DDrare: Database of Drug Development for Rare Diseases

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	NCT01530659 (ClinicalTrials.gov)	January 2012	19/1/2012	Retinal Imaging of Subjects Implanted With Ciliary Neurotrophic Factor (CNTF)-Releasing Encapsulated Cell Implant for Early-stage Retinitis Pigmentosa	Photoreceptor Structure in A Phase 2 Study of Encapsulated Human NTC-201 Cell Implants Releasing Ciliary Neurotrophic Factor (CNTF) for Participants With Retinitis Pigmentosa Using Rates of Change in Cone Spacing and Density	Retinitis Pigmentosa;Usher Syndrome Type 2;Usher Syndrome Type 3	Drug: NT-501;Procedure: Sham	Neurotech Pharmaceuticals	University of California, San Francisco;FDA Office of Orphan Products Development	Unknown status	18 Years	55 Years	All	30	Phase 2	United States

No.

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registration

Public_title

Scientific_title

Condition

Intervention

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sponsor

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sponsor

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Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

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NCT01530659
(ClinicalTrials.gov)

January 2012

19/1/2012

Retinal Imaging of Subjects Implanted With Ciliary Neurotrophic Factor (CNTF)-Releasing Encapsulated Cell Implant for Early-stage Retinitis Pigmentosa

Photoreceptor Structure in A Phase 2 Study of Encapsulated Human NTC-201 Cell Implants Releasing Ciliary Neurotrophic Factor (CNTF) for Participants With Retinitis Pigmentosa Using Rates of Change in Cone Spacing and Density

Retinitis Pigmentosa;Usher Syndrome Type 2;Usher Syndrome Type 3

Drug: NT-501;Procedure: Sham

Neurotech Pharmaceuticals

University of California, San Francisco;FDA Office of Orphan Products Development

Unknown status

18 Years

55 Years

All

Phase 2

United States