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Gene Therapy Trial Report

Summary

Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis


NCTID NCT00749957 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Leber Congenital Amaurosis-Type 2
Disease Ontology Term DOID:0110016
Compound Name RAAV2-CB-hRPE65
Sponsor Beacon Therapeutics
Funder Type Industry
Recruitment Status
Completed
Enrollment Count 12 (ACTUAL)
Results Posted View Results

Therapy Information


Target Gene/Variant RPE65
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 1.8E11 vg/eye
Dose 2 6E11 vg/eye

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2008-09-08
Completion Date 2017-09-22
Last Update 2017-12-28

Participation Criteria


Eligible Age >=6 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Retinal disease consistent with a diagnosis of Leber congenital amaurosis and documented mutations in the RPE65 gene (including null mutations and mutations that code for abnormal RPE65 protein); * At least 6 years of age; * Good general health without significant physical examination findings or clinically significant abnormal laboratory results; * Able to perform tests of visual and retinal function; * Visual acuity not better than 20/60 and not worse than hand motion in both the treated eye and the fellow eye; * Visible photoreceptor (outer nuclear) layer on a standard optical coherence tomography (OCT) scan; * Acceptable hematology, clinical chemistry and urine laboratory parameters; * For females of childbearing potential, a negative pregnancy test at screening and at baseline, and agreement to use effective contraception for 12 months after administration of rAAV2-CB-hRPE65, for sexual activity that could lead to pregnancy; * For males of reproductive potential, agreement to use effective contraception for 12 months after administration of rAAV2-CB-hRPE65, for sexual activity that could lead to pregnancy Exclusion Criteria: * Pre-existing eye conditions that would preclude the planned surgery or interfere with interpretation of study endpoints or complications of surgery (e.g. glaucoma, corneal or lenticular opacities, or history or retinal detachment); * Presence of epiretinal membrane on OCT; * History of immunodeficiency or other medical conditions that might increase the risk of rAAV2-CB-hRPE65 administration; * Use of anticoagulants or anti-platelet agents within 7 days prior to study agent administration; * History of allergy or sensitivity to medications planned for use in the peri-operative period; * For females of childbearing potential, a positive pregnancy test at screening or baseline (within 2 days before rAAV2-CB-hRPE65 administration); * Females who are breast feeding; * Use of any investigational agent, or systemic corticosteroids or other immunosuppressive drug(s), within 3 months prior to enrollment; * Prior receipt of any AAV gene therapy product; * Any condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 2
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Company discontinued development due to competing therapies for the same indication

Resources/Links