Status:
COMPLETED
Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations
Lead Sponsor:
Hadassah Medical Organization
Conditions:
Leber Congenital Amaurosis
Eligibility:
All Genders
8+ years
Phase:
PHASE1
Brief Summary
The purpose of this clinical trial is to examine the safety of gene therapy for Lebers Congenital Amaurosis (LCA) caused by RPE65 mutations using a recombinant adeno-associated virus serotype 2 (rAAV2...
Eligibility Criteria
Inclusion
- Retinal disease caused by homozygous or compound heterozygote RPE65 mutations;
- Clinical diagnosis of Leber congenital amaurosis (LCA) with severely impaired visual and retinal function, and best corrected visual acuity of 20/50 or worse in the study eye;
- Ability to perform tests of visual and retinal function;
- Good general health;
- Ability to comply with research procedures;
- Specific for Cohort 1 and 2: 18 years of age and older;
- Specific for Cohort 3: Over 8 years of age;
Exclusion
- Immune deficiency or use of immunosuppressive medications;
- Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints (for example, glaucoma or ocular media opacities);
- Complicating systemic diseases;
- Impaired coagulation or use of anti-platelet agents within 7 days prior to study agent administration;
- 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;
- Any other condition that, in the opinion of the investigator, makes the subject unsuitable for the study;
- Current or recent participation in any other research protocol involving investigational agents or therapies, including recent (within past 6 months) receipt of an investigational biologic therapeutic agent.
- Subjects will not be excluded based on their gender, race or ethnicity.
Key Trial Info
Start Date :
February 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2017
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT00821340
Start Date
February 1 2009
End Date
January 1 2017
Last Update
April 10 2018
Active Locations (1)
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1
Hadassah Medical Organization
Jerusalem, Israel, 91120