Health TrialsbyHealthPanel

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

ConditionPrimary Open Angle Glaucoma
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age18-65
SponsorShanghai BDgene Co., Ltd.

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

Signed ICF;

Aged 18 to 65 years old;

Primary open Angle glaucoma (POAG) with elevated intraocular pressure (IOP) was diagnosed with ≥1 year medical history record ;

Good function level of organs;

Disqualifiers

Secondary glaucoma;

Any active or recurrent intraocular infection or inflammation, including but not limited to uveitis;

The target intervenning eye has severe xerophthalmia or clinically significant active corneal disease;

Any condition no accepting the measure of IOP;

Trial design

Design model

Sequential

Treatments tested in this trial

  • BD113vVLP

    Genetic

    CRISPR/Cas9 gene editing technology, called BD113vVLP (also BD113 virus-like particle) which is a developing product of gene therapy from modified third-generation integrated defective lentivirus, can deliver gRNA/Cas9 ribonucleoprotein complex (RNP). It works to knock out or knock down the mutated MYOC gene. The BD113vVLP is administrated by intracamerally injecton (sigle-dose: 4ug/p24) for each target interventional eye.

Treatment groups

9 Participants
are divided into 2 treatment groups
Group A: Group 1: POAG without vision for interventional eyeExperimental treatment 1 intervention
Group B: Group 2: POAG with vision for interventional eyeExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Ocular adverse events (AEs)

The charactaristics of ocular adverse events include endophthalmitis, hypopyon, hyphaema and corneal injection site reaction etc. will be evaluated at Week 1, Week 2, Week 3, Week 4, Month 6 and Month 12 after BD113vVLP administration.

Time frame
12 months
2

Number and percentage of participants whose IOP decrease ≤21 mmHg

at Month 1, Month 2, Month 3, Month 6 and Month 12 after BD113vVLP administration

Time frame
12 months

Secondary outcomes

1

Systemic adverse events (AEs): The type, number and incidence of AEs and serious adverse events (SAEs)

Systemic adverse events (AEs): The type, number and incidence of AEs and serious adverse events (SAEs) will be analysized within 12 months after BD113vVLP administration.

Time frame
12 months
2

Number and percentage of participants whose IOP decrease by ≥ 20% from baseline

at Month 1, Month 2, Month 3, Month 6 and Month 12 after BD113vVLP administration

Time frame
12 months
3

Any ocular maligancies related to BD113vVLP

after BD113vVLP administration

Time frame
12 months
4

Changes in BCVA from baseline

at Month 3, Month 6 and Month 12 after BD113 vVLP administration, not applicable to group 1.

Time frame
12 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Shanghai BDgene Co., Ltd.

Lead sponsor

Beijing Tongren Hospital

Collaborator

Source ClinicalTrials.gov
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