RGX-314 Gene Therapy for Diabetic Retinopathy

(ALTITUDE® Trial)

Not currently recruiting at 45 trial locations

Overseen By Patient Advocacy

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: REGENXBIO, Inc.

Must be taking: Anti-VEGF

Disqualifiers: Retinal detachment, Vitrectomy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 12

Duration: 7 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new gene therapy called RGX-314 for diabetic retinopathy, a common eye condition in people with diabetes that can lead to vision loss. The study aims to determine if this one-time treatment can reduce the need for regular eye injections. Participants will receive either the new treatment, a standard treatment, or be observed without intervention. Those with diabetic retinopathy who have had diabetes for some time might be suitable candidates, especially if they frequently experience eye issues related to their condition. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important advancements in diabetic retinopathy care.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, it mentions that anti-VEGF injections can be safely deferred for at least 6 months for some participants.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that ABBV-RGX-314, a gene therapy being tested for diabetic eye disease, is generally well tolerated. In studies with 50 patients who received the first two doses, no serious side effects were directly linked to the treatment. This is encouraging news for those considering participation in clinical trials.

It's important to remember that ABBV-RGX-314 remains under investigation, so researchers are closely monitoring its safety and effectiveness. While no serious side effects have emerged so far, ongoing studies will provide more detailed safety information. For now, current data suggests that the treatment does not raise major concerns at the doses tested. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about RGX-314 for diabetic retinopathy because it introduces a novel gene therapy approach. Unlike standard treatments like anti-VEGF injections, RGX-314 aims to provide long-term benefits by delivering a gene that produces an anti-VEGF antibody fragment directly to the eye, potentially reducing or eliminating the need for frequent injections. This gene-based delivery could offer a more convenient and sustained treatment option for patients, which is a significant advancement over current therapies. Additionally, the inclusion of a topical steroid in some treatment arms may help manage inflammation, further enhancing the treatment's effectiveness.

What evidence suggests that this trial's treatments could be effective for diabetic retinopathy?

Research has shown that RGX-314 gene therapy, which participants in this trial may receive, offers promising results for diabetic retinopathy. In earlier studies, patients required 97% fewer ongoing treatments nine months after receiving RGX-314. This therapy is designed as a one-time treatment, potentially reducing the severity of diabetic retinopathy and lowering the risk of worsening vision problems. It delivers a gene that helps the eye control harmful blood vessel growth and reduce swelling. Although still under study, these early results suggest it could significantly reduce the treatment burden and improve outcomes for people with diabetic retinopathy. ^{1 2 3 4 5}

Are You a Good Fit for This Trial?

This trial is for adults aged 25-89 with diabetic retinopathy, who have good vision (20/40 or better) and can delay other treatments like PRP or anti-VEGF injections for at least 6 months. It's not for those with active macular edema, prior eye surgeries, potential pregnancy, or retinal issues not caused by diabetes.

Inclusion Criteria

I am 25-89 years old with diabetic retinopathy and can delay certain treatments for 6 months.

Your vision in the study eye is 20/40 or better.

It's okay if you have had a certain eye condition in the past in the eye being studied.

Exclusion Criteria

I have active diabetic macular edema.

I have had PRP or retinal laser treatment in my study eye.

My eye condition is not due to diabetic retinopathy.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ABBV-RGX-314 gene therapy via a single suprachoroidal space injection, with some cohorts receiving a protocol-mandated steroid regimen

7 weeks

Multiple visits for administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

48 weeks

Regular visits for assessment at Week 12, Week 24, Week 36, and Week 48

Open-label extension (optional)

Participants may opt into continuation of treatment long-term

Up to Week 82

What Are the Treatments Tested in This Trial?

Interventions

• RGX-314

Trial Overview The study tests RGX-314 gene therapy as a one-time treatment for diabetic retinopathy. Participants will receive different doses of RGX-314 administered into the suprachoroidal space of the eye to see if it can improve their condition without frequent treatments.

How Is the Trial Designed?

Group I: Part 2: ABBV-RGX-314 Treatment Arm (Dose 4) and Topical Steroid Experimental Treatment 2 Interventions

ABBV-RGX-314 Dose 4 and Topical Steroid

Group II: Part 1: ABBV-RGX-314 Treatment Arm (Dose 3) and Topical Steroid Experimental Treatment 2 Interventions

ABBV-RGX-314 Dose 3 and Topical Steroid

Group III: Part 1: ABBV-RGX-314 Treatment Arm (Dose 2) Experimental Treatment 1 Intervention

ABBV-RGX-314 Dose 2

Group IV: Part 1: ABBV-RGX-314 Treatment Arm (Dose 1) Experimental Treatment 1 Intervention

ABBV-RGX-314 Dose 1

Group V: Part 1: Observation Control Arm Active Control 1 Intervention

Observation Control

Group VI: Part 2: Aflibercept Control Active Control 1 Intervention

Control treatment arm

Who Is Running the Clinical Trial?

REGENXBIO, Inc.

Lead Sponsor

Trials

20

Recruited

2,800+

Regenxbio Inc.

