Inherited Retinal Disease Clinical Trials

6 recruitingLast updated: June 24, 2026

There are 6 actively recruiting inherited retinal disease clinical trials across 3 countries. Studies span Phase 1, Phase 2, Early Phase 1, Not Applicable. Top locations include Beijing, Beijing Municipality, China, Ann Arbor, Michigan, United States, Dallas, Texas, United States. Updated daily from ClinicalTrials.gov.


Inherited Retinal Disease Trials at a Glance

6 actively recruiting trials for inherited retinal disease are listed on ClinicalTrialsFinder across 6 cities in 3 countries. The largest study group is Phase 1 with 3 trials, with the heaviest enrollment activity in Beijing, Ann Arbor, and Dallas. Lead sponsors running inherited retinal disease studies include Peking Union Medical College Hospital, BlueRock Therapeutics, and HuidaGene Therapeutics Co., Ltd..

Browse inherited retinal disease trials by phase

About Inherited Retinal Disease Clinical Trials

Looking for clinical trials for Inherited Retinal Disease? There are currently 1 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Inherited Retinal Disease trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Inherited Retinal Disease clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 16 of 6 trials

Recruiting
Early Phase 1

Safety and Efficacy Study of PUMCH-E111 Injection in Subjects With RLBP1 Related Inherited Retinal Dystrophy

Inherited Retinal DystrophyInherited Retinal Disease
Peking Union Medical College Hospital6 enrolled1 locationNCT07656753
Recruiting
Phase 1Phase 2

A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)

Retinitis pigmentosa (RP)Retinal DegenerationPrimary Photoreceptor Disease+6 more
BlueRock Therapeutics54 enrolled4 locationsNCT06789445
Recruiting
Not Applicable

Optimizing Low Vision Rehabilitation in Emotionally Distressed Patients With Inherited Retinal Diseases

Inherited Retinal Diseases
University of Michigan180 enrolled1 locationNCT06651736
Recruiting
Phase 1Phase 2

A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS)

Retinitis PigmentosaChoroideremiaNon-Syndromic Rod-Dominant Inherited Retinal Diseases
Kiora Pharmaceuticals, Inc.48 enrolled3 locationsNCT05282953
Recruiting
Early Phase 1

Safety and Efficacy Study of PUMCH-E101 Injection in Subjects with RDH12 Retinopathy

Inherited Retinal Diseases
Peking Union Medical College Hospital10 enrolled1 locationNCT06749639
Recruiting
Phase 1Phase 2

Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)

Leber Congenital AmaurosisInherited Retinal Diseases Caused by RPE65 Mutations
HuidaGene Therapeutics Co., Ltd.20 enrolled3 locationsNCT05906953