Safety and Efficacy Study of Novel Gene Therapy ZM-01 for X-linked Retinoschisis Patients

Plain-language summary

This study examined the safety and effectiveness of ZM-01, a novel gene therapy, in patients with X-linked retinoschisis, an inherited eye disease. Boys aged 3-18 with an RS1 gene mutation took part. It was conducted in Early Phase 1 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Early Phase 1

Status

Completed

Sponsor

Zhongmou Therapeutics

Intervention

ZM-01-L, ZM-01-H

Start date

9/25/2022

Estimated completion

1/31/2026

Countries

China

Related genes

Locations

• Xiaogan, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

Subjects who meet all of the following criteria will be enrolled into the study

1. Diagnosis of X-linked retinoschisis consistent with the presence of RS1 gene mutation
2. Male, aged between 3 and 18 years old, in overall good health except for XLRS condition
3. Capable of undergoing visual and retinal function assessment.
4. The visual acuity of the study eye not better than: 0.4 (68 ETDRS letters equivalent)
5. No carbonic anhydrase inhibitors have been used at present and for 3 months before treatment
6. Laboratory tests meet the following criteria:

   1. Hemoglobin ≥ 11.0 g/dL
   2. White blood cell counts ranged from 3,300 to 12,000 cells /mm³;
   3. Platelet count 125,000-550,000 /mm³;
   4. Alanine aminotransferase (ALT) is not higher than 1.5 times the upper limit of the normal range of laboratory tests;
   5. Serum creatinine was no higher than 1.1 times the upper limit of the normal range for laboratory tests;
   6. Prothrombin time (PT) ≤14.5 seconds and partial thromboplastin time (PTT) ≤ 36.0 seconds.
7. Willing to discontinue aspirin, aspirin-containing products, and any other medications that may alter clotting function at least 7 days before dosing.
8. Be able to understand and sign informed consent.

Exclusion Criteria:

Subjects who meet any of the following exclusion criteria before enrollment were excluded from the study

1. Previously received any AAV gene therapy
2. The following mutations in RS1 gene: R141H, C59S or C223S
3. 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)
4. Ocular diseases in which there is opacity of the lens, cornea, or other media, hindering adequate observation and examination of the retina
5. Use anticoagulant or antiplatelet drugs within 7 days before dosing
6. Use any experimental drug within 3 months prior to registration
7. 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