
200Phase 3United States;Australia;Israel;Germany;Netherlands