Clinical Research

The physicians at Vitreoretinal Associates are actively involved in clinical research.

Dr. Christine Kay is the Director of Clinical Research and has a particular interest in inherited retinal disease, clinical trials involving gene therapy, age-related macular degeneration, and diabetic macular edema.

If you are interested in learning more about a clinical trial you can directly reach out to our Clinical Research team for more information:

Hailey Ahrens
352-300-8412
Hailey@vra-pa.com

Dr. Christine Kay on Gene Therapy, Stem Cell Therapy, and living with XLRS and Stargardt disease:

See Brendon’s story about living with XLRS:

 

See Sam’s story about living with Stargardt disease:

 

Additional Video Links

Phase 2 Data on Gene Therapy for AMD
The PRISM clinical trial assesses 4D-150 (4D Molecular Therapeutics) in adults with neovascular age-related macular degeneration, also known as wet AMD
 

Clinical Trials Training at Women in Ophthalmology

Experts advise taking advantage of research opportunities and developing relationships with industry partners.

Click here for the article.

By: Christine Kay, MD,  Lisa M. Nijm, MD, JD

 

ONGOING CLINICAL RESEARCH TRIALS

To find out more about any of these trials, click this link and search for the identifier code

https://clinicaltrials.gov/

 

Wet Age-Related Macular Degeneration

1.  Phase 1/2 Dose-Escalation and Randomized, Controlled, Masked Expansion Trial of Intravitreal 4D-150 Gene Therapy in Adults with Neovascular (Wet) Age-Related Macular Degeneration  NCT05197270                                                                                                                         

2.A Randomized, Partially Masked, Controlled, Phase 2b/3 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 Gene Therapy in Participants with nAMD (ATMOSPHERE) NCT04704921                                                                           

3. A Randomized, Partially Masked, Controlled, Phase 3 Clinical Study to Evaluate the Efficacy and Safe  y of RGX-314 Gene Therapy in Participants with nAMD (ASCENT)   NCT05407636

4. A Long-term Follow-Up Study to Evaluate the Safety and Efficacy of RGX-314 Following Subretinal Administration in Participants with Neovascular Age-related Macular Degeneration and Fellow Eye Treatment Substudy NCT03999801                   

 5. A Phase 1/2 Dose Escalation and Dose Expansion Study to Evaluate the Safety, Tolerability, and Efficacy of One-Time                Intravitreal Dose of SAR402663 in Participants with Neovascular Age-Related Macular  IND 30528 

6. A Phase 3, Multicenter, Prospective, Randomized, Double-Masked, Parallel-Group Study of EYP-1901, a Tyrosine Kinase Inhibitor (TKI), Compared to Aflibercept in Subjects with Wet AMD   NCT04747197                                                                                                                                                                                                                         

7. A Multi-Center, Randomized, Double-Masked, Active-Comparator-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-Related Macular Degeneration (ARTEMIS) NCT06856577

 

      Dry Age-Related Macular Degeneration and Geographic Atrophy

  1. ORACLE: A Long-Term Follow-up Study to Evaluate the Safety of GT005 in PARTICIPANTS with GEOGRAPHIC ATROPHY SECONDARY TO Age-Related Macular Degeneration treated in a Gyroscope-sponsored Antecedent Study

          NCT05481827                 Sponsor: Gyroscope/Novartis                                                                                                                                                     Not currently enrolling.

  1. ReNEW: A Phase 3, Randomized, Double-Masked, Placebo-Controlled Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects who have Dry Age-Related Macular Degeneration (Dry AMD)

          NCT0637373  Sponsor: Stealth BioTherapeutics Inc.                                                                                                                                             

  1. Long-term Extension Study for Participants with Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD) in JNJ-81201887 Parent Clinical Studies                                                                                                                                                       NCT06635148  Sponsor: Janssen 
  2. 4. JADE: Phase II Trial in Patients With Geographic Atrophy: A Randomized, Double-masked, Placebo-controlled, Dose-finding Study to Evaluate the Efficacy and Safety of BI 1584862  NCT06769048 Sponsor: Boehringer Ingelheim
 

