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
19	ライソゾーム病	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	NCT02843035 (ClinicalTrials.gov)	January 2017	20/7/2016	Venglustat in Combination With Cerezyme in Adult and Pediatric Patients With Gaucher Disease Type 3	A 3-part Study to Evaluate the Efficacy and Safety of Venglustat in Combination With Cerezyme in Adult and Pediatric Patients With Gaucher Disease Type 3 (GD3) With Open-label Long-term Treatment	Gaucher Disease Type 1-Gaucher Disease Type 3	Drug: placebo;Drug: venglustat (GZ/SAR402671);Drug: imiglucerase	Genzyme, a Sanofi Company	NULL	Recruiting	12 Years	N/A	All	49	Phase 2;Phase 3	United States;Germany;Japan;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

NCT02843035
(ClinicalTrials.gov)

January 2017

20/7/2016

Venglustat in Combination With Cerezyme in Adult and Pediatric Patients With Gaucher Disease Type 3

A 3-part Study to Evaluate the Efficacy and Safety of Venglustat in Combination With Cerezyme in Adult and Pediatric Patients With Gaucher Disease Type 3 (GD3) With Open-label Long-term Treatment

Gaucher Disease Type 1-Gaucher Disease Type 3

Drug: placebo;Drug: venglustat (GZ/SAR402671);Drug: imiglucerase

Genzyme, a Sanofi Company

NULL

Recruiting

12 Years

N/A

All

Phase 2;Phase 3

United States;Germany;Japan;United Kingdom