Trial of Subretinal Injection of (rAAV2-VMD2-hMERTK)
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|ClinicalTrials.gov Identifier: NCT01482195|
Recruitment Status : Completed
First Posted : November 30, 2011
Results First Posted : July 15, 2021
Last Update Posted : January 26, 2022
|Condition or disease||Intervention/treatment||Phase|
|Retinal Disease Retinitis Pigmentosa||Biological: Subretinal administration of rAAV2-VMD2-hMERTKRecombinant Adeno-Associated Virus||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||6 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Ocular Subretinal administration of rAAV2-VMD2-hMERTK .|
|Masking:||None (Open Label)|
|Official Title:||Phase I Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus (rAAV2-VMD2-hMERTK) Gene Vector to Patients With Retinal Disease Due to MERTK Mutations|
|Actual Study Start Date :||August 2011|
|Actual Primary Completion Date :||August 2013|
|Actual Study Completion Date :||August 2019|
Experimental: Subretinal Injection of rAAV2-VMD2-hMERTKRecombinant Adeno-Associated Virus
Single arm of 6 patients undergoing subretinal injection of Gene Therapy using rAAV2-VMD2-hMERTKRecombinant Adeno-Associated Virus. Each patient received the injection in one eye.
Biological: Subretinal administration of rAAV2-VMD2-hMERTKRecombinant Adeno-Associated Virus
The study is an open-label, dose-escalation, phase I clinical trial of subretinal administration of rAAV2-VMD2-hMERTK in patients with retinitis pigmentosa due to MERTK mutation.
No Intervention: fellow eye without intervention
fellow eye without intervention
- Systemic and Ocular Safety [ Time Frame: 2 years ]
Detailed history & physical exam were obtained at baseline visit and each post-injection protocol visit searching for systemic adverse events. Included were electrocardiogram, chest X-ray, complete blood count & differential, prothrombin time & INR, partial thromboplastin time, serum electrolytes, full serum chemistries including liver and renal function, urinalysis; serum antibody titers to AAV2 capsid components and antigen-specific reactivity (ASR) assays; blood analysis by DNA PCR to detect vector spread.
Ophthalmic safety monitored changes from baseline included 1) corneal abnormalities, afferent pupillary defect, intraocular inflammation, cataract & intraocular pressure changes; 2) retinal changes based on fundus photos; 3) Macular SD-OCT changes in Central macular (CMT) and central foveal thickness (CFT) measurements when patient fixation allowed it, and 4) full field stimulus threshold (FST) to detect any retinal toxicity .
- Visual Acuity Measurement [ Time Frame: 2 years and up to 5 years ]Although candidates may have very severe loss of function, an attempt was made to measure a best-corrected visual acuity using Early Treatment Diabetic Retinopathy Study (ETDRS) charts, and measurements were recorded as the number of letters read on each line of the chart (Diabetic Retinopathy Study Research Group 1985). If a patient was unable to read at least three letters of the first line correctly, the chart distance was progressively halved from the standard 4 m until either the first line was correctly read or the shortest distance of 0.5 m was reached. Patients who were unable to read any letters on the chart were tested for light perception and if they perceived light they were assigned the acuity score equivalent of <20/6400. Measurements were performed at baseline and each protocol follow up visit. Improvement in patients who could read was defined as a gain in 5 letters, and in those with those LP vision only to start seeing hand motion.
- Full-field Stimulus Threshold Testing (FST). [ Time Frame: 2 years ]Full-field stimulus threshold testing (FST) measures sensitivity of the entire visual field by estimating the lowest luminance of a flash that elicits a visual sensation. The FST measurements were performed at baseline and throughout the protocol visits over two years in the study and fellow eyes, and changes in FST results were analyzed.
- Central Foveal Thickness (CFT) on Optical Coherence Tomography (OCT). [ Time Frame: 2 years ]Central foveal thickness (CFT) measurements were performed at baseline and throughout the protocol visits over two years in the study and fellow eyes (whenever possible), and changes in CFT values were analyzed.
- Central Macular Thickness (CMT) on Optical Coherence Tomography (OCT). [ Time Frame: 2 years ]Central macular thickness (CMT) measurements were performed at baseline and throughout the protocol visits over two years in the study and fellow eyes (whenever possible), and changes in CMT values were analyzed.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01482195
|King Khaled Eye Specialist Hospital|
|Riyadh, Saudi Arabia, 11462|
|Principal Investigator:||Fowzan S Alkuraya, MD||King Faisal Specialist Hospital & Research Center|