Stargardt Disease study: ASP2020
Official title A Study About the Safety of a Single ASP2020 Eye Injection and if it Helps People With Vision Loss From Stargardt-type Eye Conditions
ClinicalTrials.gov ID: NCT07734064
What this study is testing
What is ASP2020?
ASP2020 is an investigational stem‑cell‑based therapy being studied as a potential treatment for Stargardt‑type macular dystrophies. ASP2020 is designed to replace damaged macular cells with laboratory‑grown human stem‑cell‑derived retinal cells delivered by subretinal injection.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is testing whether a single injection of ASP2020 (stem cells turned into eye cells) is safe and tolerated in people with Stargardt‑type macular dystrophies.
- Who it's for
- You may be able to join if you are a child, teenager, or adult with Stargardt disease or a similar macular dystrophy.
- What's involved
- You would have one surgery where you are asleep and receive an injection into the eye, plus a steroid shot to reduce swelling. You will then attend several clinic visits for eye exams, imaging, and blood tests over about a year.
- Time commitment
- The study lasts about one year and includes multiple follow‑up visits after the injection.
- Phase 1: an early, usually small safety study
- Time commitment: about 1 year
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 and older
You may be able to join if
- STGD: presence of biallelic (pathogenic or likely pathogenic) ABCA4 variants, or one definite disease-causing ABCA4 variant together with a typical...
- STGD-like macular dystrophy: presence of one or more pathogenic variants in a gene known to cause macular dystrophy, as appropriate for its expected...
- Sufficiently clear ocular media and adequate pupillary dilation to allow for all imaging procedures.
- Intraocular pressure (IOP) of ≤ 21 mmHg
- Participant has a spherical equivalent refractive error between +8.00 D and -10.00 D.
You likely can't join if
- Participant has a known history of significant systemic disease that could impact ocular health or confound study assessments, based on medical...
- Participant has an autoimmune condition that requires treatment with immunomodulatory therapy and/or biologics that cause immunosuppression.
- Participant has known diagnosis of diabetes mellitus with a documented glycated hemoglobin (HbA1c) value ≥ 7% 3 months prior to screening and based...
- Participant has a history or evidence of severe cardiac disease, cardiovascular or cerebrovascular disease, including a history of stroke within 12...
- Participant has any complicating systemic disease or active malignancy.
- Participant has a known history of any systemic or metabolic condition, or physical examination finding that may significantly affect ocular health or...
See the full eligibility criteria
- STGD: presence of biallelic (pathogenic or likely pathogenic) ABCA4 variants, or one definite disease-causing ABCA4 variant together with a typical phenotype consistent with STGD.
- STGD-like macular dystrophy: presence of one or more pathogenic variants in a gene known to cause macular dystrophy, as appropriate for its expected inheritance mode.
- Sufficiently clear ocular media and adequate pupillary dilation to allow for all imaging procedures.
- Intraocular pressure (IOP) of ≤ 21 mmHg
- Participant has a spherical equivalent refractive error between +8.00 D and -10.00 D.
- BCVA ranging from 20/500 to 20/40 (equivalent to 15 to 70 ETDRS letters)
- For participants in the \> 20/80 to ≤ 20/40 BCVA range (moderate visual impairment [MVI]): presence of a visible definite or probable residual ellipsoid zone (EZ) on SD-OCT, and a total retinal SD-OCT central subfield...
- For participants in the ≥ 20/500 to ≤ 20/80 BCVA range (severe visual impairment): Presence of a residual ONL within the macular optical coherence tomography (OCT) scan area and Evidence of RPE disease/damage by means...
- Participant has a known history of significant systemic disease that could impact ocular health or confound study assessments, based on medical history or prior clinical documentation.
- Participant has an autoimmune condition that requires treatment with immunomodulatory therapy and/or biologics that cause immunosuppression.
- Participant has known diagnosis of diabetes mellitus with a documented glycated hemoglobin (HbA1c) value ≥ 7% 3 months prior to screening and based on available medical records.
- Participant has a history or evidence of severe cardiac disease, cardiovascular or cerebrovascular disease, including a history of stroke within 12 months prior to screening.
- Participant has any complicating systemic disease or active malignancy.
- Participant has a known history of any systemic or metabolic condition, or physical examination finding that may significantly affect ocular health or interfere with the interpretation of study assessments.
- Participant has presence of another known or suspected molecular diagnosis of macular or retinal disease that could confound interpretation of study outcomes, indicate a second concomitant retinal condition, or suggest...
- Participant has macular atrophy due to any cause other than a genetically or clinically confirmed diagnosis of STGD or STGD-like macular dystrophy.
- Participant has evidence or history of choroidal neovascularization.
- Participant has diagnosis of any form of uncontrolled glaucoma (for high-tension glaucoma IOP \> 25 mmHg).
- Participant has a history of steroid-induced IOP elevation or known steroid responder status.
- Participant has and/or is receiving treatment for thyroid eye disease.
- Participant has diabetic retinopathy in excess of mild nonproliferative diabetic retinopathy
- Participant has any other disease(s) affecting the optic nerve.
- Participant has a history of anterior or posterior uveitis and/or presence of intraocular inflammation (trace anterior chamber cell or flare), or history of idiopathic or autoimmune-associated uveitis in either eye.
- Participant has media opacities impeding the visualization of the fundus and/or the reliable performance of the visual function tests required by the protocol.
- Participant has aphakia.
- Participant has a clinically significant epiretinal membrane or evidence of clinically significant vitreomacular traction syndrome.
- Participant has any other disorders which could interfere with or confound visual acuity and other ocular assessments, including OCT or FAF.
- Participant has history of any of the following procedures: posterior vitrectomy, retinal detachment surgery, glaucoma filtering surgery, glaucoma drainage device implantation, selective laser trabeculoplasty...
- Participant has had any intraocular surgery within 3 months of screening.
- Participant has a history of intraocular metallic foreign bodies.
- Participant has received any treatment including gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips, or any prior intravitreal treatment for any indication in either eye that may be...
- Participant has received within 1 month prior to screening or is receiving concomitant treatment with any ocular or systemic medication known to be toxic to the lens, retina, or optic nerve.
- Participant has received any investigational therapy within 3 months prior to screening.
- Participant has any condition, which makes the participant unsuitable for study participation.
The study team makes the final eligibility decision.
Where it is enrolling
- Phoenix, Arizona, United States
- Southaven, Mississippi, United States
- Cincinnati, Ohio, United States
- Dallas, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The study runs about 1 year per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 6 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Phoenix, Arizona, United States; Southaven, Mississippi, United States; Cincinnati, Ohio, United States; Dallas, Texas, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.