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Non-syndromic Retinitis PigmentosaPhase 1/2PIONEERNCT03326336

Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa

A Phase 1/2 study testing Gene therapy: GS030-DP AND Medical device: GS030-MD in people with Non-syndromic Retinitis Pigmentosa, run by GenSight Biologics at 3 sites in 3 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

The objective of this study is to evaluate the safety and tolerability of escalating doses of a gene therapy called GS030-DP (injected study treatment) administered via a single intravitreal injection and repeated light stimulation using a medical device called GS030-MD (stimulating glasses) in subjects with… Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Non-syndromic Retinitis Pigmentosa
What is being tested?
Gene therapy: GS030-DP AND Medical device: GS030-MD
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
3 sitesin 3 countries
Study dates
26 Sep 2018to 26 Oct 2027
Phase
Phase 1/2
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 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age ≥18 years to ≤75 years at the time of ICF signature.
  • Diagnosis of non-syndromic RP defined as:
  • Clinical diagnosis of non-syndromic RP based on history, mid-peripheral visual dysfunction, and fundoscopic appearance.
  • Diagnosis of non-syndromic RP is confirmed on full-field ERG
  • Visual acuity:
  • Visual acuity in the dose-escalation cohorts of no better LP.
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  • Visual acuity in the extension cohort of no better than CF pending review of dose-escalation cohort data by the DSMB.
  • Relatively preserved ganglion cell layer volume and retinal nerve fiber layer thickness, as measured with spectral domain optical coherence tomography (SD-OCT).
  • Interpupillary distance of ≥51 mm and ≤72 mm.
  • Refractive error of the study eye between -6 diopters and +6 diopters.

Who cannot join

  • Prior receipt of any gene therapy.
  • Subjects who have undergone significant ocular surgery (per investigator determination) within 3 months prior to Visit 1.
  • Presence of narrow iridocorneal angles contraindicating pupillary dilation.
  • Presence of disorders of the ocular media which interfere with visual acuity and other ocular assessments, including SD-OCT, during the study period.
  • Presence of any systemic or ocular diseases, or pathologies, other than non-syndromic RP, or their associated therapies, that can cause or have the potential to cause vision loss.
  • Prior vitrectomy or vitreomacular surgery.
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  • Presence of vitreo-macular adhesion or traction, epiretinal membrane, macular pucker and macular hole, evident by ophthalmoscopy and/or by SD-OCT examinations and assessed by the investigator to significantly affect central vision.
  • Current evidence of retinal detachment assessed by the investigator to significantly affect central vision.
  • Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.
  • Presence of an Active Implantable Medical Device.
  • Subjects who have undergone thermal laser procedure to the retina within 3 months of trial entry, or any prior thermal laser procedure to the macular region.

Trial locations and contacts

3 sites in 3 countries.

3 sites in 3 countries

United States1 site

  • UPMC Eye Center

    Pittsburgh, Pennsylvania, 15213

Other trials in United States

France1 site

  • Centre Hospitalier National d'Ophtalmologie (CHNP) des Quinze-Vingts

    Paris, 75012

Other trials in France

United Kingdom1 site

  • Moorfields Eye Hospital NHS Foundation Trust, 162 City Road

    London

Other trials in United Kingdom

About this study

Official title: A Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa

The objective of this study is to evaluate the safety and tolerability of escalating doses of a gene therapy called GS030-DP (injected study treatment) administered via a single intravitreal injection and repeated light stimulation using a medical device called GS030-MD (stimulating glasses) in subjects with documented diagnosis of non-syndromic Retinitis Pigmentosa

Read the detailed description

Study GS030_CLIN_001 is a multicenter, Phase 1/2a, open-label, non-randomized, dose-escalation, safety and tolerability study of GS030-DP in association with GS030-MD in subjects with non-syndromic RP that is confirmed by full-field ERG. The study design includes a dose escalation of the vector encoding the ChR-tdT. This first-in-human study design evaluates the safety and tolerability of GS030, the associated GS030-MD and GS030-DP, which is the treatment to be developed and considered for marketing.

Being tested
Gene therapy: GS030-DP AND Medical device: GS030-MD
Keywords
Eye Diseases, Hereditary Eye Diseases, Retinal degeneration, Inherited retinal diseases, Rod & cone dystrophies, Retinitis Pigmentosa, Gene Therapy, Optogenetic, Intravitreal Injections, AAV Vectors, Medical device, Visual Interface
Medical subject headings
Eye Diseases, Eye Diseases, Hereditary, Retinal Degeneration, Cone-Rod Dystrophies, Retinitis Pigmentosa

What participants receive

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

  • Gene therapy: GS030-DP AND Medical device: GS030-MDCombination product

    GS030-Drug Product (GS030-DP) - Recombinant adeno-associated viral vector, derived from serotype 2 (rAAV2.7m8), containing the optimized channelrhodopsin ChrimsonR-tdTomato gene under the control of the ubiquitous CAG promoter (rAAV2.7m8-CAG-ChrimsonR-tdTomato) GS030-Medical Device (GS030-MD) - Visual Interface Stimulating Glasses (that amplify the external visual stimulus to the optogenetically engineered retina)

