DDrare: Database of Drug Development for Rare Diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
201	アンジェルマン症候群	1

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	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	GeneTX Biotherapeutics, LLC	Ultragenyx Pharmaceutical Inc	Active, not recruiting	4 Years	17 Years	All	20	Phase 1;Phase 2	United States

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

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

GeneTX Biotherapeutics, LLC

Ultragenyx Pharmaceutical Inc

Active, not recruiting

4 Years

17 Years

All

Phase 1;Phase 2

United States