Lead Sponsor

Trials

20

Recruited

2,800+

AbbVie

Lead Sponsor

Trials

1,079

Recruited

535,000+

Founded

2013

Headquarters

North Chicago, USA

Known For

Immunology treatments

Top Products

Humira (adalimumab), Skyrizi (risankizumab), Rinvoq (upadacitinib)

Dr. Roopal Thakkar

AbbVie

Chief Medical Officer since 2023

MD from Wayne State University School of Medicine

Robert A. Michael

AbbVie

Chief Executive Officer

Bachelor's degree in Finance from the University of Illinois

REGENXBIO Inc.

Industry Sponsor

Trials

25

Recruited

3,100+

Published Research Related to This Trial

Gene therapy for neovascular age-related macular degeneration (nAMD) aims to provide a long-term solution by using adeno-associated virus (AAV) vectors to deliver anti-VEGF proteins, potentially reducing the need for frequent injections and improving visual outcomes.

Current research is focusing on optimizing gene delivery methods and exploring various therapeutic proteins, such as soluble fms-like tyrosine kinase-1 (sFLT-1) and other anti-angiogenic agents, to enhance the efficacy and safety of treatments for AMD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Gene therapy for age-related macular degeneration. Moore, NA., Bracha, P., Hussain, RM., et al.[2018]

The first-in-human phase 1/2 clinical trial of retinal gene therapy for X-linked retinitis pigmentosa (RP) involving 18 patients showed that the treatment was generally safe, with only mild steroid-responsive inflammation noted at higher doses.

Significant visual field improvements were observed in six patients starting from one month after treatment and lasting through the 6-month follow-up, indicating potential efficacy of the gene therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR. Cehajic-Kapetanovic, J., Xue, K., Martinez-Fernandez de la Camara, C., et al.[2023]

The gene therapy rAAV.sFLT-1 was found to be safe and well tolerated in a phase 1 trial involving nine patients with wet age-related macular degeneration, with no drug-related adverse events reported.

A significant portion of the treatment group (67%) did not require any rescue injections after the therapy, suggesting that a single subretinal injection could provide long-term benefits compared to the frequent injections currently needed.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Gene therapy with recombinant adeno-associated vectors for neovascular age-related macular degeneration: 1 year follow-up of a phase 1 randomised clinical trial. Rakoczy, EP., Lai, CM., Magno, AL., et al.[2022]

Retinal gene therapies, particularly for age-related macular degeneration (AMD), are showing promise in clinical trials, with five therapies (Luxturna, ADVM-022, RGX-314, GT-005, and HMR59) being evaluated for their effectiveness against this common condition.

These therapies could potentially provide long-lasting treatment with a single administration, significantly changing the treatment landscape for chronic retinal diseases and reducing the ongoing burden of care for patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Review of gene therapies for age-related macular degeneration. Khanani, AM., Thomas, MJ., Aziz, AA., et al.[2023]

In a follow-up phase 1 trial involving 11 patients aged 11-46 years, the subretinal injection of AAV2-hRPE65v2 into the contralateral eye showed no serious adverse events related to the treatment, indicating a favorable safety profile.

Most patients experienced significant improvements in mobility and light sensitivity in the newly treated eye, with effects lasting up to 3 years, demonstrating the efficacy of AAV2-hRPE65v2 as a gene therapy for inherited retinal dystrophy caused by RPE65 mutations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial. Bennett, J., Wellman, J., Marshall, KA., et al.[2022]

Citations

1. ir.regenxbio.com ir.regenxbio.com/news-releases/news-release-details/regenxbio-presents-positive-data-phase-ii-study-subretinal-abbv

Release Details 97% reduction in treatment burden at nine months after treatment with ABBV-RGX-314 · Data consistent with that from multiple previous studies ...

2. regenxbio.gcs-web.com regenxbio.gcs-web.com/news-releases/news-release-details/regenxbio-presents-positive-one-year-data-phase-ii-altituder

Release Details - Regenxbio Inc A one-time, in-office injection of ABBV-RGX-314 gene therapy has the potential to stabilize and improve DR severity score and reduce the long-term risk of ...

3. news.abbvie.com news.abbvie.com/2025-01-13-AbbVie-and-REGENXBIO-Announce-Updates-on-the-ABBV-RGX-314-Clinical-Program

AbbVie and REGENXBIO Announce Updates on the ABBV ... ABBV-RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy and potentially other chronic retinal conditions.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT04567550

Study Details | NCT04567550 | RGX-314 Gene Therapy ... This phase 2, randomized, dose-escalation study is designed to evaluate the efficacy, safety and tolerability of ABBV-RGX-314 gene therapy in subjects with DR

5. ir.regenxbio.com ir.regenxbio.com/static-files/ec4713fe-ca17-4e07-800b-0fd194153a8c

Suprachoroidal Delivery of Sura-vec (ABBV-RGX-314) ABBV-RGX-314 is an investigational product that has not been approved by any regulatory authority. No conclusions regarding the safety and ...