GVB-2001 Gene Therapy Via Intracameral Injection for the Treatment of Primary Open Angle Glaucoma

NCT06921317 | Recruiting Phase 1

• 1 other identifier: GVB-2001-GLU-IIT
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

This is an investigator initiated, single center, open label, non-randomized, exploratory clinical study. The purpose is to assess the safety and preliminary efficacy of gene therapy GVB-2001 (ScAAV2-dnRhoA) delivered via intracameral injection to the anterior segment of the eye for the treatment of subjects with primary open-angle glaucoma. At least 6 primary open angle glaucoma (POAG) patients with high intraocular pressure (IOP) will be divided into two experimental groups. The first subject was given low-dose treatment, and the follow-up subjects were determined by the Safety Review Committee (SRC). Each experimental group was given low-dose and high-dose drug treatment respectively, and low-dose or high-dose treatment was given. The optimal dose will be selected based on the trial results for future development of GVB-2001. Safety Review Committee, SRC will be set up for safety assessment of the study. The study subjects included will be 18 to 75 years of age (inclusion) and are diagnosed with primary open-angle glaucoma (POAG) for 1 year or more. Only participants who provide informed consent prior to all screening procedures will be eligible for enrollment into the trial.

Conditions

Primary Open-Angle Glaucoma (POAG)

Keywords

glaucoma; gene therapy; rho gene; trabecular meshwork; dnRhoA; IOP reduction; AAV; scAAV

Outcome Measures

Primary Outcomes (3)

• The number and proportion of subjects with IOP ≤21mmHg

  Day 28, Day 60, Day 90, 6-Month, and 12-Month after GVB-2001 administration.

• The number and proportion of subjects whose IOP decreased by ≥20% from baseline

  Day 28, Day 60, Day 90, 6-Month, and 12-Month after GVB-2001 administration.

• Ocular adverse events (AEs) : the characteristics of AES, including endophthalmitis, eyelid edema, and corneal injection site reaction

  Day 1, Day 3, Day 7, Day 14, Day 28, Day 60, Day 90, 6-Month and 12--Month after GVB-2001 administration.

Secondary Outcomes (12)

• Type, number, and incidence of systemic adverse events and serious adverse events

  Adverse events (AEs): The type, number and incidence of AEs and serious adverse events (SAEs) will be analysized within 12 months after GVB-2001 administration.

• The number of ocular malignancies related to GVB-2001 injection occurred after treatment

  After GVB-2001 Administration up to 5 years.

• RhoA protein concentration in blood

  Day 7, Day 28, and 12-Month after GVB-2001 administration

• RhoA protein concentration in urine

  Day 7, Day 28, and 12-Month after GVB-2001 administration

• RhoA protein concentration in tears

  Day 7, Day 28, and 12-Month after GVB-2001 administration

Study Arms (2)

Group 1: POAG patient without vision

EXPERIMENTAL

Low dose or high dose GVB-2001 25ul will be injected once into the anterior chamber by the investigator at visit 2.

Drug: GVB-2001-high dose; Drug: GVB-2001-low dose

Group 2: POAG patient with vision

EXPERIMENTAL

Low dose or high dose GVB-2001 will be injected once into the anterior chamber by the investigator at visit 2.

Drug: GVB-2001-high dose; Drug: GVB-2001-low dose

Interventions

GVB-2001-high dose DRUG

GVB-2001 is a self-complementary adeno-associated viral vector (scAAV) injection to deliver human dnRhoA gene to trabecular meshwork cells in patients with primary open angle glaucoma. A single dose of high dose GVB-2001 will be injected intracamerally to the target interventional eye.

Group 1: POAG patient without vision; Group 2: POAG patient with vision

GVB-2001-low dose DRUG

GVB-2001 is a self-complementary adeno-associated viral vector (scAAV) injection to deliver human dnRhoA gene to trabecular meshwork cells in patients with primary open angle glaucoma. A single dose of low dose GVB-2001 will be injected intracamerally to the target interventional eye.

Group 1: POAG patient without vision; Group 2: POAG patient with vision

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• The subjects volunteered to participate in the clinical study and signed written informed consent;
• Aged 18 to 75 years old (inclusive), men and women;
• Primary open angle glaucoma (POAG) with a history of diagnosis ≥1 year;
• Participants in good general health and have no clinically significant systemic disease, as determined by medical history, physical examination, and screening laboratory evaluation.
• To comply with the requirements, willing to accept all the diagnosis and treatment plan, laboratory tests and other specified testing, etc.;
• Consent is obtained for an extended safety visit after 1 year.
• Special eligibility criteria for trial group 1:
• no vision in the target eye;
• The intraocular pressure (IOP) of the target eye was ≤50mmHg and \> 21 mmHg after combined treatment with 2 or more IOP-lowering drugs.
• The intraocular pressure (IOP) of the target intervention eye was no more than 40mmHg, and the IOP was more than 21 mmHg after receiving combination therapy of 2 or more IOP-lowering drugs;
• The Shaffer gonioscopy scores of the target intervention eyes were all greater than 3.
• The best corrected distance visual acuity of the target intervention eyes was at least 0.3 (logMAR4.5) and above.

You may not qualify if:

• Secondary glaucoma;
• Any active or recurrent intraocular infection or inflammation, including but not limited to uveitis;
• Severe dry eye or clinically significant active keratopathy in the target eye;
• No intraocular pressure measurement was performed under any circumstances;
• Allergies to drugs or their excipient to be used in clinical studies;
• Ocular trauma in either eye within 6 months before screening, or eye surgery or nonrefractive laser therapy within 3 months before screening;
• A clinically significant history of herpes simplex or herpes zoster keratitis;
• A positive test for hepatitis B virus (HBV) HBsAg or HBV-DNA, hepatitis C virus (HCV) HCAb, or Epstein-Barr virus, or cytomegalovirus (CMV);
• Syphilis antibody and HIV antibody were positive.
• Severe active systemic bacterial, viral, fungal, malaria, or parasitic infections;
• Any past or present malignant tumor, myeloproliferative disorder or immunodeficiency disease;
• May interfere with the clinical significance of this study of systemic disease (including, active hepatitis, liver cirrhosis, liver fibrosis uncontrolled hypertension, myocardial infarction and myocarditis, arrhythmia, stroke, acute or chronic renal insufficiency, uncontrolled endocrine system diseases such as diabetes, thyroid function hyperfunction, Severe pulmonary hypertension, chronic obstructive pulmonary disease (copd), interstitial pneumonia, etc.);
• Any serious mental illness;
• Have participated in other clinical trials and received a medication or medical device intervention within 1 month before screening;
• And women of childbearing age who are pregnant, breastfeeding, planning to become pregnant, or who are not using a medically acceptable form of birth control. Excludes women of childbearing age who have been sterilized for 1 year after menopause or 3 months after surgery;

Sponsors & Collaborators

• IVIEW Therapeutics Inc.: lead
• The First Affiliated Hospital of Soochow University: collaborator

Study Sites (1)

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, China

RECRUITING

MeSH Terms

Conditions

Glaucoma, Open-Angle; Glaucoma; Night Blindness, Congenital Stationary, Autosomal Dominant 1

Condition Hierarchy (Ancestors)

Ocular Hypertension; Eye Diseases

Study Officials

• Peirong Lu, MD

  The First Affiliated Hospital of Soochow University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Zuliang Yao, MD

CONTACT

+1 609 773 8580; zuliang.yao@iviewinc.com

Jun Gao, MD

CONTACT

jun.gao@iviewinc.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 25, 2025
• First Posted: April 10, 2025
• Study Start: November 19, 2025
• Primary Completion (Estimated): December 30, 2027
• Study Completion (Estimated): February 28, 2028
• Last Updated: May 4, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)