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"
3 trials found
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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Inclusion_
agemin
Inclusion_
agemax
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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