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2026 data Public-data reference. official source

Stargardt Disease

Open-data reference.

17 US clinical trials · 14 currently recruiting

Active & Recent Trials

RECRUITING NA 500 participants

Stem Cell Ophthalmology Treatment Study II

MD Stem Cells

NCT03011541

RECRUITING 120 participants

Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal Degeneration

Ray Therapeutics

NCT06375239

RECRUITING 90 participants

A Study to Learn How Stargardt-type Eye Conditions Progress in Children and Adults

Astellas Pharma Global Development

NCT07425574

RECRUITING 80 participants

An Observational Study in Children and Adults With Stargardt Disease

AAVantgarde Bio Srl

NCT06591806

RECRUITING 75 participants

An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene

Splice Bio

NCT06435000

RECRUITING Phase 1 75 participants

A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)

AAVantgarde Bio Srl

NCT07161544

RECRUITING Phase 1 57 participants

A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)

Splice Bio

NCT06942572

RECRUITING Phase 1 56 participants

Oral Metformin for Treatment of ABCA4 Retinopathy

National Eye Institute (NEI)

NCT04545736

RECRUITING Phase 2 51 participants

A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease

Ocugen

NCT05956626

RECRUITING 50 participants

Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH)

Ascidian Therapeutics

NCT06445322

RECRUITING 30 participants

A Prospective Observational Study to Assess the Reliability and Validity of the MLSDT

Nanoscope Therapeutics

NCT06805474

RECRUITING Phase 1 18 participants

Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease

Ray Therapeutics

NCT07439887

RECRUITING Phase 1 15 participants

The Effects of Disulfiram (Antabuse®) on Visual Acuity in Patients With Retinal Degeneration

University of Rochester

NCT06319872

RECRUITING Phase 1 15 participants

Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)

Ascidian Therapeutics

NCT06467344

ACTIVE NOT RECRUITING Phase 2 200 participants

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

Alkeus Pharmaceuticals

NCT04239625

ACTIVE NOT RECRUITING 6 participants

Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study

Nanoscope Therapeutics

NCT06048185

COMPLETED Phase 2 23 participants

Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease

Kubota Vision

NCT03033108

Phase Distribution

PhaseTrial count
Early Phase 1 6
Phase 2 3

Top Sponsors

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Reading the Stargardt Disease Trial Landscape

ClinicalTrials.gov lists 17 US studies indexed under Stargardt Disease, and 14 of those are currently open to recruitment — roughly 82% of the total volume on the registry. That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.

The phase distribution for Stargardt Disease shows 0 late-stage studies (Phase 3 and Phase 4 combined) alongside 9 earlier-phase entries (Phase 1 through Phase 2). Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.

Top sponsor activity for Stargardt Disease is led by Nanoscope Therapeutics with 2 indexed trials, alongside 9 other organizations in the top contributor list. The list on this page surfaces up to 17 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation — eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.

Frequently Asked Questions

How many clinical trials are there for Stargardt Disease?

PlainTrial tracks 17 US clinical trials for Stargardt Disease, of which 14 are currently recruiting participants. Data sourced from ClinicalTrials.gov.

How do I find a recruiting trial for Stargardt Disease?

Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.

Is this data current?

Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.

Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2024 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.

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