Phase 1
N=6
Trial of Subretinal Injection of (rAAV2-VMD2-hMERTK)
Retinal Disease · Retinitis Pigmentosa
Bottom Line
View on ClinicalTrials.gov: NCT01482195 ↗Enrolled (actual)
6
Serious AEs
0.0%
Results posted
Jul 2021
Primary outcome: Primary: Systemic and Ocular Safety — 0; 0; 1; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- Subretinal administration of rAAV2-VMD2-hMERTKRecombinant Adeno-Associated Virus (Biological)
- Age
- Pediatric, Adult, Older Adult · 14+ yrs
- Sex
- All
- Sponsor
- King Khaled Eye Specialist Hospital
- Primary completion
- Aug 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Systemic and Ocular Safety |
0; 0; 1; 0; 0; 0 | — |
| SECONDARY Visual Acuity Measurement |
3; 1; 0; 2; 3; 3 | — |
| SECONDARY Full-field Stimulus Threshold Testing (FST). |
-23.34; -24.14; -19.58; -20.13; -19.22; -23.40 | — |
| SECONDARY Central Foveal Thickness (CFT) on Optical Coherence Tomography (OCT). |
65.80; 74.80; 69.20; 75.00 | — |
| SECONDARY Central Macular Thickness (CMT) on Optical Coherence Tomography (OCT). |
128.00; 132.67; 132.33; 123.76 | — |
Summary
This study was to assess the safety of gene transfer via subretinal administration of rAAV2-VMD2-hMERTK in subjects with MERTK-associated retinitis pigmentosa (RP).
Eligibility Criteria
Inclusion Criteria
- MERTK-associated retinal disease;
- VA: 20/100 or less in worse eye
- Ability to perform tests of visual and retinal function;
- Good general health based on a complete physical examination and hematology and chemistry studies performed at a pre-treatment evaluation;
- Ability to comply with research procedures;
Exclusion Criteria
- Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints or surgical complications (for example, glaucoma, corneal or lenticular opacities);
- Complicating systemic diseases (such as medical conditions causing immunosuppression) that would preclude the gene transfer, ocular surgery or known sensitivity or allergy to medications planned for use in the peri-operative period;
- Use of anti-platelet agents that may alter coagulation within 7 days prior to study agent administration;
- Use of immunosuppressive medications;
- 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 and that, in the opinion of the investigator, makes the subject unsuitable for the study.
- Current, or recent (within the past 30 days, or 10 half lives of the drug) participation, in any other research protocol involving investigational agents or therapies.
- Recent (within past 6 months) receipt of an investigational biologic therapeutic agent.Subjects will not be excluded based on their gender, race or ethnicity.
Data sourced from ClinicalTrials.gov (NCT01482195). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.