Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
NCT ID: NCT00749957
Last Updated: 2017-12-28
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE1/PHASE2
12 participants
INTERVENTIONAL
2009-06-17
2017-09-22
Brief Summary
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Funding Source - FDA OOPD
Detailed Description
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Enrollment will begin with Group 1 and will proceed to Group 2 after review of safety data by a Data and Safety Monitoring Committee.
Safety will be monitored by evaluation of ocular and non ocular adverse events and hematology and clinical chemistry parameters. Efficacy will be measured by evaluation of visual fields, visual acuity and electroretinography.
Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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1
Subjects at least 6 y/o treated with a lower dose of the vector by subretinal injection
rAAV2-CB-hRPE65
Recombinant adeno-associated virus vector expressing RPE65
2
Subjects at least 6 y/o treated with a higher dose of the vector by subretinal injection
rAAV2-CB-hRPE65
Recombinant adeno-associated virus vector expressing RPE65
Interventions
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rAAV2-CB-hRPE65
Recombinant adeno-associated virus vector expressing RPE65
Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
6 Years
ALL
No
Sponsors
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Oregon Health and Science University
OTHER
University of Massachusetts, Worcester
OTHER
Beacon Therapeutics
INDUSTRY
Responsible Party
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Principal Investigators
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J Timothy Stout, MD, PhD, MBA
Role: PRINCIPAL_INVESTIGATOR
Casey Eye Institute, Oregon Health & Science University
Locations
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University of Massachusetts Medical School
Worcester, Massachusetts, United States
Casey Eye Institue, Oregon Health & Science University
Portland, Oregon, United States
Countries
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References
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Acland GM, Aguirre GD, Bennett J, Aleman TS, Cideciyan AV, Bennicelli J, Dejneka NS, Pearce-Kelling SE, Maguire AM, Palczewski K, Hauswirth WW, Jacobson SG. Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness. Mol Ther. 2005 Dec;12(6):1072-82. doi: 10.1016/j.ymthe.2005.08.008. Epub 2005 Oct 14.
Jacobson SG, Boye SL, Aleman TS, Conlon TJ, Zeiss CJ, Roman AJ, Cideciyan AV, Schwartz SB, Komaromy AM, Doobrajh M, Cheung AY, Sumaroka A, Pearce-Kelling SE, Aguirre GD, Kaushal S, Maguire AM, Flotte TR, Hauswirth WW. Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis. Hum Gene Ther. 2006 Aug;17(8):845-58. doi: 10.1089/hum.2006.17.845.
Weleber RG, Pennesi ME, Wilson DJ, Kaushal S, Erker LR, Jensen L, McBride MT, Flotte TR, Humphries M, Calcedo R, Hauswirth WW, Chulay JD, Stout JT. Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy. Ophthalmology. 2016 Jul;123(7):1606-20. doi: 10.1016/j.ophtha.2016.03.003. Epub 2016 Apr 19.
Ku CA, Igelman AD, Huang SJ, Bailey ST, Lauer AK, Duncan JL, Weleber RG, Yang P, Pennesi ME. Perimacular Atrophy Following Voretigene Neparvovec-Rzyl Treatment in the Setting of Previous Contralateral Eye Treatment With a Different Viral Vector. Transl Vis Sci Technol. 2024 Jun 3;13(6):11. doi: 10.1167/tvst.13.6.11.
Pennesi ME, Weleber RG, Yang P, Whitebirch C, Thean B, Flotte TR, Humphries M, Chegarnov E, Beasley KN, Stout JT, Chulay JD. Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy. Hum Gene Ther. 2018 Dec;29(12):1428-1437. doi: 10.1089/hum.2018.014. Epub 2018 Jul 24.
Related Links
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Applied Genetic Technologies Corporation home page
Other Identifiers
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AGTC-RPE65-002
Identifier Type: -
Identifier Source: org_study_id