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Retinitis PigmentosaPhase 1NCT06291935

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

A Phase 1 study testing VG901 in people with Retinitis Pigmentosa, run by VeonGen Therapeutics GmbH at 1 site in Germany.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Retinitis Pigmentosa
What is being tested?
VG901
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
1 siteGermany
Study dates
1 Sep 2023to Apr 2026
Phase
Phase 1
Are there results?
Not yet: the study is under way

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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 25Show fewer
  • 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
  • Systemic illness (e.g., hepatitis or human immunodeficiency virus [HIV] infection) or medically relevant abnormal laboratory values (3 x upper limit of normal [ULN]) in blood analysis including renal and hepatic function
  • Current, or recent, participation in other study/ or administration of investigational biologic agent within 3 months of Screening; Use of any investigational agent, or systemic corticosteroids, or other immunosuppressive drug(s) within 3 months before Screening
  • History of allergy or sensitivity to any compound used in the study
  • Contraindications to systemic immunosuppression
  • Subjects with increased risk of bleeding (i.e., use of anticoagulants or anti-platelet agents within 7 days before VG901 administration and subjects with international normalized ratio > 2 or Quick < 50% or partial thromboplastin time > 50 seconds, thrombocytopenia, as well as any other known coagulopathy)
  • Subject/partner of childbearing potential unwilling to use adequate contraception for the period between Screening and 30 days after treatment, defined as the period from Screening until 30 days after treatment (defined as administration of therapeutic to the eye)
  • For females of childbearing potential, a positive pregnancy test at Screening or Baseline
  • Females who are breastfeeding
  • Previous receipt of any AAV gene therapy product
  • Any condition which leads the investigator to believe that subject cannot comply with the protocol requirements or that may place the subject at an unacceptable risk from participating

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Andrea Rindtorff contact

+49 7071 29 87747

andrea.rindtorff@med.uni-tuebingen.de

Germany1 site, 1 recruiting

  • Center for Ophthalmology, University of Tuebingen

    Tübingen, 72076

    Andrea Rindtorff, +49 7071 29 87747, andrea.rindtorff@med.uni-tuebingen.de

    Recruiting

Other trials in Germany

About this study

Official title: A Prospective, Open-label, Phase 1b, Single-arm, Safety Study of an Intravitreal Application of a Recombinant Adeno-associated Virus Vector Expressing CNGA1 (AAV2.NN-CNGA1) in Patients With Retinitis Pigmentosa Due to CNGA1 Mutations

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.

Being tested
VG901
Keywords
CNGA1 mutation, AAV2, Gene Therapy, Eye disease
Medical subject headings
Retinitis Pigmentosa, Eye Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • VG901Drug

    Also called AAV2.NN-CNGA1

    Administered as specified in the treatment arm. Study Cohort 1 - Low dose; Study Cohort 2 - High dose

    Other Names:

    Gene Therapy (AAV2.NN-CNGA1)

Study groups in NCT06291935
Study groupRoleWhat they receive
VG901ExperimentalParticipants will receive a single dose of intravitreal injection of VG901 in most affected eye at Day 0.
Receives: Drug: VG901

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Baseline to Month 12

Secondary outcomes (12)

  1. Physical Examination

    A complete physical examination will be done at Screening and at Baseline and then at Month 12. On the other timepoints during the 1-year active follow-up phase, symptom-directed physical examination will be conducted. Abnormal examination results will be recorded.

    Measured: Screening to Month 12

  2. Pulse rate

    Will be recorded in beats per minute.

    Measured: Screening to Month 12

  3. Blood pressure

    Systolic and diastolic blood pressure will be recorded in mmHg.

    Measured: Screening to Month 12

  4. Body temperature

    Will be recorded in °C.

    Measured: Screening to Month 12

  5. Respiratory rate

    Will be recorded in breaths per minute.

    Measured: Screening to Month 12

  6. Slit lamp examination

    Abnormal examination results of conjunctiva, cornea, sclera, lens, anterior segment, and anterior chamber cells as well as quantification of vitreous inflammation and grading of anterior chamber flare will be recorded.

    Measured: Screening to Month 12

  7. Fundus biomicroscopy

    Cup-to-Disc (C/D) ratio and abnormal examination results will be recorded.

    Measured: Screening to Month 12

  8. Optical coherence tomography (OCT)

    Abnormal examination results will be recorded.

    Measured: Screening to Month 12

  9. Fundus autofluorescence

    Abnormal examination results will be recorded.

    Measured: Screening to Month 12

  10. Tonometry

    Intraocular Pressure (IOP) will be recorded in mmHg.

    Measured: Screening to Month 12

  11. Adeno-associated virus (AAV) spread

    As detected by quantitative polymerase chain reaction (qPCR) in peripheral blood, urine, and tear. Recorded in vector copies per µL of fluid DNA.

    Measured: From Baseline until two consecutive samples test negative

  12. Immunopathology

    Using specific enzyme-linked immunosorbent assays (ELISA) for humoral antibodies against rAAV2 capsid protein.

    Measured: Baseline to Month 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Mar 2024
  2. Study start1 Sep 2023
  3. Primary completionApr 2026 (estimated)
  4. Study completionApr 2026 (estimated)

Resources

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 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Masking
None (open label)
Enrolment
6 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06291935
Sponsor's protocol ID
VG901-2021 A
Other IDs
EU CT: 2023-504383-42-00
Status last verified
Mar 2025
First submitted
1 Feb 2024
Last updated
28 Mar 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing VG901 in people with Retinitis Pigmentosa, run by VeonGen Therapeutics GmbH at 1 site in Germany.

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 6 inclusion and 25 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: looking for participants now, as of the sponsor's last update on 28 Mar 2025. 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 Germany. 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.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Andrea Rindtorff (+49 7071 29 87747, andrea.rindtorff@med.uni-tuebingen.de), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Baseline to Month 12). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06291935.

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 28 Mar 2025.

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