Primary Open Angle GlaucomaNCT06465537
CRISPR/Cas9 Instantaneous Gene Editing Therapy to Intraocular Hypertensive POAG With MYOC Mutation
A study testing BD113vVLP in people with Primary Open Angle Glaucoma, run by Shanghai BDgene Co., Ltd. at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Primary Open Angle Glaucoma
- What is being tested?
- BD113vVLP
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 10 Jun 2024to Jul 2027
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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;
- Good compliance and willing to comply with the visit schedule, laboratory tests and other specified test etc. per protocol;
- Agreeing to accept a long-term safety follow-up after 1 year of study.
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- 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.
Who cannot join
- 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;
- Any positive of human immunodeficiency virus type 1/2 (HIV-1/HIV-2) antibody, treponema pallidum (TP) specific antibody, human T-lymphotropic virus type 1 or 2 (HTLV-1/HTLV-2) antibody, or vesicular stomatitis virus G (VSV-G) antibody;
- Any of hepatitis B virus (HBV) HbsAg or HBV-DNA, hepatitis C virus (HCV) HCAb, or epstein-barr virus (EBV), or cytomegalovirus (CMV) nucleic acid test is positive;
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- Severe active bacterial, viral, fungal, malaria or parasitic systemic infection;
- Any past or present malignancy, myeloproliferative or immunodeficient disease;
- History of major organ diseases or abnormalities in laboratory tests, including:
- Liver cirrhosis, liver fibrosis or active hepatitis, and/or abnormal liver function tests (serum total bilirubin (TBIL) ≥1.5 x upper limit of normal (ULN); Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≥2.5×ULN; Alkaline phosphatase ≥2.5 × ULN);
- Cardiovascular and cerebrovascular diseases, including uncontrolled hypertension, myocardial infarction, myocarditis, arrhythmia, stroke, etc.;
- Kidney disease, or creatinine ≥ 1.5ULN and creatinine clearance < 30% normal level (using the Cockcroft-Gault equation);
- Endocrine disorders, such as insulin-dependent diabetes mellitus, hyperthyroidism or hypothyroidism;
- Severe pulmonary hypertension, chronic obstructive pulmonary disease, interstitial pneumonia;
- Any severe psychiatric disorders;
- Participating in another clinical study of a drug or device, or administrated the investigational drug within 42 days prior to the screening visit;
- Pregnant or lactating women;
- Refusing to accept any contraception measures;
- Allergic to clinical investigational drugs or their excipients;
- Other conditions assessed by the investigator as unsuitable for participation in this study.
- Special Exclusion Criteria for Group 2:
- Retinal diseases: complicated with unexplained quadrant blindness, neovascularization age-related macular degeneration, retinal branch vein obstruction, central retinal vein obstruction, cystoid macular edema, macular hiatal hole and central serous retinopathy;
- A history of anterior chamber angle stenosis, congenital glaucoma, or angle closure, clinically significant anterior peripheral adhesion, or extensive cicatricial adhesion caused by surgery/laser therapy in the anterior chamber angle;
- The central corneal thickness is less than 480 μm or more than 620 μm.
Trial locations and contacts
1 site in 1 country.
China1 site
Beijing Tongren Hospital, Capital Medical University
Beijing, Beijing Municipality, 100730
About this study
Official title: A Clinical Study on CRISPR/Cas9 Instantaneous Gene Editing Therapy to Primary Open-angle Glaucoma With Elevated Intraocular Pressure and MYOC Gene Mutation
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.
Read the detailed description
This is an open, single-dose, two-arm, non-randomised clinical study. A total of 6 to 9 POAG patients with high intraocular pressure were enrolled and divided into two test groups. Test Group 1 recruits 3 POAG patients, who have elevated IOP and positive or negative MYOC mutation and target interventing eye is no vision. Test Group 2 will recruit 3 to 6 POAG patients with MYOC mutations and visual acuity. In order to better verify the lowering IOP effectiveness of BD113vVLP, another 2 or 3 participants will be recruied in Group 2 on-demand. Each participant will receive single dosing BD113vVLP (4µg p24) by intracameral injection in the interventing eye, then conduct the evaluations of the safety and efficacy according to visit schedule in 1 year follow-up。
- Conditions
- Primary Open Angle Glaucoma
- Being tested
- BD113vVLP
- Keywords
- primary open angle glaucoma, POAG, elevated intraocular pressure, MYOC gene mutation
- Medical subject headings
- Glaucoma, Open-Angle, Ocular Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BD113vVLPGenetic
Also called BD113 virus-like particle, CRISPR/Cas9 mRNA instantaneous gene editing technology
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.
| Study group | Role | What they receive |
|---|---|---|
| Group 1: POAG without vision for interventional eye | Experimental | Interventional eye of participants with POAG has no vision, with mutated or unmutated MYOC gene. Single dose of BD113vVLP will be administrated intracamerally for target interventional eye. Receives: Genetic: BD113vVLP |
| Group 2: POAG with vision for interventional eye | Experimental | Interventional eye of participants with POAG has vision acuity, and MYOC gene mutation test is positive. Single dose of BD113vVLP will be administrated intracamerally for target interventional eye. Receives: Genetic: BD113vVLP |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 12 months
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
Measured: 12 months
Secondary outcomes (9)
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.
Measured: 12 months
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
Measured: 12 months
Any ocular maligancies related to BD113vVLP
after BD113vVLP administration
Measured: 12 months
Changes in BCVA from baseline
at Month 3, Month 6 and Month 12 after BD113 vVLP administration, not applicable to group 1.
