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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