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Recruiting NCT06465537

NCT06465537 CRISPR/Cas9 Instantaneous Gene Editing Therapy to Intraocular Hypertensive POAG With MYOC Mutation

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Clinical Trial Summary
NCT ID NCT06465537
Status Recruiting
Phase
Sponsor Shanghai BDgene Co., Ltd.
Condition Primary Open Angle Glaucoma
Study Type INTERVENTIONAL
Enrollment 9 participants
Start Date 2024-06-10
Primary Completion 2025-12

Trial Parameters

Condition Primary Open Angle Glaucoma
Sponsor Shanghai BDgene Co., Ltd.
Study Type INTERVENTIONAL
Phase N/A
Enrollment 9
Sex ALL
Min Age 18 Years
Max Age 65 Years
Start Date 2024-06-10
Completion 2025-12
Interventions
BD113vVLP

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Brief Summary

This study is intented to evaluate the safety, tolerability and preliminary efficacy of CRISPR/Cas9 Instantaneous Gene Editing Therapy (BD113 virus-like particle, also BD113vLVP) in patients with primary open-angle glaucoma (POAG) with elevated intraocular pressure and MYOC gene mutation. The main objectives to evaluate the safety and tolerability BD113vLVP) in POAG patients with intraocular hypertension and MYOC mutation, and secondary objectives is to explore the preliminary efficacy and the metabolism characteristics of BD113vLVP in participants.

Eligibility Criteria

Inclusion Criteria: 1. Signed ICF; 2. Aged 18 to 65 years old; 3. Primary open Angle glaucoma (POAG) with elevated intraocular pressure (IOP) was diagnosed with ≥1 year medical history record ; 4. Good function level of organs; 5. Good compliance and willing to comply with the visit schedule, laboratory tests and other specified test etc. per protocol; 6. Agreeing to accept a long-term safety follow-up after 1 year of study. Special Inclusion Criteria for Group 1: * Target intervenning eye is no visual acuity; * The intraocular pressure (IOP) was ≤35 mmHg and \> 21 mmHg after receiving a combination therapy of 2 or more drugs lowering IOP. Special Inclusion Criteria for Group 2: * MYOC gene mutation was detected in peripheral blood nucleated cells ; * The intraocular pressure (IOP) was ≤30 mmHg and \> 21 mmHg after receiving a combination therapy of 2 or more drugs lowering IOP; * Both eyes have a Shaffer Angle mirror rating greater than 3. Exclusion Criteria: 1. Secondary glaucoma; 2.

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