DDrare: Database of Drug Development for Rare Diseases

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.

Drug = Aknemin; Aknemin 50; Betaine; Carbidopa; Creatine; GABOXADOL MONOHYDRATE; GTX-102; Gaboxadol; Gaboxadol monohydrate 0.5 mg; Gaboxadol monohydrate 0.5 mg (intended commercial formulation); Gaboxadol monohydrate 0.5 mg capsule; Gaboxadol monohydrate 2 mg; Gaboxadol monohydrate 2 mg (intended commercial formulation); Gaboxadol monohydrate 2 mg capsule; Gaboxadol monohydrate 5 mg; Gaboxadol monohydrate 5 mg (intended commercial formulation); Levodopa; Levodopa/Carbidopa (4:1); MINOCYCLINE; MINOCYCLINE HYDROCHLORIDE; Metafolin; Minocycline; Nutritional Formulation Containing Exogenous Ketones; OV101; OV101 Regimen 1; OV101 regimen 2; RO7248824; RO7248824/F02-01; RO7248824/F03-01 (Diluent for RO7248824/F02-01; Vitamin B12

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	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
2	NCT04428281 (ClinicalTrials.gov)	August 29, 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	66	Phase 1	United States;Italy;Netherlands;Spain;Canada;United Kingdom
3	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

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

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

Phase 1

United States;Canada;Spain;Netherlands;United Kingdom;Italy

NCT04428281
(ClinicalTrials.gov)

August 29, 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

Phase 1

United States;Italy;Netherlands;Spain;Canada;United Kingdom

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

F. Hoffmann-La Roche

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 1

United States;Canada;Spain;Netherlands;Italy;United Kingdom