201. Angelman syndrome
19 clinical trials,   30 drugs   (DrugBank: 7 drugs),   22 drug target genes,   20 drug target pathways

Searched query = "Angelman syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2019-003787-48-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
2NCT04428281
(ClinicalTrials.gov)
August 29, 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 YearsAll66Phase 1United States;Italy;Netherlands;Spain;Canada;United Kingdom
3EUCTR2019-003787-48-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
4EUCTR2019-004478-24-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
170Phase 3United States;Australia;Israel;Netherlands;Germany
5NCT04259281
(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 IncActive, not recruiting4 Years17 YearsAll20Phase 1;Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2019-002907-17-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
7NCT04106557
(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
8NCT03882918
(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
9NCT03644693
(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
10NCT03109756
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT02996305
(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
12NCT02056665
(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
13EUCTR2013-002154-67-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
14NCT03235037
(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
15NCT01531582
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT01281475
(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
17NCT00829439
(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
18NCT00348933
(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
19EUCTR2019-004478-24-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.NULLNAFemale: yes
Male: yes
200Phase 3United States;Australia;Israel;Germany;Netherlands