Measured: 12 months
Changes in visual fields from baseline
at Month 3, Month 6 and Month 12 after BD113 vVLP administration, not applicable to group 1.
Measured: 12 months
Changes in RNFL from baseline
at Month 3, Month 6 and Month 12 after BD113 vVLP administration, not applicable to group 1.
Measured: 12 months
P24 and Cas9 proteins concentration in aqueous humor
at hour 0 and Month 1 after BD113vVLP administration
Measured: 1 months
P24 and Cas9 proteins concentration in blood
at hour 0 and Day 7 after BD113vVLP administration
Measured: 7 days
Blood antibodies of anti-p24 protein and anti-Cas9 proteins
at Month 6 and Month 12 after BD113vVLP administration
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jun 2024
- Study start10 Jun 2024
- Primary completionApr 2027 (estimated)
- Study completionJul 2027 (estimated)
Why it stopped: Per the requirement of China regulation.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Open Angle GlaucomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai BDgene Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Beijing Tongren Hospital
- Responsible party
- Sponsor
- Investigators
- Yufei Teng, M.D., Study director, Beijing Tongren Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06465537
- Sponsor's protocol ID
- BD-MMG-113001
- Status last verified
- Aug 2026
- First submitted
- 13 Jun 2024
- Last updated
- 20 Aug 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Study on CRISPR/Cas9 Instantaneous Gene Editing Therapy to Primary Open-angle Glaucoma With Elevated Intraocular Pressure and MYOC Gene Mutation
- ClinicalTrials.gov ID
- NCT06465537
- Acronym / sponsor ID
- BD-MMG-113001
- Conditions
- Primary Open Angle Glaucoma
- Keywords
- primary open angle glaucoma, POAG, elevated intraocular pressure, MYOC gene mutation
- Study type
- Interventional
- Recruitment status
- SuspendedReason stopped: Per the requirement of China regulation.
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Shanghai BDgene Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Beijing Tongren Hospital
- Study start
- 10 Jun 2024 (actual)
- Primary completion
- Apr 2027 (estimated)
- Study completion
- Jul 2027 (estimated)
- First posted
- 20 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is an open, single-dose, two-arm, non-randomised clinical study. A total of 6 to 9 POAG patients with high intraocular pressure were enrolled and divided into two test groups. Test Group 1 recruits 3 POAG patients, who have elevated IOP and positive or negative MYOC mutation and target interventing eye is no vision. Test Group 2 will recruit 3 to 6 POAG patients with MYOC mutations and visual acuity. In order to better verify the lowering IOP effectiveness of BD113vVLP, another 2 or 3 participants will be recruied in Group 2 on-demand. Each participant will receive single dosing BD113vVLP (4µg p24) by intracameral injection in the interventing eye, then conduct the evaluations of the safety and efficacy according to visit schedule in 1 year follow-up。
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1: POAG without vision for interventional eye | Experimental | Genetic: BD113vVLPInterventional eye of participants with POAG has no vision, with mutated or unmutated MYOC gene. Single dose of BD113vVLP will be administrated intracamerally for target interventional eye. |
| Group 2: POAG with vision for interventional eye | Experimental | Genetic: BD113vVLPInterventional eye of participants with POAG has vision acuity, and MYOC gene mutation test is positive. Single dose of BD113vVLP will be administrated intracamerally for target interventional eye. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- 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;
- Good compliance and willing to comply with the visit schedule, laboratory tests and other specified test etc. per protocol;
Key exclusion criteria (24 listed)
- 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;
- Any positive of human immunodeficiency virus type 1/2 (HIV-1/HIV-2) antibody, treponema pallidum (TP) specific antibody, human T-lymphotropic virus type 1 or 2 (HTLV-1/HTLV-2) antibody, or vesicular stomatitis virus G (VSV-G) antibody;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BD113vVLPAlso: BD113 virus-like particle, CRISPR/Cas9 mRNA instantaneous gene editing technologyCRISPR/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. | Genetic | Group 1: POAG without vision for interventional eye, Group 2: POAG with vision for interventional eye |
Primary outcome
Ocular adverse events (AEs)
Time frame: 12 months
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.
Number and percentage of participants whose IOP decrease ≤21 mmHg
Time frame: 12 months
at Month 1, Month 2, Month 3, Month 6 and Month 12 after BD113vVLP administration
9 secondary outcomes
Systemic adverse events (AEs): The type, number and incidence of AEs and serious adverse events (SAEs)
Time frame: 12 months
Number and percentage of participants whose IOP decrease by ≥ 20% from baseline
Time frame: 12 months
Any ocular maligancies related to BD113vVLP
Time frame: 12 months
Changes in BCVA from baseline
Time frame: 12 months
Changes in visual fields from baseline
Time frame: 12 months
Changes in RNFL from baseline
Time frame: 12 months
P24 and Cas9 proteins concentration in aqueous humor
Time frame: 1 months
P24 and Cas9 proteins concentration in blood
Time frame: 7 days
Blood antibodies of anti-p24 protein and anti-Cas9 proteins
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Yufei Teng, M.D. study director
Beijing Tongren Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing BD113vVLP in people with Primary Open Angle Glaucoma, run by Shanghai BDgene Co., Ltd. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: paused, as of the sponsor's last update on 20 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Ocular adverse events (AEs) (time frame: 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Aug 2026.