Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients

Recruiting Phase 1 Phase 2 Interventional Study
LCA5
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
4 and older
Sex
Any
Trial phase
Phase 1/2
Study type
Interventional
Purpose
Other
Participants needed
22 (estimated)
Sponsor
Opus Genetics, Inc · Industry
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About This Trial

The goals of this clinical trial are assess the natural course of LCA5-IRD over 6 months and to evaluate the safety and preliminary efficacy of subretinal gene therapy with OPGx-001 in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene. Funding Source- FDA Office of Orphan Products Development (OOPD).

Trial Locations
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Eligibility Criteria
Inclusion Criteria: 1. Are willing and able to provide written informed consent (ICF) and, where appropriate, willing to sign an assent prior to any study procedures. 2. Are willing to adhere to the clinical protocol and able to perform testing procedures. 3. In part A participants must be 13 years…
Contacts

Tomas Aleman, M.D.

1 (215) 662-6396

ct.gov_inquiries@opusgtx.com

CONTACT

Mariejel Weber

1 (215) 662-6396

mariejel.weber@pennmedicine.upenn.edu

CONTACT