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Retinitis Pigmentosa (RP)Phase 1UGX202NCT07847723

Phase I/II Open-Label Trial of UGX202 for Advanced Retinitis Pigmentosa

A Phase 1 study testing UGX202 Injection in people with Retinitis Pigmentosa (RP), run by Suzhou UgeneX Therapeutics Co., Ltd..

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Official record
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Trial summary

This is a single-arm, open-label study employing a 3+3 dose escalation design to evaluate two UGX202 dose groups: the low-dose group (1.2×10¹¹ vg/eye) and the high-dose group (3.5×10¹¹ vg/eye). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Retinitis Pigmentosa (RP)
What is being tested?
UGX202 Injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
No sites listed yet
Study dates
28 Sep 2026to 15 Jun 2029
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

  • The participant voluntarily signs the informed consent form (ICF).
  • The participant is older than 18 years at the time of signing the ICF.
  • The participant has a confirmed diagnosis of non-syndromic retinitis pigmentosa (RP)
  • Best-corrected visual acuity (BCVA) in the study eye is <0.05, or the ETDRS letter score is ≤19 letters.
  • The participant's pupil can be sufficiently dilated to allow comprehensive ophthalmic examination and testing.
  • The participant agrees to use effective contraception from enrollment until at least 1 year after the last administration of study treatment. Effective methods include complete abstinence (if this is the participant's preferred and usual lifestyle), an intrauterine device, bilateral tubal ligation, a partner who has undergone vasectomy with confirmed azoospermia, or a dual-barrier method (a male condom used with a cervical cap, diaphragm, or spermicidal sponge).
  • The spherical equivalent refractive error of the study eye is between -6.00 D and +6.00 D, inclusive.

Who cannot join

  • The study eye has previously received any form of gene therapy.
  • The participant has participated in another interventional clinical trial within 90 days before screening.
  • The participant has a disease of the study eye or a systemic disease that, in the investigator's opinion, may affect visual function assessment, such as macular degeneration, retinal detachment, retinal arteriolar or venous occlusion, glaucoma, or a central nervous system disorder affecting visual function.
  • The study eye has an abnormality that may interfere with visual acuity or other ophthalmic assessments, such as opacity of the ocular media that affects fundus examination. At the investigator's discretion, participants with cataracts that do not significantly impair visual acuity may be enrolled.
  • The participant has an ocular disease or abnormality that may affect safety assessment, such as an active ocular infection, chronic uveitis, or ocular trauma within the preceding 3 months.
  • The participant has a contraindication to pupil dilation, such as a narrow anterior chamber angle.
  • The study eye has undergone vitrectomy or macular surgery.
  • The study eye has undergone major surgery or intraocular surgery within the 3 months before screening, as determined by the investigator.

About this study

Official title: An Open-label, Dose-escalation and Expanded Phase I/II Clinical Trial to Evaluate the Safety and Preliminary Efficacy of UGX202 Injection in Patients With Advanced Retinitis Pigmentosa

This is a single-arm, open-label study employing a 3+3 dose escalation design to evaluate two UGX202 dose groups: the low-dose group (1.2×10¹¹ vg/eye) and the high-dose group (3.5×10¹¹ vg/eye). According to the "Dose Escalation Rule" (see below), each dose group comprised 3-6 study participants; thus, a total of 6-12 study participants were enrolled in this phase. Participants were enrolled in ascending order from the lowest to the highest dose; each study participant could participate in only one dose group (defined as having received the investigational drug - participants who had not received the drug were not considered to have participated in any dose group), and could not participate in multiple dose groups sequentially.

Being tested
UGX202 Injection
Keywords
RP, Retinitis Pigmentosa, UGX202, UGX202-S101
Medical subject headings
Retinitis Pigmentosa

What participants receive

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

  • UGX202 InjectionDrug

    This is a single-arm, open-label study employing a 3+3 dose escalation design to evaluate two UGX202 dose groups: the low-dose group (1.2×10¹¹ vg/eye) and the high-dose group (3.5×10¹¹ vg/eye). According to the "Dose Escalation Rule" (see below for details), each dose group comprised 3-6 study participants; a total of 6-12 study participants were enrolled in this phase.

Study groups in NCT07847723
Study groupRoleWhat they receive
a 3+3 dose escalation design to evaluate two UGX202 dose groupsExperimentalNot described
Receives: Drug: UGX202 Injection

What is being measured

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

  1. Safety and tolerability

    From baseline until week 52 after administration of UGX202 Injection: incidence rates of ocular and non-ocular adverse events (AEs), treatment-emergent adverse events (TEAEs), treatment-related adverse events (TRAEs), adverse events related to study procedures, serious adverse events (SAEs), treatment-related serious adverse events (TRSAEs), and adverse events related to study procedures, etc.

    Measured: From baseline until week 52

Secondary outcomes (6)

  1. Change From Baseline in Best-Corrected Visual Acuity (BCVA) in the Study Eye

    Change from baseline in BCVA in the study eye, measured in logMAR using the [FrACT] method.

    Measured: Baseline and Week 4, 8, 12, 24, 36 and W52 after administration

  2. Change From Baseline in Best-Corrected Visual Acuity (BCVA) in the Study Eye

    Change from baseline in BCVA in the study eye, measured in logMAR using the ETDRS method.

    Measured: Baseline and Weeks 4,8,12,24,36 and 52 after administration

  3. Change From Baseline in CLVQOL Total Score

    Change from baseline in the total score of the Chinese-version Low Vision Quality of Life (CLVQOL) questionnaire.The total score ranges from 0 to 100. Higher scores indicate better vision-related quality of life.

    Measured: Baseline and Week 12,24,36 and 52 after administration

  4. Change From Baseline in [Prespecified VEP Parameter] in the Study Eye

    Change from baseline in [parameter, e.g., P100 amplitude], measured by flash visual evoked potentials in the study eye.

    Measured: Baseline and [protocol-specified visits] through Week 52

  5. Change From Baseline in [Prespecified ERG Parameter] in the Study Eye

    Change from baseline in [parameter, e.g., b-wave amplitude], measured by [ERG test type] in the study eye.

    Measured: Baseline and [protocol-specified visits] through Week 52

  6. Change From Baseline in [Prespecified Visual Field Parameter] in the Study Eye

    Change from baseline in [parameter and unit], measured using [visual field test and program] in the study eye.

    Measured: Baseline and [protocol-specified visits] through Week 52

Timeline

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

  1. First posted29 Sep 2026
  2. Study start28 Sep 2026 (estimated)
  3. Primary completion15 Feb 2029 (estimated)
  4. Study completion15 Jun 2029 (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
Single group
Model description
This is a single-arm, open-label study employing a 3+3 dose escalation design to evaluate two UGX202 dose groups: the low-dose group (1.2×10¹¹ vg/eye) and the high-dose group (3.5×10¹¹ vg/eye). According to the "Dose Escalation Rule" (see below for details), each dose group comprised 3-6 study participants; a total of 6-12 study participants were enrolled in this phase.
Masking
None (open label)
Enrolment
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07847723
Sponsor's protocol ID
UGX202-S101
Acronym
UGX202
Status last verified
Sep 2026
First submitted
17 Sep 2026
Last updated
2 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No: Individual participant data will not be shared, as this was not included in the informed consent obtained from study participants.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing UGX202 Injection in people with Retinitis Pigmentosa (RP), run by Suzhou UgeneX Therapeutics Co., Ltd..

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 7 inclusion and 8 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: opening soon, as of the sponsor's last update on 2 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

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?

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?

Safety and tolerability (time frame: From baseline until week 52). 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 NCT07847723.

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 2 Oct 2026.

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