Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 (NCT01208389) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
United States12 participantsStarted 2010-11
Plain-language summary
The study is a follow-on to a Phase 1 dose-escalation and safety study.
Who can participate
Age range8 Years
SexALL
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Inclusion Criteria:
* Prior participation in Phase 1 study with unilateral, subretinal administration of AAV2-hRPE65v2.
* Visual acuity equal to or greater than light perception.
* Sufficient viable retinal cells in contralateral, previously uninjected eye, as determined by non-invasive means, such as optical coherence tomography (OCT) and/or ophthalmoscopy. Must have either: 1) an area of retina within the posterior pole of \> 100 µm shown on OCT; 2) ≥ 3 disc areas of retina without atrophy or pigmentary degeneration within the posterior pole; or 3) remaining visual field within 50 degrees of fixation.
* Willingness to adhere to protocol and long-term follow-up as evidenced by written informed consent or parental permission and subject assent (where applicable).
Exclusion Criteria:
* Unable or unwilling to meet requirements of the study.
* Participation in any other study of an investigational drug within the past six months.
* Use of retinoid compounds or precursors that could potentially interact with the biochemical activity of the RPE65 enzyme; individuals who discontinue use of these compounds for 18 months may become eligible.
* Prior intraocular surgery within six months.
* Known sensitivity to medications planned for use in the peri-operative period.
* Pre-existing eye conditions, such as glaucoma, or complicating systemic diseases that would preclude the planned surgery or could interfere with the interpretation of study. Complicating systemic diseases would incl…
What they're measuring
1
Adverse events as a measure of safety and tolerability