NCT03316560

Brief 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.

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
29

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Apr 2018

Longer than P75 for phase_1

Geographic Reach
1 country

4 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 10, 2017

Completed
10 days until next milestone

First Posted

Study publicly available on registry

October 20, 2017

Completed
6 months until next milestone

Study Start

First participant enrolled

April 16, 2018

Completed
5.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 9, 2023

Completed
1.3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2025

Completed
Last Updated

May 20, 2024

Status Verified

May 1, 2024

Enrollment Period

5.6 years

First QC Date

October 10, 2017

Last Update Submit

May 16, 2024

Conditions

X-Linked Retinitis Pigmentosa

Keywords

XLRPretinal degenerationRPGRadeno-associated virusgene therapyAAV

Outcome Measures

Primary Outcomes (2)

  • 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;

    Day 0 - Month 36

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

    Day 0 - Month 36

Secondary Outcomes (8)

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

    Day 0 - Month 36

  • Changes from baseline in visual acuity

    Day 0 - Month 36

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

    Day 0 - Month 36

  • Changes from baseline in quality of life questionnaire responses

    Day 0 - Month 36

  • Change from baseline in visual function by light-adapted perimetry

    Day 0 - Month 36

  • +3 more secondary outcomes

Study Arms (6)

Group 1: Phase 1/2 Dose Escalation

EXPERIMENTAL

Male subjects at least 18 y/o treated with Dose 1 of rAAV2tYF-GRK1-RPGR study drug.

Biological: rAAV2tYF-GRK1-RPGR

Group 2: Phase 1/2 Dose Escalation

EXPERIMENTAL

Male subjects at least 18 y/o treated with Dose 2 of rAAV2tYF-GRK1-RPGR study drug.

Biological: rAAV2tYF-GRK1-RPGR

Group 3 Phase 1/2 Dose Escalation

EXPERIMENTAL

Group 3 male subjects at least 18 y/o treated with Dose 3 of rAAV2tYF-GRK1-RPGR study drug.

Biological: rAAV2tYF-GRK1-RPGR

Group 4 Phase 1/2 Dose Escalation

EXPERIMENTAL

Group 4 male subjects at least 6 y/o treated with Dose 3 of rAAV2tYF-GRK1-RPGR study drug.

Biological: rAAV2tYF-GRK1-RPGR

Group 5 Phase 1/2 Dose Escalation

EXPERIMENTAL

Male subjects at least 18 y/o treated with Dose 5 of rAAV2tYF-GRK1-RPGR study drug.

Biological: rAAV2tYF-GRK1-RPGR

Group 6 Phase 1/2 Dose Escalation

EXPERIMENTAL

Male subjects at least 18 y/o treated with Dose 6 of rAAV2tYF-GRK1-RPGR study drug.

Biological: rAAV2tYF-GRK1-RPGR

Interventions

rAAV2tYF-GRK1-RPGRBIOLOGICAL

Adeno-associated virus vector expressing a human RPGR gene

Group 1: Phase 1/2 Dose EscalationGroup 2: Phase 1/2 Dose EscalationGroup 3 Phase 1/2 Dose EscalationGroup 4 Phase 1/2 Dose EscalationGroup 5 Phase 1/2 Dose EscalationGroup 6 Phase 1/2 Dose Escalation

Eligibility Criteria

Age6 Years - 50 Years
Sexmale(Gender-based eligibility)
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Duke University

Durham, North Carolina, 27701, United States

Location

Cincinnati Eye Institute

Cincinnati, Ohio, 45242, United States

Location

Casey Eye Institute

Portland, Oregon, 97239, United States

Location

Retina Foundation of the Southwest

Dallas, Texas, 75231, United States

Location

Related Publications (2)

  • 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.

    PMID: 40547876DERIVED

  • 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.

    PMID: 39680391DERIVED

MeSH Terms

Conditions

Retinal Degeneration

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal Diseases

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 10, 2017

First Posted

October 20, 2017

Study Start

April 16, 2018

Primary Completion

November 9, 2023

Study Completion

March 1, 2025

Last Updated

May 20, 2024

Record last verified: 2024-05

Data Sharing

IPD Sharing
Will not share

Locations

US(4)