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
256	筋型糖原病	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	NCT03687333 (ClinicalTrials.gov)	December 4, 2018	24/9/2018	Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment	A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment	Glycogen Storage Disease Type II	Drug: ALGLUCOSIDASE ALFA (MYOZYME)	Genzyme, a Sanofi Company	NULL	Active, not recruiting	N/A	12 Months	All	10	Phase 4	China

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

NCT03687333
(ClinicalTrials.gov)

December 4, 2018

24/9/2018

Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment

A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment

Glycogen Storage Disease Type II

Drug: ALGLUCOSIDASE ALFA (MYOZYME)

Genzyme, a Sanofi Company

NULL

Active, not recruiting

N/A

12 Months

All

Phase 4

China