Status:
RECRUITING
Safety and Efficacy Study of Novel Gene Therapy ZM-01 for X-linked Retinoschisis Patients
Lead Sponsor:
Zhongmou Therapeutics
Conditions:
X-linked Retinoschisis
Eligibility:
MALE
3-18 years
Phase:
EARLY_PHASE1
Brief Summary
This trial is meant to evaluate the safety and efficacy of ZM-01 of X-linked retinoschisis. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye.
Detailed Description
X-linked retinoschisis (XLRS) is a rare, inherited retinal disease caused by mutations in the RS1 gene. Individuals affected by XLRS often experience progressive visual impairment from a young age, po...
Eligibility Criteria
Inclusion
- Subjects who meet all of the following criteria will be enrolled into the study
- Diagnosis of X-linked retinoschisis consistent with the presence of RS1 gene mutation
- Male, aged between 3 and 18 years old, in overall good health except for XLRS condition
- Capable of undergoing visual and retinal function assessment.
- The visual acuity of the study eye not better than: 0.4 (68 ETDRS letters equivalent)
- No carbonic anhydrase inhibitors have been used at present and for 3 months before treatment
- Laboratory tests meet the following criteria:
- Hemoglobin ≥ 11.0 g/dL
- White blood cell counts ranged from 3,300 to 12,000 cells /mm³;
- Platelet count 125,000-550,000 /mm³;
- Alanine aminotransferase (ALT) is not higher than 1.5 times the upper limit of the normal range of laboratory tests;
- Serum creatinine was no higher than 1.1 times the upper limit of the normal range for laboratory tests;
- Prothrombin time (PT) ≤14.5 seconds and partial thromboplastin time (PTT) ≤ 36.0 seconds.
- Willing to discontinue aspirin, aspirin-containing products, and any other medications that may alter clotting function at least 7 days before dosing.
- Be able to understand and sign informed consent.
Exclusion
- Subjects who meet any of the following exclusion criteria before enrollment were excluded from the study
- Previously received any AAV gene therapy
- The following mutations in RS1 gene: R141H, C59S or C223S
- Pre-existing eye conditions that cause severe vision loss or increase the risk of intravitreal injections (e.g., advanced glaucoma, uveitis, or severe retinal detachment)
- Ocular diseases in which there is opacity of the lens, cornea, or other media, hindering adequate observation and examination of the retina
- Use anticoagulant or antiplatelet drugs within 7 days before dosing
- Use any experimental drug within 3 months prior to registration
- Presented any situation that causes the investigator to believe the subject might not adhere to the study protocol or that participation might pose an unacceptable risk to the subject
Key Trial Info
Start Date :
September 25 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
October 1 2027
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT06066008
Start Date
September 25 2022
End Date
October 1 2027
Last Update
February 21 2024
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
Wuhan University Renmin Hospital affiliated with Hanchuan Hospital
Xiaogan, Hubei, China