An investigator-initiated phase I/IIa trial of subretinal injection of DVC1-0401 as neuroprotective gene therapy for retinitis pigmentosa
- Conditions
- Retinitis pigmentosa
- Registration Number
- JPRN-UMIN000034081
- Lead Sponsor
- Kyushu University Hospital
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete: follow-up continuing
- Sex
- All
- Target Recruitment
- 12
Not provided
1) HIV antibody-positive 2) Decimal visual acuities of both eyes are less than 0.1, or lateral or bilateral blindness 3) Cone-rod dystrophy 4) Interocular difference in the condition of lens 5) Co-existence of glaucoma or ocular hypertension (intraocular pressure is more than or equal to 22 mmHg) 6) Co-existence of uveitis or optic neuritis 7) Retinal or subretinal pathological change observed by fundus examination (including fluorescein fundus angiography, laser scanning fundus photography), that is independent of retinitis pigmentosa 8) Visual field measured by Goldmann perimeter V-4e is less than 10 degree at the center area of vision 9) Loss or massive structural irregularity of the layer of retinal pigment epithelium on OCT (optical coherence tomography) at the supposed site of administration 10) Having severe allergy or previous history 11) Receiving chronic hemodialysis therapy 12) Severe heart dysfunction or heart failure 13) Sever hepatic dysfunction or liver cirrhosis 14) Active inflammatory diseases 15) History of cerebral hemorrhage or infarction within 6 months before agreement 16) Hematopoietic disorders 17) Alcoholism and/or drug dependence 18) Female subjects with pregnant or doubt of pregnancy. Breast-feeding female. 19) Subjects who are diagnosed with cancer or suspected to have cancer. History of treatment of malignancies within 5 years. 20) Disapprove of birth control during at least 12 months after the administration 21) Administration of Helenien, Unoprostone, and/or Ca antagonist intended for treatment of eye disease within 30 days before agreement 22) Taking the antiviral drug at the registration 23) Plan of operation other than administration of the investigational drug during this clinical trial 24) Entry into other clinical trial and/or clinical study, or history of entry within 6 months before agreement 25) Others: Subjects who are judged to be inappropriate by principal investigator or subinvestigator
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Incidence of adverse events
- Secondary Outcome Measures
Name Time Method 1) Rate of change of the mean retinal sensitivity (central 12 points) : Primary outcome measure of efficiency 2) Rate of change of the mean retinal sensitivity (central 4 points) : Secondary outcome measure of efficiency-1 3) Rate of change of the visual acuity (logMAR converted value) : Secondary outcome measure of efficiency-2 4) Amount of hPEDF protein in the aqueous humor : Exploratory outcome measure