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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT03316560
Other study ID # AGTC-RPGR-001 HORIZON
Secondary ID
Status Active, not recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date April 16, 2018
Est. completion date March 2025

Study information

Verified date May 2024
Source Beacon Therapeutics
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical 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.


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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 29
Est. completion date March 2025
Est. primary completion date November 9, 2023
Accepts healthy volunteers No
Gender Male
Age group 6 Years to 50 Years
Eligibility 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

Study Design


Intervention

Biological:
rAAV2tYF-GRK1-RPGR
Adeno-associated virus vector expressing a human RPGR gene

Locations

Country Name City State
United States Cincinnati Eye Institute Cincinnati Ohio
United States Retina Foundation of the Southwest Dallas Texas
United States Duke University Durham North Carolina
United States Casey Eye Institute Portland Oregon

Sponsors (1)

Lead Sponsor Collaborator
Beacon Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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
Primary Number and proportion of participants experiencing abnormal clinically relevant hematology or clinical chemistry parameters. Day 0 - Month 36
Secondary Changes from baseline in visual function as measured by mesopic microperimetry in the treated eye compared to the untreated eye Day 0 - Month 36
Secondary Changes from baseline in visual acuity Day 0 - Month 36
Secondary Changes from baseline in retinal structure as assessed by spectral-domain optical coherence tomography (SD-OCT) Day 0 - Month 36
Secondary Changes from baseline in quality of life questionnaire responses Day 0 - Month 36
Secondary Change from baseline in visual function by light-adapted perimetry Day 0 - Month 36
Secondary Change from baseline in fundus imaging Day 0 - Month 36
Secondary Change from baseline in full-field light sensitivity threshold (FST) Day 0 - Month 36
Secondary Changes from baseline in visual function by dark-adapted full field perimetry (for subjects treated peripherally) Day 0 - Month 36
See also
  Status Clinical Trial Phase
Completed NCT04926129 - Natural History of the Progression of X-Linked Retinitis Pigmentosa
Recruiting NCT05926583 - A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa Phase 3
Enrolling by invitation NCT06275620 - A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN) Phase 2
Completed NCT03116113 - A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112 Phase 1/Phase 2
Recruiting NCT04850118 - A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP Phase 2/Phase 3
Active, not recruiting NCT04517149 - 4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP) Phase 1/Phase 2
Enrolling by invitation NCT03584165 - Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa Phase 3
Active, not recruiting NCT04312672 - Long-term Follow-up Gene Therapy Study for RPGR- XLRP
Completed NCT04868916 - An Observational Study of Japanese Participants With X-linked Retinitis Pigmentosa
Completed NCT03252847 - Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR) Phase 1/Phase 2
Recruiting NCT05874310 - Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa Early Phase 1
Active, not recruiting NCT04671433 - Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene Phase 3
Active, not recruiting NCT04794101 - Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene Phase 3
Completed NCT03314207 - Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)
Active, not recruiting NCT06333249 - A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE) Phase 2