A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)
- Conditions
- X-Linked Retinitis Pigmentosa
- Interventions
- Other: No intervention (Follow-Up assessment)
- Registration Number
- NCT06646289
- Lead Sponsor
- Janssen Research & Development, LLC
- Brief Summary
The purpose of this study is to assess the safety and tolerability of subretinal delivery of Adeno-associated Virus Vector (AAV5 hRKp.RPGR) gene therapy in adults and children with X-linked retinitis pigmentosa.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Male
- Target Recruitment
- 39
- Have been treated with AAV5-hRKp.RPGR in study MGT009 and have completed or is currently enrolled in Study MGT010
- Must sign an informed consent form indicating that they understand the purpose and procedures of the study and is willing to participate in the study
- Willing to adhere to the protocol and long-term follow-up
- There are no specific exclusion criteria to enroll in this study
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Cohort 1 AAV5-hRKp.RPGR Participants will receive a dose of AAV5 hRKp.RPGR under the retina (low-dose or intermediate-dose) on Day 1 depending on the dosage administered in study MGT009 (NCT03252847) in the past. After receiving the treatment, participants will be assessed for safety. Cohort 2 AAV5-hRKp.RPGR Participants will receive the treatment dose of AAV5 hRKp.RPGR under the retina (low-dose or intermediate-dose) on Day 1 in the second eye once the safety will be determined in Cohort 1. Cohort 3 No intervention (Follow-Up assessment) The participants who are not willing to undergo surgery or are not eligible for surgery will be assessed in this cohort. They will be assessed yearly until 5 years after their initial eye surgery in previous study MGT009.
- Primary Outcome Measures
Name Time Method Number of Participants With Adverse Events (AE) From baseline up to 5.5 years An AE is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non investigational) product. An AE does not necessarily have a causal relationship with the intervention. An AE can be any unfavorable and unintended sign (including an abnormal finding), symptom, or disease temporally associated with the use of a medicinal (investigational or non investigational) product, whether or not related to that medicinal (investigational or non investigational) product.
- Secondary Outcome Measures
Name Time Method Change from Baseline in Mean Retinal Sensitivity Within the Full Visual Field (MRS90) Baseline, Months 12, 24, 36, 48 and 60 Change from baseline in MRS90 in static perimetry of the second treated eye will be assessed.
Change from Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees (MRS10) Baseline, Months 12, 24, 36, 48 and 60 Change from baseline in MRS10 in static perimetry of the second treated eye will be measured.
Change in Retinal Function as Assessed by Pointwise Response in Static Perimetry From Month 12 up to Month 60 Pointwise response in static perimetry within the full visual field in the second treated eye will be assessed.
Change in Retinal Function as Assessed by Pointwise Response in Static Perimetry Within Central 30 Degrees Visual Field From Month 12 up to Month 60 Pointwise response in static perimetry within the central 30 degrees visual field in the second treated eye will be assessed.
Change from Baseline in the Low Luminance Questionnaire (LLQ) Domain Scores Baseline, Month 12 LLQ is a 32-item disease-specific questionnaire to assess self reported visual problems under low luminance and at night. The range of responses is different for different questions, with the overall pattern being that "1" means "No difficulty at all" and that increasingly higher numbers mean increasingly more difficult.
Change from Baseline in Low Luminance Visual Acuity (LLVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score Baseline, Months 12, 24, 36, 48 and 60 Change in LLVA by ETDRS chart letter scores from baseline in monocular assessment of second treated eye will be assessed. Visual function assessments included LLVA assessment in each eye by ETDRS letters. The letter score ranges from 0 (worse score) to 100 (best score), and a gain in LLVA from baseline indicates an improvement in visual acuity.
Change from Baseline in Best Corrected Visual Acuity (BCVA) by ETDRS Chart Letter Scores Baseline, Months 12, 24, 36, 48 and 60 Change from baseline in BCVA by ETDRS chart letter scores in monocular assessment of the second treated eye will be assessed. BCVA was measured using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. An increase in the number of letters read correctly means that vision has improved and vice-versa.
Trial Locations
- Locations (2)
Massachusetts Eye and Ear Infirmary
🇺🇸Boston, Massachusetts, United States
University of Michigan Kellogg Eye Center
🇺🇸Ann Arbor, Michigan, United States