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	NCT04355689 (ClinicalTrials.gov)	September 3, 2020	17/4/2020	Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome	Safety and Efficacy of NPI-001 Tablets Versus Placebo for Treatment of Retinitis Pigmentosa Associated With Usher Syndrome	Usher Syndromes	Drug: NPI-001;Other: Placebo	Nacuity Pharmaceuticals, Inc.	Foundation Fighting Blindness	Recruiting	18 Years	N/A	All	48	Phase 1;Phase 2	Australia

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registration

Public_title

Scientific_title

Condition

Intervention

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sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
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NCT04355689
(ClinicalTrials.gov)

September 3, 2020

17/4/2020

Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome

Safety and Efficacy of NPI-001 Tablets Versus Placebo for Treatment of Retinitis Pigmentosa Associated With Usher Syndrome

Usher Syndromes

Drug: NPI-001;Other: Placebo

Nacuity Pharmaceuticals, Inc.

Foundation Fighting Blindness

Recruiting

18 Years

N/A

All

Phase 1;Phase 2

Australia