A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa



Status:Recruiting
Conditions:Ocular
Therapuetic Areas:Ophthalmology
Healthy:No
Age Range:10 - Any
Updated:3/8/2019
Start Date:March 16, 2017
End Date:August 2020
Contact:Nightstar Therapeutics
Email:researchenquiries@nightstartx.com
Phone:+44 207 611 2077

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A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)

A clinical trial of AAV8-RPGR retinal gene therapy for patients with X-linked retinitis
pigmentosa

This is a Phase 1/2/3, first-in-human, multi-centre, dose-escalation interventional study of
AAV8-RPGR in male subjects with genetically confirmed XLRP. Part I is a dose-selection study;
Part II is a dose-expansion study, comparing 2 doses, and a third untreated group to allow
for a controlled comparison of efficacy and safety.

Key inclusion Criteria:

- Male

- Part I: >= 18 years

- Part II: >= 10 years

- Documentation of a mutation in the RPGR gene

Key exclusion Criteria:

- participated in a gene therapy trial previously or a clinical trial with an
investigational drug in the past 12 weeks
We found this trial at
5
sites
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mi
from
Gainesville, FL
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from
Manchester,
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Miami, FL
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mi
from
Philadelphia, PA
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mi
from
Portland, OR
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