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Gene Therapy Trial Report

Summary

Trial of Subretinal Injection of (rAAV2-VMD2-hMERTK)


NCTID NCT01482195 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Retinal Disease, Retinitis Pigmentosa
Disease Ontology Term DOID:0110367
Compound Name AAV2-VMD2-hMERTK
Sponsor King Khaled Eye Specialist Hospital
Funder Type Other gov
Recruitment Status
Completed
Enrollment Count 6 (ACTUAL)
Results Posted View Results

Therapy Information


Target Gene/Variant MERTK
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Subretinal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV2
Dose 1 5.96E10 vg/150ul (n=2)
Dose 2 1.788E11 vg/450ul (n=4)

Study Record Dates


Current Stage Phase1
Submit Date 2011-09-28
Completion Date 2019-08
Last Update 2022-01-26

Participation Criteria


Eligible Age 14 Years - 70 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * MERTK-associated retinal disease; * VA: 20/100 or less in worse eye * Ability to perform tests of visual and retinal function; * Good general health based on a complete physical examination and hematology and chemistry studies performed at a pre-treatment evaluation; * Ability to comply with research procedures; Exclusion Criteria: * Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints or surgical complications (for example, glaucoma, corneal or lenticular opacities); * Complicating systemic diseases (such as medical conditions causing immunosuppression) that would preclude the gene transfer, ocular surgery or known sensitivity or allergy to medications planned for use in the peri-operative period; * Use of anti-platelet agents that may alter coagulation within 7 days prior to study agent administration; * Use of immunosuppressive medications; * Pregnancy or breastfeeding; * Individuals (males and females) of childbearing potential who are unwilling to use effective contraception for 1 year following agent administration and barrier contraception for 3 months following agent administration; * Any other condition that would prevent a subject from completing follow-up examinations during the course of the study and that, in the opinion of the investigator, makes the subject unsuitable for the study. * Current, or recent (within the past 30 days, or 10 half lives of the drug) participation, in any other research protocol involving investigational agents or therapies. * Recent (within past 6 months) receipt of an investigational biologic therapeutic agent.Subjects will not be excluded based on their gender, race or ethnicity.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations Saudi Arabia

Regulatory Information


Has US IND
FDA Designations
Recent Updates Therapy was well tolerated, 3/6 patients displayed improved visual acuity, but the effects persisted less than 2 years in 2 of the 3

Resources/Links