DDrare: Database of Drug Development for Rare Diseases

303. アッシャー症候群
［臨床試験数：6，薬物数：11（DrugBank：0），標的遺伝子数：0，標的パスウェイ数：0］

Searched query = "Usher syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = Blood draw for the laboratory assessment; Lentiviral vector containing MYO7A gene; Lentiviral vector containing the human MY07A gene; NPI-001; NT-501; Other: Sham-procedure (dose cohort 1&2 only); Procedure: Sham; QR-421a; SAR421869; UshStat; UshStat (SAR421869)

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	NCT02065011 (ClinicalTrials.gov)	September 12, 2013	14/2/2014	A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B	An Open-label Study to Determine the Long-term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B	Usher's Syndrome	Drug: Blood draw for the laboratory assessment	Sanofi	NULL	Active, not recruiting	18 Years	N/A	All	9	Phase 1;Phase 2	United States;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

NCT02065011
(ClinicalTrials.gov)

September 12, 2013

14/2/2014

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B

An Open-label Study to Determine the Long-term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B

Usher's Syndrome

Drug: Blood draw for the laboratory assessment

Sanofi

NULL

Active, not recruiting

18 Years

N/A

All

Phase 1;Phase 2

United States;France