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Virus Therapy

Gene Therapy for Leber Congenital Amaurosis

Phase 1 & 2
Waitlist Available
Research Sponsored by Spark Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Prior participation in Phase 1 study with unilateral, subretinal administration of AAV2-hRPE65v2
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
Must not have
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 include those in which the disease itself, or the treatment for the disease, can alter ocular function. Examples are malignancies whose treatment could affect central nervous system function (for example: radiation treatment of the orbit; leukemia with CNS/optic nerve involvement). Subjects with diabetes or sickle cell disease would be excluded if they had any manifestation of advanced retinopathy (e.g. macular edema or proliferative changes). Also excluded would be subjects with immunodeficiency (acquired or congenital) as there could be susceptibility to opportunistic infection (such as CMV retinitis)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 15 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is to assess the safety and effectiveness of an experimental drug in people with a specific type of cancer.

Who is the study for?
This trial is for individuals with Leber Congenital Amaurosis who have participated in a prior Phase 1 study and received treatment in one eye. They must have some visual acuity, viable retinal cells in the other eye, and be willing to follow long-term protocols. Pregnant individuals or those not using effective contraception are excluded, as well as anyone with conditions that could affect the study's outcome.
What is being tested?
The trial is testing voretigene neparvovec-rzyl, a gene therapy previously administered to one eye of participants. Now it will be given to their other eye which hasn't been treated yet. The focus is on safety following this second administration.
What are the potential side effects?
While specific side effects aren't listed here, typical risks may include immune reactions due to the viral vector used for gene delivery, inflammation inside the eye, changes in intraocular pressure or vision disturbances post-treatment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have previously been in a study where I received a specific gene therapy in one eye.
Select...
My other eye has enough healthy retina cells, confirmed by eye scans.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I do not have eye conditions or systemic diseases that could affect my eyes or interfere with the surgery.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~15 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 15 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Adverse events as a measure of safety and tolerability
Secondary study objectives
Contrast sensitivity
Full-field light threshold sensitivity testing
Mobility testing
+3 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: voretigene neparvovec-rzyl (AAV2-hRPE65v2)Experimental Treatment1 Intervention
Administration of study agent (AAV2-hRPE65v2) to the previously, uninjected contralateral eye:
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
voretigene neparvovec-rzyl
2007
Completed Phase 1
~20

Find a Location

Who is running the clinical trial?

Spark Therapeutics, Inc.Lead Sponsor
14 Previous Clinical Trials
342 Total Patients Enrolled
2 Trials studying Leber Congenital Amaurosis
43 Patients Enrolled for Leber Congenital Amaurosis
Spark TherapeuticsLead Sponsor
13 Previous Clinical Trials
342 Total Patients Enrolled
2 Trials studying Leber Congenital Amaurosis
43 Patients Enrolled for Leber Congenital Amaurosis
Clinical DirectorStudy DirectorSpark Therapeutics, Inc.
19 Previous Clinical Trials
4,814 Total Patients Enrolled
1 Trials studying Leber Congenital Amaurosis
12 Patients Enrolled for Leber Congenital Amaurosis

Media Library

voretigene neparvovec-rzyl (Virus Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT01208389 — Phase 1 & 2
Leber Congenital Amaurosis Research Study Groups: voretigene neparvovec-rzyl (AAV2-hRPE65v2)
Leber Congenital Amaurosis Clinical Trial 2023: voretigene neparvovec-rzyl Highlights & Side Effects. Trial Name: NCT01208389 — Phase 1 & 2
voretigene neparvovec-rzyl (Virus Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01208389 — Phase 1 & 2
~3 spots leftby Mar 2030
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