AzTrials
AzTrials

X-Linked Retinitis PigmentosaPhase 1/2HORIZONNCT03316560

Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations

A Phase 1/2 study testing rAAV2tYF-GRK1-RPGR in people with X-Linked Retinitis Pigmentosa, run by Beacon Therapeutics at 4 sites in United States.

Active, not recruiting

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

Official record
Search all trials

Trial summary

This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations. Read more about this study

Who is it for?
Ages 6 to 50 years, men only
Conditions the study is for
X-Linked Retinitis Pigmentosa
What is being tested?
rAAV2tYF-GRK1-RPGR
Could I receive a placebo?
No, everyone receives an active treatment
How many people
29 participants
Where
4 sitesUnited States
Study dates
16 Apr 2018to Mar 2025
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 6 to 50 years
  • Men only
  • No healthy volunteers
  • Child, Adult

Phase 1/2 Dose Escalation Inclusion Criteria:

  • Male subjects with a documented RPGR mutation
  • Clinical diagnosis of X-linked retinitis pigmentosa (XLRP)
  • Best-corrected visual acuity not better than 78 ETDRS letters (20/32) in the study eye;
  • Ability to perform tests of visual and retinal function and structure and ability to comply with other research procedures;
  • Detectable baseline mean macular sensitivity, as measured by microperimetry.
  • Have detectable Ellipsoid Zone (EZ) line during the pre-treatment period as assessed by OCT and confirmed by the CRC.

Phase 1/2 Dose Escalation Exclusion Criteria:

  • Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints or increase the risk of surgical complications (for example, glaucoma, corneal or lenticular opacities, diabetic retinopathy, retinal vasculitis);
  • Use of anti-coagulant agents that may alter coagulation within 7 days prior to study agent administration;
  • Use of systemic corticosteroids or other immunosuppressive medications within 3 months prior to enrollment;
  • Any other condition that would prevent a subject from completing follow-up examinations during the course of the study;
  • Any other condition or reason that, in the opinion of the investigator, makes the subject unsuitable for the study;
  • Previous receipt of any AAV gene therapy product;
  • Monocular or having BCVA less than 20/800 in the fellow eye

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • Cincinnati Eye Institute

    Cincinnati, Ohio, 45242

  • Retina Foundation of the Southwest

    Dallas, Texas, 75231

  • Duke University

    Durham, North Carolina, 27701

  • Casey Eye Institute

    Portland, Oregon, 97239

Other trials in United States

About this study

Official title: A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene

This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.

Read the detailed description

This protocol includes a non-randomized, open-label, Phase 1/2 study (HORIZON). Approximately 30 participants will be enrolled into the dose escalation study (HORIZON). Each participant will receive the study agent by subretinal injection in one eye on a single occasion. Enrollment will begin with the lowest dose and will proceed to higher doses only after review of safety data by a Data and Safety Monitoring Committee (DSMC). There are a total of 15 visits over approximately 36 months, and long-term follow-up evaluations annually at years 4 and 5.

Being tested
rAAV2tYF-GRK1-RPGR
Keywords
XLRP, retinal degeneration, RPGR, adeno-associated virus, gene therapy, AAV
Medical subject headings
Retinal Degeneration

