An Open-label, Multicenter, Phase 2 Follow-on Study for Second Eye Treatment of Patients Previously Treated With a Recombinant Adeno-associated Virus Vector (AAV5 hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)
概览
- 阶段
- 2 期
- 干预措施
- AAV5-hRKp.RPGR
- 疾病 / 适应症
- X-Linked Retinitis Pigmentosa
- 发起方
- Janssen Research & Development, LLC
- 入组人数
- 24
- 试验地点
- 4
- 主要终点
- Number of Participants With Adverse Events (AE)
- 状态
- 进行中（未招募）
- 最后更新
- 19天前
概览
简要总结
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.
研究者
入排标准
入选标准
- •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
排除标准
- •\- There are no specific exclusion criteria to enroll in this study
研究组 & 干预措施
Cohort 1
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.
干预措施: AAV5-hRKp.RPGR
Cohort 2
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.
干预措施: AAV5-hRKp.RPGR
Cohort 3
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.
干预措施: No intervention (Follow-Up assessment)
结局指标
主要结局
Number of Participants With Adverse Events (AE)
时间窗: 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.
Number of Participants With Adverse Events (AE)
时间窗: 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.
Change from Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Scores
时间窗: 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.
Change from Baseline in Low Luminance Visual Acuity (LLVA) by ETDRS Chart Letter Score
时间窗: 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.