Ongoing Clinical Trials for Stargardt’s Disease
Currently, there are 3 ongoing clinical trials investigating potential treatments for Stargardt’s disease, a genetic eye disorder that causes progressive vision loss. These trials are taking place across several European countries and are testing different approaches, including gene therapy and oral medications, to slow disease progression and preserve vision in affected patients.
Clinical trial locations
- Belgium
- France
- Germany
- Netherlands
Evaluation of SB-007 Safety and Efficacy in Patients with Stargardt Disease Type 1 (STGD1) Caused by ABCA4 Gene Mutations
This Phase 1/2 clinical trial is investigating SB-007, an experimental gene therapy treatment for Stargardt Disease Type 1. The treatment is delivered through a subretinal injection directly under the retina of the eye and contains an adeno-associated virus vector that delivers a corrected version of the ABCA4 gene to retinal cells.
Who can participate: Adults between 18 and 65 years old who have been diagnosed with Stargardt Disease Type 1 and have confirmed mutations in both copies of their ABCA4 gene. Participants must have clear eye tissue and adequate pupil dilation to allow for high-quality retinal imaging. Women of childbearing potential must have negative pregnancy tests and agree to use acceptable birth control for 4 months following treatment. The study eye must show clinical signs consistent with the disease.
Who cannot participate: People with other eye conditions that may affect vision beyond Stargardt Disease, those without confirmed mutations in both ABCA4 genes, anyone who has had eye surgery within 6 months, those with uncontrolled glaucoma, active eye infections or inflammation, or a history of retinal detachment. Pregnant or breastfeeding women, people with a history of cancer in the past 5 years (except certain skin cancers), those with serious medical conditions, and individuals with drug or alcohol abuse history are excluded. People who have participated in another clinical trial within 30 days or who have known allergies to the study medication components also cannot join.
What the study involves: The trial aims to evaluate whether SB-007 is safe, well-tolerated, and potentially effective at slowing disease progression. Participants will be randomly assigned to either receive the treatment in one eye or serve as a control group. Throughout the 96-week (approximately 2-year) follow-up period, researchers will monitor participants using various imaging techniques including fundus autofluorescence and optical coherence tomography to measure changes in lesion size, retinal layer integrity, and retinal sensitivity. Vision will be assessed through multiple tests measuring visual acuity and contrast sensitivity. Regular check-ups will document any changes in vision and disease progression.
This study is being conducted in Germany and is expected to run until September 2028.
Study on the Long-Term Safety of SAR422459 for Patients with Stargardt’s Disease
This is a long-term follow-up study evaluating the safety of SAR422459, a gene therapy treatment delivered through subretinal injection. The treatment uses a lentiviral vector containing the human ABCA4 gene to address the genetic cause of the disease.
Who can participate: This study is open only to patients who previously participated in a related study called protocol TDU13583, received a subretinal injection of SAR422459 in that study, and completed that study through Week 48 or had an early discontinuation visit. Participants must provide signed informed consent and any required local authorization such as HIPAA.
Who cannot participate: Patients with other congenital or hereditary diseases unrelated to Stargardt’s macular degeneration, those with other neonatal diseases not related to the condition, patients outside the specified age range, those not part of the specified trial group, and individuals considered part of a vulnerable population requiring special protection or care.
What the study involves: The primary objective is to evaluate the long-term safety and tolerability of SAR422459. Researchers will monitor the occurrence of adverse events and observe any clinically important changes in eye health. The study also aims to observe whether there is any delay in retinal degeneration. Participants will attend regular monitoring visits where their eye health will be assessed.
This study is taking place in France and is expected to continue until December 2033.
Study on Tinlarebant for Treating Stargardt Disease in Adolescents
This Phase 3 clinical trial is testing Tinlarebant (also known as LBS-008), an oral medication taken in tablet form at a 5 mg dosage. Unlike the gene therapy approaches, this treatment works by inhibiting the formation of toxic byproducts in the retina that contribute to retinal cell degeneration.
Who can participate: Male or female adolescents and young adults between 12 and 20 years old who have a clinical diagnosis of Stargardt Disease (STGD1) with at least one mutation found in the ABCA4 gene. The size of the affected area in the eye must be within 3 disc areas (7.62 mm²) as seen on fundus autofluorescence imaging, and participants must have visual acuity of 20/200 or better in the study eye. Both the participant and their parent or legal guardian must agree to sign the consent form approved by an ethics committee.
Who cannot participate: Patients with any other eye disease that could affect study results, those who have had eye surgery in the last 3 months, anyone currently taking medications that could interfere with the study treatment, people with a history of allergic reactions to similar medications, pregnant or breastfeeding individuals, those who have participated in another clinical trial within the last 30 days, and anyone with a serious health condition that could affect their ability to participate.
What the study involves: This is a double-masked, randomized study, meaning neither participants nor researchers will know who receives the actual medication versus placebo. After initial assessment including genetic testing and eye imaging, participants will be randomly assigned to receive either Tinlarebant or placebo. Regular follow-up visits will include eye examinations and imaging to assess changes in atrophic lesions (damaged areas in the retina). The main goal is to see if Tinlarebant can slow the growth of these areas and preserve retinal thickness and structure.
This study is being conducted across multiple countries including Belgium, Germany, the Netherlands, and France, and is expected to conclude by September 2025.
Summary
The current landscape of clinical trials for Stargardt’s disease reflects two distinct therapeutic approaches. Two of the three trials focus on gene therapy treatments (SB-007 and SAR422459) that aim to address the underlying genetic cause by delivering corrected versions of the ABCA4 gene directly to retinal cells through subretinal injection. The third trial tests an oral medication (Tinlarebant) that takes a different approach by preventing the accumulation of toxic substances in the retina.
Geographically, these trials span several European countries, with France and Germany hosting two trials each, while Belgium and the Netherlands participate in one trial. Notably, the Tinlarebant study is the only trial specifically designed for adolescents and young adults aged 12 to 20, while the SB-007 trial focuses on adults aged 18 to 65. The SAR422459 study represents a unique long-term safety follow-up for patients who previously received treatment, with monitoring extending until 2033.
All three trials share a common emphasis on monitoring disease progression through advanced retinal imaging techniques and assessing both safety and potential benefits for preserving vision in this progressive genetic eye disorder.
