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

告示番号

疾患名（ページ内リンク）

臨床試験数

256

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	NCT03533673 (ClinicalTrials.gov)	November 1, 2018	13/4/2018	AAV2/8-LSPhGAA in Late-Onset Pompe Disease	A Phase 1 Study of the Safety of AAV2/8-LSPhGAA in Late-onset Pompe Disease	Pompe Disease	Biological: AAV2/8LSPhGAA	Asklepios Biopharmaceutical, Inc.	Duke University;National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)	Recruiting	18 Years	N/A	All	8	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

November 1, 2018

13/4/2018

AAV2/8-LSPhGAA in Late-Onset Pompe Disease

A Phase 1 Study of the Safety of AAV2/8-LSPhGAA in Late-onset Pompe Disease

Pompe Disease

Biological: AAV2/8LSPhGAA

Asklepios Biopharmaceutical, Inc.

Duke University;National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Recruiting

18 Years

N/A

All

Phase 1;Phase 2

United States