Study groups in NCT03326336
Study groupRoleWhat they receive
CohortOther3 dose escalation cohorts (low, medium and high dose) with 3 subjects per cohort followed by an extension cohort at the highest-well tolerated dose with 3 to 9 subjects.
Receives: Combination Product: Gene therapy: GS030-DP AND Medical device: GS030-MD

What is being measured

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

  1. The safety and tolerability of escalating doses of GS030-DP administered via a single IVT and repeated light stimulation using GS030-MD in subjects with non-syndromic Retinitis Pigmentosa

    Safety and tolerability of GS030 treatment at Week 52/Year 1, by assessments based on local and systemic safety issues, specifically those related to IVT of GS030-DP and the subsequent repeated use of GS030-MD, as assessed by incidence of Adverse Events.

    Measured: Week 52/Year 1

Secondary outcomes (7)

  1. Evaluate the treatment effect of GS030 as assessed by visual acuity

    Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with the Freiburg Visual Acuity \& Contrast Test (FrACT) free computer program that uses graphics capabilities and psychometric methods to provide automated, self-paced measurement and the full field threshold stimulus test (FST) measuring the illuminance necessary to stimulate the most sensitive parts of the retina, and thus determines a quantifiable stimulation threshold (before and after gene transfer, with GS030-MD turned ON and turned OFF).

    Measured: Week 52/Year 1

  2. Evaluate the treatment effect of GS030 as assessed by visual function

    Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with Humphrey visual field 10-2 Standardized automated perimetry, square localization test, displaying white square at a random location on a black background and direction of motion test measuring the ability of subjects to determine the direction of an object moving in the visual field (before and after gene transfer, with GS030-MD turned ON and turned OFF).

    Measured: Week 52/Year 1

  3. Evaluate the treatment effect of GS030 as assessed by mobility (door task).

    Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with door task (before and after gene transfer, with GS030-MD turned ON and turned OFF).

    Measured: Week 52/Year 1

  4. Evaluate the treatment effect of GS030 as assessed by mobility (line task).

    Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with line task (before and after gene transfer, with GS030-MD turned ON and turned OFF).

    Measured: Week 52/Year 1

  5. Evaluate the treatment effect of GS030 as assessed by QoL (VFQ25)

    Assessment of the treatment effect on quality of life changes from baseline to Week 52 with the Visual Functioning Questionnaire-25 (VFQ-25). VFQ25 is a 25-item questionnaire with 47 questions, each question has several responses scored on a scale from 0 to 5, 0 to 6, or 0 to 10. Values are calculated in percentages.

    Measured: Week 52/Year 1

  6. Evaluate the treatment effect of GS030 as assessed by QoL (SF36)

    Assessment of the treatment effect on quality of life changes from baseline to Week 52 with the Short Form Survey 36 Version 2 (SF-36v2). The SF-36v2 is a subject-rated 36-item questionnaire assessing subject health. There are 8 scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0 to 100 scale. A lower score indicates more disability, and a higher score indicates less disability (a score of 0 is equivalent to maximum disability, and a score of 100 is equivalent to no disability).

    Measured: Week 52/Year 1

  7. Evaluate immune response to recombinant adeno associated viral vector, derived from serotype 2 (rAAV2.7m8) and ChR tdT protein.

    Immune response to rAAV2.7m8 and ChR-tdT protein from baseline to week 52

    Measured: Week 52/Year 1

Timeline

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

  1. First posted31 Oct 2017
  2. Study start26 Sep 2018
  3. Primary completion26 Oct 2027 (estimated)
  4. Study completion26 Oct 2027 (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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Model description
3 dose-escalation cohorts with 3 subjects per each cohort plus 1 extension cohort at the highest well-tolerated dose.
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03326336
Sponsor's protocol ID
GS030_CLIN_001
Other IDs
2017-002204-27, 2017
Acronym
PIONEER
Status last verified
Feb 2026
First submitted
11 Oct 2017
Last updated
2 Mar 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
Yes

Publications

  • Sahel JA, Futterknecht S, Audo I, Martel JN, Ling S, Pagot C, Arleo A, Boulanger-Scemama E, Duret A, de Saint Aubert JB, Delaux A, Degli Esposti S, Coen C, de Montleau C, Aravich D, Smith W, Hayem C, Taiel M, Behrmann M, Roska B. Optogenetic Therapy for Restoring Aspects of Visual Function. N Engl J Med. 2026 Oct 8;395(14):1399-1408. doi: 10.1056/NEJMoa2602215.

    PubMed 42842903

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Gene therapy: GS030-DP AND Medical device: GS030-MD in people with Non-syndromic Retinitis Pigmentosa, run by GenSight Biologics at 3 sites in 3 countries.

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 10 inclusion and 11 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: under way, not looking for participants, as of the sponsor's last update on 2 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States, France, United Kingdom. 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

The safety and tolerability of escalating doses of GS030-DP administered via a single IVT and repeated light stimulation using GS030-MD in subjects with non-syndromic Retinitis Pigmentosa (time frame: Week 52/Year 1). 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 NCT03326336, which also lists 1 linked publication.

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 Mar 2026.

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