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Sponsors and Collaborators: |
University of Pennsylvania National Eye Institute (NEI) |
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Information provided by: | University of Pennsylvania |
ClinicalTrials.gov Identifier: | NCT00481546 |
A recombinant adeno-associated virus serotype 2 (rAAV2) vector has been altered to carry the human RPE65 (hRPE65) gene. This vector has been shown to restore vision in animal models that resemble human RPE65-associated Leber congenital amaurosis (LCA), an incurable retinal degeneration that causes severe vision loss. The proposed study is an open label, Phase I clinical trial of subretinal rAAV2-CBSB-hRPE65 administration to individuals with RPE65-associated retinal disease. Three cohorts of three subjects each will be included in this trial. Cohorts 1 and 2 will consist of individuals 18 years of age and older and Cohort 3 will consist of individuals between the ages of 8 and 17, inclusive. Enrollment in Cohort 3 will begin only after confirming the safety of rAAV2-CBSB-hRPE65 administration in the older groups of participants. This trial will lead to a greater understanding of the safety and thereby potential value of gene transfer in RPE65-associated retinal disease and will have implications for other forms of retinal degenerative disease amenable to this type of intervention.
The goal of this clinical trial is to determine the safety of uniocular subretinal administration of rAAV2-CBSB-hRPE65 in individuals with RPE65-associated retinal disease. Ocular and systemic toxicity will be assessed prior to and following vector administration to determine if there are adverse changes that may be associated with vector administration.
Condition | Intervention | Phase |
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Amaurosis of Leber Retinal Diseases |
Genetic: rAAV2-CBSB-hRPE65 |
Phase I |
Study Type: | Interventional |
Study Design: | Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety Study |
Official Title: | Phase I Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus (rAAV2-CBSB-hRPE65) Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations (Clinical Trials of Gene Therapy for Leber Congenital Amaurosis) |
Estimated Enrollment: | 9 |
Study Start Date: | July 2007 |
Estimated Study Completion Date: | July 2022 |
Estimated Primary Completion Date: | July 2022 (Final data collection date for primary outcome measure) |
Ages Eligible for Study: | 8 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contact: Sharon Wolfe-Schwartz, MS, CGC | 215-662-9981 | chrd@uphs.upenn.edu |
United States, Florida | |
Shands Children's Hospital, University of Florida | Recruiting |
Gainesville, Florida, United States, 32610 | |
Contact: Sharon Wolfe-Schwartz, MS, CGC 215-662-9981 chrd@uphs.upenn.edu | |
Principal Investigator: Barry J. Byrne, MD, PhD | |
United States, Pennsylvania | |
Scheie Eye Institute, University of Pennsylvania | Recruiting |
Philadelphia, Pennsylvania, United States, 19104 | |
Contact: Sharon Wolfe-Schwartz, MS, CGC 215-662-9981 chrd@uphs.upenn.edu | |
Principal Investigator: Samuel G. Jacobson, MD, PhD |
Principal Investigator: | Samuel G. Jacobson, MD, PhD | University of Pennsylvania |
Responsible Party: | University of Pennsylvania ( Samuel G. Jacobson, MD, PhD ) |
Study ID Numbers: | 547233, Grant: 1 U10 EY017280-01;, UP IRB: 804582;, UP IBC: 06-105;, UF GCRC: 675;, UF IBC RD: 2795;, WIRB: 20061300 |
Study First Received: | May 31, 2007 |
Last Updated: | January 28, 2009 |
ClinicalTrials.gov Identifier: | NCT00481546 History of Changes |
Health Authority: | United States: Food and Drug Administration |
Leber congenital amaurosis LCA RPE65 Retinal disease due to RPE65 mutations RPE65-associated Leber congenital amaurosis |
Sensation Disorders Vision Disorders Eye Diseases Amaurosis Congenita of Leber Optic Atrophy Blindness Neurodegenerative Diseases Virus Diseases Signs and Symptoms |
Heredodegenerative Disorders, Nervous System Genetic Diseases, Inborn Optic Nerve Disorder Neurologic Manifestations Eye Diseases, Hereditary Atrophy Optic Nerve Diseases Optic Atrophies, Hereditary Retinal Diseases |
Sensation Disorders Vision Disorders Eye Diseases Nervous System Diseases Optic Atrophy Blindness Neurodegenerative Diseases Signs and Symptoms |
Heredodegenerative Disorders, Nervous System Genetic Diseases, Inborn Neurologic Manifestations Eye Diseases, Hereditary Optic Nerve Diseases Cranial Nerve Diseases Optic Atrophies, Hereditary Retinal Diseases |