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
90	網膜色素変性症	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	NCT03328130 (ClinicalTrials.gov)	November 6, 2017	5/10/2017	Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene	Safety and Efficacy of a Unilateral Subretinal Administration of HORA-PDE6B in Patients With Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expression	Retinitis Pigmentosa	Biological: AAV2/5-hPDE6B	Horama S.A.	NULL	Recruiting	18 Years	N/A	All	15	Phase 1;Phase 2	France

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

NCT03328130
(ClinicalTrials.gov)

November 6, 2017

5/10/2017

Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene

Safety and Efficacy of a Unilateral Subretinal Administration of HORA-PDE6B in Patients With Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expression

Retinitis Pigmentosa

Biological: AAV2/5-hPDE6B

Horama S.A.

NULL

Recruiting

18 Years

N/A

All

Phase 1;Phase 2

France