Health TrialsbyHealthPanel

Stargardt Disease 1

4

Review clinical trials related to Stargardt Disease 1. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

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

This study is an open-label, single ascending dose clinical trial in participants who have ABCA4-related retinopathies. This is the first-in-human clinical trial in which ACDN-01 will be evaluated for safety, tolerability, and preliminary efficacy following a single subretinal injection of ACDN-01.

Participants needed: 15
Trial details
Phase: Phase 1, Phase 2Age: 12+Biological sex: AllType: InterventionalSponsor: Ascidian Therapeutics, IncUpdated: Oct 7, 2026Locations: 11
Eligibility criteria

Presence of mutations in the ABCA4 gene [+3]

The presence of pathogenic or likely pathogenic mutations in other genes known t... [+2]

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Status: Recruiting

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

This is an observational prescreening study. Individuals who are eligible for prescreening will undergo testing procedures that may be used to determine eligibility in ACDN-01 clinical trials.

Participants needed: 50
Trial details
Age: 5+Biological sex: AllType: ObservationalSponsor: Ascidian Therapeutics, IncUpdated: Oct 9, 2026Locations: 9
Eligibility criteria

Presence of mutations in the ABCA4 gene [+1]

The presence of pathogenic or likely pathogenic mutations in other genes known t... [+2]

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Status: Recruiting

A Phase 1, First-in-Human Study of CITY-RBP4 in Healthy Volunteers and STGD1 Participants

This is a first-in-human (FIH), multicenter, randomized, double-masked, placebo-controlled adaptive clinical study evaluating the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) of CITY-RBP4, following single-ascending doses and multiple doses, via subcutaneous (SC) administration.

Participants needed: 48
Trial details
Phase: Phase 1Age: 12-45Biological sex: AllType: InterventionalSponsor: City TherapeuticsUpdated: Sep 23, 2026Locations: 1
Eligibility criteria

Willing and able to provide written informed consent form [+7]

Clinically significant ocular disease or prior ocular surgery that could interfe... [+18]

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Status: Recruiting

Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)

This is a single-arm, open-label, non-randomized, single dose-escalation, first-in-human (FIH) clinical trial to evaluate the safety and preliminary efficacy of VG801 for treatment of patients with retinal dystrophy (Stargardt disease) due to biallelic ABCA4 mutations.

Participants needed: 18
Trial details
Phase: Phase 1, Phase 2Age: 6+Biological sex: AllType: InterventionalSponsor: VeonGen Therapeutics GmbHUpdated: Sep 2, 2026Locations: 1
Eligibility criteria

Written informed consent. [+4]

Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy o... [+5]

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