What participants receive

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

  • rAAV2tYF-GRK1-RPGRBiological

    Adeno-associated virus vector expressing a human RPGR gene

Study groups in NCT03316560
Study groupRoleWhat they receive
Group 1: Phase 1/2 Dose EscalationExperimentalMale subjects at least 18 y/o treated with Dose 1 of rAAV2tYF-GRK1-RPGR study drug.
Receives: Biological: rAAV2tYF-GRK1-RPGR
Group 2: Phase 1/2 Dose EscalationExperimentalMale subjects at least 18 y/o treated with Dose 2 of rAAV2tYF-GRK1-RPGR study drug.
Receives: Biological: rAAV2tYF-GRK1-RPGR
Group 3 Phase 1/2 Dose EscalationExperimentalGroup 3 male subjects at least 18 y/o treated with Dose 3 of rAAV2tYF-GRK1-RPGR study drug.
Receives: Biological: rAAV2tYF-GRK1-RPGR
Group 4 Phase 1/2 Dose EscalationExperimentalGroup 4 male subjects at least 6 y/o treated with Dose 3 of rAAV2tYF-GRK1-RPGR study drug.
Receives: Biological: rAAV2tYF-GRK1-RPGR
Group 5 Phase 1/2 Dose EscalationExperimentalMale subjects at least 18 y/o treated with Dose 5 of rAAV2tYF-GRK1-RPGR study drug.
Receives: Biological: rAAV2tYF-GRK1-RPGR
Group 6 Phase 1/2 Dose EscalationExperimentalMale subjects at least 18 y/o treated with Dose 6 of rAAV2tYF-GRK1-RPGR study drug.
Receives: Biological: rAAV2tYF-GRK1-RPGR

What is being measured

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

  1. Number and proportion of Adverse Events

    Number and proportion of participants experiencing Grade 3 or higher local (ocular) or systemic treatment-emergent adverse events that occur during the 36 months after study agent administration; number and proportion of participants experiencing treatment-emergent AEs, including treatment-emergent serious AEs;

    Measured: Day 0 - Month 36

  2. Number and proportion of participants experiencing abnormal clinically relevant hematology or clinical chemistry parameters.

    Measured: Day 0 - Month 36

Secondary outcomes (8)

  1. Changes from baseline in visual function as measured by mesopic microperimetry in the treated eye compared to the untreated eye

    Measured: Day 0 - Month 36

  2. Changes from baseline in visual acuity

    Measured: Day 0 - Month 36

  3. Changes from baseline in retinal structure as assessed by spectral-domain optical coherence tomography (SD-OCT)

    Measured: Day 0 - Month 36

  4. Changes from baseline in quality of life questionnaire responses

    Measured: Day 0 - Month 36

  5. Change from baseline in visual function by light-adapted perimetry

    Measured: Day 0 - Month 36

  6. Change from baseline in fundus imaging

    Measured: Day 0 - Month 36

  7. Change from baseline in full-field light sensitivity threshold (FST)

    Measured: Day 0 - Month 36

  8. Changes from baseline in visual function by dark-adapted full field perimetry (for subjects treated peripherally)

    Measured: Day 0 - Month 36

Timeline

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

  1. First posted20 Oct 2017
  2. Study start16 Apr 2018
  3. Primary completion9 Nov 2023
  4. Study completionMar 2025 (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
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
29 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03316560
Sponsor's protocol ID
AGTC-RPGR-001 HORIZON
Acronym
HORIZON
Status last verified
May 2024
First submitted
10 Oct 2017
Last updated
20 May 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Wang CY, Chen L, Lin TY, Huang SP. Systematic Identification of Candidate Genes for Inherited Retinal Disease Gene Therapy Integrating Worldwide IRD Cohort and Single-Cell Analysis. J Ophthalmol. 2025 Jun 12;2025:7014745. doi: 10.1155/joph/7014745. eCollection 2025.

    PubMed 40547876

  • Alibhai AY, De Pretto LR, Yaghy A, Woo KM, Dos Santos Xilau NR, Siddiqui H, Pandiscio CA, Homer A, Curtiss D, Waheed NK. Comparing the Thin Plate Spline and Gaussian Interpolation Methods in Generating Hill of Visions for X-Linked Retinitis Pigmentosa Patients. Transl Vis Sci Technol. 2024 Dec 2;13(12):26. doi: 10.1167/tvst.13.12.26.

    PubMed 39680391

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing rAAV2tYF-GRK1-RPGR in people with X-Linked Retinitis Pigmentosa, run by Beacon Therapeutics at 4 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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 20 May 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. 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?

Number and proportion of Adverse Events (time frame: Day 0 - Month 36). The study lists 2 primary outcomes in all. 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 NCT03316560, which also lists 2 linked publications.

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 20 May 2024.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in