DIABETIC MACULAR EDEMA

  1. A Randomized, Double-Masked, Multi-Center, 3-Arm Pivotal Phase 2/3 Study to Evaluate the Efficacy and Safety of Intravitreal EYE103 Compared With Intravitreal Ranibizumab (0.5mg) in Participants With Diabetic Macular Edema

          NCT06571045 Sponsor: EyeBio 

 

RETINITIS PIGMENTOSA

1. A Phase III, Multicenter, Randomized, Double Masked, Placebo-Controlled Study of the Efficacy and Safety of Oral N-Acetylcysteine in Patients with Retinitis Pigmentosa (NAC ATTACK)

Sponsor: National Eye Institute https://www.nei.nih.gov/research/clinical-trials

ClinicalTrials.gov Identifier: NCT05537220

2. Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene Protocol MGT-RPGR-022, Phase 3

Sponsor: MeiraGTx UK II Ltd. https://meiragtx.com/

ClinicalTrials.gov Identifier: NCT04794101

3. Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene

Protocol MGT-RPGR-021, Phase 3 https://meiragtx.com/

Sponsor: MeiraGTx UK II Ltd.

ClinicalTrials.gov Identifier: NCT04671433

4. An Open-label, Phase 1/2 Trial of Gene Therapy 4D-125 in Males with X-linked Retinitis Pigmentosa (XLRP) Caused by Mutations in the RPGR Gene

Sponsor: 4D Molecular Therapeutics, Inc. https://4dmoleculartherapeutics.com/

ClinicalTrials.gov Identifier: NCT04517149

 

STARGART DISEASE

1. Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

Sponsor: Alkeus Pharmaceuticals, Inc. https://www.alkeuspharma.com/

ClinicalTrials.gov Identifier: NCT04239625

 

2. Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease.

Sponsor: Alkeus Pharmaceuticals, Inc. https://www.alkeuspharma.com/

ClinicalTrials.gov identifier: NCT02402660

 

3. Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Adolescent Subjects With Stargardt Disease

Sponsor: Belite Bio, Inc https://belitebio.com/

Protocol Number: LBS-008-CT03

ClinicalTrials.gov Identifier: NCT05244304

 

4. A Phase 2b/3 Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of STG-001 in Subjects with Stargardt Disease (STGD1) Caused by Autosomal Recessive Mutation in ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene

Sponsor: Stargazer Pharmaceuticals, Inc.

ClinicalTrials.gov Identifier: NCT04489511

 

5. Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)

Sponsor: Ophthotech Corporation (IVERIC bio, Inc.) https://ivericbio.com/clinical-programs/

ClinicalTrials.gov Identifier: NCT03364153

6. ACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal     ACDN-01 in Participants with ABCA4-related Retinopath NCT06467344

 

RETINAL DEGENERATION NATURAL HISTORY STUDIES

1. Rate of Progression in EYS Related Retinal Degeneration (Pro-EYS)

Sponsor: Jaeb Center for Health Research https://www.jaeb.org/projects/

ClinicalTrials.gov Identifier: NCT04127006

2. Rate of Progression in USH2A related Retinal Degeneration (RUSH2A)

Sponsor: Jaeb Center for Health Research https://www.jaeb.org/projects/

Clinical Trials Identifier: NCT03146078

 

CHOROIDEREMIA

Long-term Safety and Efficacy Follow-up of AAV2-REP1 for the Treatment of Choroideremia (SOLSTICE)

Sponsor: NightstaRx Ltd, a Biogen Company https://www.bioindustry.org/member/nightstarx-ltd.html

ClinicalTrials.gov Identifier: NCT03584165

 

ACHROMATOPSIA

Phase 1/2 Safety and Efficacy Trial of AAV Gene Therapy in Patients with CNGB3 Achromatopsia

Sponsor: Applied Genetic Technologies Corp https://agtc.com/

ClinicalTrials.gov identifier: NCT02599922

 

Phase 1/2 Safety and Efficacy Trial of AAV Gene Therapy in Patients with CNGA3 Achromatopsia

Sponsor: Applied Genetic Technologies Corp https://agtc.com/

ClinicalTrials.gov identifier: NCT02935517