Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Diabetic Retinopathy (DR) Without Center Involved-Diabetic Macular Edema (CI-DME) (ALTITUDE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04567550
Recruitment Status : Recruiting
First Posted : September 28, 2020
Last Update Posted : December 21, 2020
Sponsor:
Information provided by (Responsible Party):
Regenxbio Inc.

Brief Summary:
RGX-314 is being developed as a novel one-time gene therapy treatment for the treatment of diabetic retinopathy, a chronic and progressive complication of diabetes mellitus. Diabetic retinopathy is a sight-threatening disease characterized in the early stages by neuronal and vascular dysfunction in the retina, and later by neovascularization that leads to further deterioration of functional vision. Despite the availability of current treatments, diabetic retinopathy remains the leading cause of vision loss in working-age adults, those between the ages of 20 and 74. Existing treatment with anti-VEGF agents, although shown to be effective, are limited by short therapeutic half-lives, which then require frequent intravitreal injections over the patient's lifetime, resulting in increased risk of associated adverse events and significant treatment burden. Due to the burden of treatment, patients often do not closely adhere to treatment regimens and experience sub-optimal outcomes and a decline in vision. RGX-314 is being developed as a potential one time treatment for diabetic retinopathy, which may deliver advantages over conventional treatments, such as potentially providing a longer duration of therapeutic effect and intervening at an earlier stage of the disease.

Condition or disease Intervention/treatment Phase
Diabetic Retinopathy (DR) Biological: RGX-314 Dose 1 Biological: RGX-314 Dose 2 Phase 2

Detailed Description:
This phase 2, randomized, dose-escalation study is designed to evaluate the efficacy, safety and tolerability of RGX-314 gene therapy in subjects with DR without CI-DME. Approximately 40 participants who meet the inclusion/exclusion criteria, will be enrolled into one of 2 dose cohorts. Efficacy will be the primary focus of the study. Participants will be evaluated for safety and tolerability of RGX-314 throughout the study.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Dose-escalation, Observation-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of RGX-314 Gene Therapy Delivered Via One or Two Suprachoroidal Space (SCS) Injections in Participants With Diabetic Retinopathy (DR) Without Center Involved-Diabetic Macular Edema (CI-DME)(ALTITUDE)
Actual Study Start Date : November 20, 2020
Estimated Primary Completion Date : May 2021
Estimated Study Completion Date : May 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
No Intervention: Observation Control Arm
Observation Control
Experimental: RGX-314 Treatment Arm (Dose 1)
RGX-314 Dose 1
Biological: RGX-314 Dose 1
AAV8 vector containing a transgene for anti-VEGF fab (Dose 1)

Experimental: RGX-314 Treatment Arm (Dose 2)
RGX-314 Dose 2
Biological: RGX-314 Dose 2
AAV8 vector containing a transgene for anti-VEGF fab (Dose 2)




Primary Outcome Measures :
  1. To evaluate the effect of RGX-314 on DR by the Early Treatment Diabetic Retinopathy Study (ETDRS)-Diabetic Retinopathy Severity Scale (-DRSS) at week 48 [ Time Frame: 48 weeks ]
    Proportion of participants achieving a 2-step or greater improvement in DR by Early Treatment Diabetic Retinopathy Study (ETDRS)-Diabetic Retinopathy Severity Scale (-DRSS) at Week 48 scored from Levels 10-85 with higher levels indicating greater DR severity.


Secondary Outcome Measures :
  1. To evaluate the effect of RGX-314 on DR (ETDRS-DRSS) over time [ Time Frame: 48 weeks ]
    Proportion of participants achieving improvement or worsening in DR per ETDRS-DRSS grading through week 48

  2. To assess the safety and tolerability of RGX-314 [ Time Frame: 48 weeks ]
    Assess the incidence of overall and ocular Adverse Events (AEs)

  3. To evaluate the need for additional Standard of Care (SOC) intervention due to ocular diabetic complications [ Time Frame: 48 weeks ]
    Proportion of participants with sight-threatening ocular diabetic complications requiring additional SOC intervention (anti-VEGF, Pan-Retinal Photocoagulation (PRP), surgical intervention)



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   25 Years to 89 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients 25-89 years of age with a diabetic retinopathy diagnosis of nonproliferative diabetic retinopathy (NPDR) and proliferative diabetic retinopathy (PDR) secondary to diabetes mellitus Type 1 or 2 for which PRP or anti-VEGF injections can be safely deferred for at least 6 months
  • Best corrected visual acuity (BCVA) Early Treatment Diabetic Retinopathy Study (ETDRS) letter score in the study eye of ≥69 letters (approximate Snellen equivalent of 20/40 or better)
  • Prior history of CI-DME in the study eye is acceptable.
  • Must be willing and able to provide written, signed informed consent.

Exclusion Criteria:

  • Neovascularization in the study eye from a cause other than DR.
  • Presence of any active CI-DME.
  • Active or history of retinal detachment in the study eye.
  • Any evidence or documented history of PRP or retinal laser in the study eye.
  • Patients who had a prior vitrectomy surgery.
  • Women of childbearing potential.

Note: Other inclusions/exclusions criteria apply


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04567550


Contacts
Layout table for location contacts
Contact: Patient Advocacy 1-866-860-0117 patientadvocacy@regenxbio.com

Locations
Layout table for location information
United States, Arizona
Phoenix Location Recruiting
Phoenix, Arizona, United States, 85014
Principal Investigator: Phoenix Site PI         
United States, California
Bakersfield Location Not yet recruiting
Bakersfield, California, United States, 93309
Principal Investigator: Bakersfield Site PI         
Poway Location Recruiting
Poway, California, United States, 92064
Principal Investigator: Poway Site PI         
Santa Barbara Location Not yet recruiting
Santa Barbara, California, United States, 93103
Principal Investigator: Santa Barbara Site PI         
United States, Nevada
Reno Location Recruiting
Reno, Nevada, United States, 89502
Principal Investigator: Reno Site PI         
United States, New Mexico
Albuquerque Location Not yet recruiting
Albuquerque, New Mexico, United States, 87109
Principal Investigator: Albuquerque Site PI         
United States, Texas
Woodlands Location Recruiting
The Woodlands, Texas, United States, 77384
Principal Investigator: Woodlands Site PI         
Sponsors and Collaborators
Regenxbio Inc.
Layout table for additonal information
Responsible Party: Regenxbio Inc.
ClinicalTrials.gov Identifier: NCT04567550    
Other Study ID Numbers: RGX-314-2202
First Posted: September 28, 2020    Key Record Dates
Last Update Posted: December 21, 2020
Last Verified: December 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Regenxbio Inc.:
DR
Additional relevant MeSH terms:
Layout table for MeSH terms
Macular Edema
Retinal Diseases
Diabetic Retinopathy
Macular Degeneration
Retinal Degeneration
Eye Diseases
Diabetic Angiopathies
Vascular Diseases
Cardiovascular Diseases
Diabetes Complications
Diabetes Mellitus
Endocrine System Diseases