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| Name | Class |
|---|---|
| Eye & ENT Hospital of Fudan University | OTHER |
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The primary objective of this clinical trial is to evaluate the safety and tolerability of a single intravitreal injection of the gene therapy drug UGX202 in patients with advanced RP. The secondary objective is, to assess the preliminary efficacy of a single intravitreal injection of the gene therapy drug UGX202 in treating patients with advanced RP.
This study is a non-randomized, open-label investigator-initiated trial (IIT). It plans to enroll approximately 6 subjects with non-syndromic retinitis pigmentosa (RP) who have extremely low vision (the study eye is the eye with lower vision, and the best corrected visual acuity [BCVA] > logMAR 1.9).
The study drug is divided into two dose groups: low dose and high dose. A modified "3+3" dose escalation approach is adopted. The low-dose group (4.2E+10 vg/eye) is planned to include 3 subjects. First, 1 subject (sentinel) will be enrolled and observed for 28 days. If no dose-limiting toxicity (DLT) occurs, 2 more subjects (non-sentinel) will be enrolled and observed for 28 days. The second and third subjects will be enrolled with a 7-day interval.
The high-dose group (1.2E+11 vg/eye) is planned to include 3 subjects. Subjects in the high-dose group will be enrolled and administered the drug in sequence after passing the screening. There will be at least a 1-week interval between each subject. The timing of enrolling the full 3 subjects or stopping enrollment will be determined by the investigator's assessment of safety.All subjects will receive intravitreal injection of the study drug UGX202 after enrollment and will be followed up for 52 weeks to evaluate the safety, tolerability, and preliminary efficacy of UGX202.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Low dose of UGX202 group | Experimental | UGX202, 4.2E+10 vg per eye, administered as a single intravitreal injection |
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| High dose of UGX202 group | Experimental | UGX202, 1.2E+11 vg/eye, administered as a single intravitreal injection |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| UGX202 injection | Genetic | Comparison of different dosages of UGX202 |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of adverse events and serious adverse events | From the time of administration of UGX202 injection until the 52nd week, based on the topical and systemic safety data, the incidence rates of AEs, TEAEs during treatment, TRAEs related to the study drug, TRAEs related to the study procedures, SAEs, TRSAEs related to the study drug, and TRSAEs related to the study procedures during the study period were summarized by the investigators, and the correlations between AEs and the study drug and study procedures were determined. | baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52 |
| The average change in IOP | The average change in IOP of the study eyes and non-study eyes from after treatment to the 52nd week compared to the baseline. The IOP was measured three times consecutively at each visit and the average value was taken. | baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| The changes in BCVA | The changes in BCVA of the study eyes and non-study eyes at each follow-up visit compared to the baseline were evaluated. BCVA was assessed using the Freiburg Vision Test (FrACT) system. If the subjects had no light perception at the baseline: the proportion of subjects whose BCVA improved to having light perception after treatment was evaluated, and the change in their vision compared to the baseline was assessed; |
| Measure | Description | Time Frame |
|---|---|---|
| The change in Latency of N2, latency of P2, N2-P2 amplitude difference will be evaluated in VEP | Latency of N2, latency of P2, N2-P2 amplitude difference will be evaluated in VEP both in the study eye and non-study eyes at each visits compared with baseline. | Baseline, week4, week 8, week 12, week 24, week 52/EoS |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Jihong Wu, MD, PHD | Contact | +86 21 6437 7134 | 1217586177@qq.com | |
| Xiuqian Yi, MD, PHD | Contact | +86 21 6437 7134 | 1217586177@qq.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Eye & ENT Hospital of Fudan University | Shanghai | Shanghai Municipality | 200031 | China |
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| ID | Term |
|---|---|
| D012174 | Retinitis Pigmentosa |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D058499 | Retinal Dystrophies |
| D012162 | Retinal Degeneration |
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| baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52 |
| The changes in the average stimulus threshold | The changes in the average stimulus threshold measured by the full-field stimulus threshold test (FST) of the study eyes and/or non-study eyes at each follow-up visit compared to the baseline; | baseline to Week 4, Week 12, Week 24, Week 52 |
| The changes in the visual function questionnaire (VFQ-25) scores | The changes in the visual function questionnaire (VFQ-25) scores of the subjects at each follow-up visit after the treatment compared to the baseline. | baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52 |
| The change in dark-adapted 0.01 ERG, dark- adapted 3.0 ERG, dark-adapted 30.0 ERG and light-adapted 3.0 ERG |
Dark-adapted 0.01 ERG, dark- adapted 3.0 ERG, dark-adapted 30.0 ERG and light-adapted 3.0 ERG will be evaluated in electroretinogram both in the study eye and non-study eyes at each visits compared with baseline. |
| Baseline, week4, week 8, week 12, week 24, week 52/EoS |
| Change of mean defect (MD) in the visual fields | Change of mean defect (MD) in the visual fields of the study eyes and non-study eyes | Baseline, week 24, week 52/EoS |
| Change of visual field index (VFI) in the visual fields | Change of visual field index (VFI) in the visual fields of the study eyes and non-study eyes | Baseline, week 24, week 52/EoS |
| The benefit outcomes of patients with retinitis pigmentosa (RP) of different genotypes in either best-corrected visual acuity (BCVA) or the multi-luminance mobility test (MLMT) | The benefit outcomes of patients with retinitis pigmentosa (RP) of different genotypes either in best-corrected visual acuity (BCVA) or the multi-luminance mobilitytest (MLMT), and will conduct correlation the above analysis between factors. | Baseline |
| Changes in the scores of MLMT | Changes in the scores of multi-luminance mobility test (MLMT) | Baseline，week4， week 8, week 12, week 24, week 52/EoS |
| Change of Color Vision Test score | Color vision test will be conducted to patients' assess judgment and discrimination abilities for different channels, with comparisons of the changes in scores at different study visits related to the baseline scores. | Baseline, week4, week 12, week 24, week 52/EoS |
| Titer of viral vector DNA detected in blood, tears, and urine | Detection of viral vector DNA in blood, tears, and urine | Baseline，Day3， Day7, week2, week 12, week 24, week 52/EoS |
| Number of participants with positive Anti-drug antibodies(ADA) and neutralizing antibodies(Nab) | From the time of administration of UGX202 injection until the 52nd cycle, the detection of anti-drug antibodies (ADA) and neutralizing antibodies (Nab) for the viral vector capsid protein was carried out. | Baseline, week2, week4, week 12, week 24, week 36, week 52/EoS |
| Number of participants with positive anti-target photosensitive protein | From the time of administration of UGX202 injection until the 52nd cycle, ADA (anti-target photosensitive protein) detection was conducted. | Baseline, week2, week4, week 12, week 24, week 36, week 52/EoS |
| Concentration of T-cell immune responses against the viral vector capsid protein and the target photosensitive protein. | From the time of UGX202 injection treatment until the 52nd cycle, ELISpot was used to detect T-cell immune responses against the viral vector capsid protein and the target photosensitive protein. | Baseline, week 12, week 24 |
| D012164 |
| Retinal Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |