Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene

NCT06291935 | Recruiting Phase 1 DMC

• 2 other identifiers: VG901-2021 AEU CT: 2023-504383-42-00
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this phase 1 clinical trial is to learn about the safety and efficacy of a gene therapy, VG901, in patients with a rare disorder of the eye called Retinitis Pigmentosa. The main questions the study aims to answer are:

• What is the best tolerated dose and are there any side effects, in particular any inflammatory reactions post drug administration?
• Are there any early signs of efficacy on visual function? Participants will be administered a single intravitreal dose of VG901 into the most affected eye through a syringe and followed up for a year to monitor safety and efficacy. There will be two cohorts of participants in this study. Study Cohort 1 will receive the low dose and Study Cohort 2 will receive the high dose as specified in the Protocol.

Conditions

Retinitis Pigmentosa

Keywords

CNGA1 mutation; AAV2; Gene Therapy; Eye disease

Outcome Measures

Primary Outcomes (1)

• Adverse Events (AEs) and Serious Adverse Events (SAEs)

  Number of Adverse Events (AEs) and Serious Adverse Events (SAEs). Ocular inflammation is defined as an adverse event of special interest (AESI). AESI follow the same reporting requirements as SAE.

  Baseline to Month 12

Secondary Outcomes (12)

• Physical Examination

  Screening to Month 12

• Pulse rate

  Screening to Month 12

• Blood pressure

  Screening to Month 12

• Body temperature

  Screening to Month 12

• Respiratory rate

  Screening to Month 12

Study Arms (1)

VG901

EXPERIMENTAL

Participants will receive a single dose of intravitreal injection of VG901 in most affected eye at Day 0.

Drug: VG901

Interventions

VG901 DRUG

Administered as specified in the treatment arm. Study Cohort 1 - Low dose; Study Cohort 2 - High dose Other Names: Gene Therapy (AAV2.NN-CNGA1)

Also known as: AAV2.NN-CNGA1

VG901

Eligibility Criteria

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

You may qualify if:

• To be eligible for study entry, subjects must satisfy all the following criteria:
• Able to understand and willing to consent to study participation by a written informed consent
• Male or female ≥ 18 years of age
• Clinical diagnosis of RP
• Confirmed pathogenic, biallelic variants in the CNGA1 gene
• Ellipsoid zone (EZ) length of the fovea of ≥ 3000 μm in the study eye

You may not qualify if:

• Subjects will be excluded from the study if one or more of the following statements are applicable to either eye:
• Additional interfering ocular conditions which would impact study results (e.g., ocular opacity and advanced cataract, uveitis, amblyopia)
• History or presence of glaucoma
• Ocular surgery, intravitreal or subretinal implantation of a medical device (within 6 months of screening)
• Mutations known to cause inherited retinal disease other than biallelic variants in the CNGA1 gene
• History of ocular infection with herpes simplex virus
• History of ocular malignancies
• History of disorders of the internal retina (e.g., retinal detachment)
• Patients with uncontrolled diabetes (HbA1c \> 7%)
• Any other retinopathy due to other diseases - including, but not limited to arterial hypertension, previous vascular retinal occlusion, trauma or acquired inflammatory diseases, contraindication to pharmacological mydriasis (e.g., history of angle block glaucoma), diabetes (diabetic retinopathy including macular oedema)
• Absence of visual function on the contralateral eye
• Any damage to the optic nerve
• Individuals performing any other therapy for RP within 3 months before the study, such as - but not limited to - transcorneal electrostimulation
• Systemic conditions (e.g., autoimmune disorders) which may affect study participation or outcome measures
• History of immunodeficiency or other medical conditions which may increase the risk of VG901 administration

Sponsors & Collaborators

• VeonGen Therapeutics GmbH: lead

Study Sites (1)

Center for Ophthalmology, University of Tuebingen

Tübingen, 72076, Germany

RECRUITING

MeSH Terms

Conditions

Retinitis Pigmentosa; Eye Diseases

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Katarina Stingl

  Center for Ophthalmology, University of Tuebingen

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Andrea Rindtorff

CONTACT

+49 7071 29 87747; andrea.rindtorff@med.uni-tuebingen.de

Study Design

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

Study Record Dates

• First Submitted: February 1, 2024
• First Posted: March 4, 2024
• Study Start: September 1, 2023
• Primary Completion: April 1, 2026
• Study Completion: April 1, 2026
• Last Updated: March 28, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)