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Gene Therapy Trial Report

Summary

Study to Evaluate Safety of RTx-015 Injection in Retinitis Pigmentosa or Choroideremia Patients (ENVISION)


NCTID NCT06460844 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Retinitis Pigmentosa, Choroideremia
Disease Ontology Term DOID:10584; DOID:9821
Compound Name RTx-015
Sponsor Ray Therapeutics, Inc.
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 10 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant Codon optimized ChR-3M
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intravitreal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV.7m8
Editor Type none
Dose 1 Undisclosed low dose
Dose 2 Undisclosed medium dose
Dose 3 Undisclosed high dose

Study Record Dates


Current Stage Phase1
Submit Date 2024-06-10
Completion Date 2030-10
Last Update 2026-02-20

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Adults ≥18 years of age * Informed consent obtained from the patient * Clinical diagnosis of Retinitis Pigmentosa independent of causative mutation OR Clinical diagnosis of Choroideremia with known causative mutation * Study eye and fellow eye Best Corrected Visual Acuity meeting study criteria * Presence of retinal ganglion cells and/or retinal nerve fiber layer on Spectral Domain Optical Coherence Tomography testing at Screening confirmed by central image reading center * Adequate organ function and general good health Exclusion Criteria: * Participation in a clinical study (ocular or non-ocular) with an investigational drug, agent, or therapy in the past six months * Concurrent participation in another interventional clinical ocular study * Prior receipt of any gene therapy (ocular or other), retinal implant, or ocular cell therapy * Pre-existing eye conditions in either eye that would preclude the planned treatment or, in the opinion of the Investigator, are significant enough to interfere with the interpretation of study endpoints or procedural complications * Known steroid responders if their intraocular pressure was not able to be managed effectively with topical pressure-lowering medications after prior use of steroid medications * Complicating systemic diseases; complicating systemic diseases include those in which the disease itself, or the treatment for the disease, can alter ocular and/or Central Nervous System (CNS) function (e.g., radiation treatment of the orbit; leukemia with CNS/optic nerve involvement) * Any immunological response dysfunction including, immuno-compromising diseases or use of immunosuppressive medications, among others * Cataract or other ocular (including refractive) surgery, intraocular and/or peri-ocular injection in either eye within the prior four months (i.e., 120 days) prior to screening * Prior vitrectomy or aphakia in the study eye * Known sensitivity to any component of the study treatment or contraindication to medications planned for use in the peri-procedural period (e.g., povidone-iodine to prep for intravitreal injection) * Known contraindication to prophylactic steroid regimen * Current pregnancy or breastfeeding * Any other condition that would not allow the patient to complete follow-up examinations during the study
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 4
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates

Resources/Links