Stargardt Disease Type 1 (STGD1)Early Phase 1STARTNCT07063251
An Clinical Study Evaluating the Safety, Tolerability, and efficAcy of HG005 in StaRgardT Disease
A Early Phase 1 study testing HG005 in people with Stargardt Disease Type 1 (STGD1), run by HuidaGene Therapeutics Co., Ltd. at 1 site in China.
Trial summary
Stargardt disease type 1 (STGD1) is a rare genetic eye condition that causes progressive vision loss, often beginning in childhood or adolescence. It is the most common form of inherited macular degeneration and can lead to legal blindness. Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Stargardt Disease Type 1 (STGD1)
- What is being tested?
- HG005
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteChina
- Study dates
- 20 Aug 2025to 28 Feb 2028
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patient ≥ 6 and ≤17 years at the time of signing informed consent, with clinical diagnosis of Stargardt disease;
- At least one ABCA4 allele on each chromosome;
- Both eyes must have well-defined macular atrophic lesions consistent with the diagnosis of Stargardt macular dystrophy.
- Meet visual acuity criteria based on ETDRS letter chart
- Subject must agree to contraception during the study.
- Acceptable hematology, clinical chemistry, urine laboratory, and protocol required eye examination.
Who cannot join
- Presence of active intraocular inflammation or uveitis history in either eye;
- Presence of ocular or periocular infection history in either eye within 2 weeks prior to selection;
- History or presence of corneal dystrophy in the study eye;
- History of HIV or hepatitis A, B, or C infection;
- Previous treatment with any gene therapy or cell therapy (e.g., stem cell transplantation);
- Additional intraocular surgery in study eye 3 months prior to baseline visit;
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- Participation in an oral therapeutic STGD clinical trial within 3 months (or within 5 half-lives after last dose) prior to Screening
- Any concomitant treatment that, in the opinion of the investigator, might interfere with the surgical procedure or healing process of the eye
- Any other conditions that would not allow the potential subject to complete follow-up examinations during the study and would, in the opinion of the investigator, make the potential subject unsuitable for the study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Eye & ENT Hospital of Fudan University
Shanghai
Gezhi Xu, +86 18017316316, drxugezhi@163.com
About this study
Official title: An Investigator-initiated, Open-label, Dose-escalation Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of HG005 in Pediatric Patients With Stargardt Disease (STGD1) Caused by Biallelic ABCA4 Mutations
Stargardt disease type 1 (STGD1) is a rare genetic eye condition that causes progressive vision loss, often beginning in childhood or adolescence. It is the most common form of inherited macular degeneration and can lead to legal blindness. STGD1 is caused by mutations in the ABCA4 gene, which normally helps clear waste from the photoreceptor cells in the retina. When ABCA4 gene doesn't function properly, toxic substances like A2E accumulate and damage the retinal pigment epithelium (RPE), leading to vision loss.
There are currently no approved treatments for STGD1. HG005 is an investigational gene therapy designed to deliver a healthy copy of the ABCA4 gene to the retina. Because the gene is too large to fit into a single AAV (adeno-associated virus) vector, HG005 used two AAV vectors that work together in retinal cells to produce the full-length, functional ABCA4 protein. The goal of HG005 is to restore normal waste removal, protect retinal cells from further damage, and slow or stop vision loss.
- Conditions
- Stargardt Disease Type 1 (STGD1)
- Being tested
- HG005
- Keywords
- Stargardt disease type 1 (STGD1), Stargardt Macular Dystrophy, Stargardt Macular Degeneration, HG005, AAV, Gene Therapy
- Medical subject headings
- Stargardt Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HG005Genetic
Once subretinal injection; The duration of the study is about 52 weeks for each subject, including a 4 weeks screening period, enrollment visit, treatment visit, and 56 weeks follow-up period.
| Study group | Role | What they receive |
|---|---|---|
| HG005 | Experimental | The study will enroll up to 2 dose cohorts Receives: Genetic: HG005 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of systemic adverse events
Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)
Measured: 26 & 52 weeks
Secondary outcomes (3)
Change of study eye versus control eye in Low Luminance Visual Acuity(LLVA)
Measured: 52 weeks
Change of study eye versus control eye in Fundus Autofluorescence (FAF)
Measured: 52 weeks
Change of study eye versus control eye in Optical Coherence Tomography (OCT)
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jul 2025
- Study start20 Aug 2025
- Primary completion28 Feb 2027 (estimated)
- Study completion28 Feb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stargardt Disease Type 1 (STGD1)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- HuidaGene Therapeutics Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, HuidaGene Therapeutics Co., Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07063251
- Sponsor's protocol ID
- HG00501
- Acronym
- START
- Status last verified
- Aug 2025
- First submitted
- 3 Jul 2025
- Last updated
- 27 Aug 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Investigator-initiated, Open-label, Dose-escalation Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of HG005 in Pediatric Patients With Stargardt Disease (STGD1) Caused by Biallelic ABCA4 Mutations
- ClinicalTrials.gov ID
- NCT07063251
- Acronym / sponsor ID
- START / HG00501
- Conditions
- Stargardt Disease Type 1 (STGD1)
- Keywords
- Stargardt disease type 1 (STGD1), Stargardt Macular Dystrophy, Stargardt Macular Degeneration, HG005, AAV, Gene Therapy
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- HuidaGene Therapeutics Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 20 Aug 2025 (actual)
- Primary completion
- 28 Feb 2027 (estimated)
- Study completion
- 28 Feb 2028 (estimated)
- First posted
- 14 Jul 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Stargardt disease type 1 (STGD1) is a rare genetic eye condition that causes progressive vision loss, often beginning in childhood or adolescence. It is the most common form of inherited macular degeneration and can lead to legal blindness. STGD1 is caused by mutations in the ABCA4 gene, which normally helps clear waste from the photoreceptor cells in the retina. When ABCA4 gene doesn't function properly, toxic substances like A2E accumulate and damage the retinal pigment epithelium (RPE), leading to vision loss.
There are currently no approved treatments for STGD1. HG005 is an investigational gene therapy designed to deliver a healthy copy of the ABCA4 gene to the retina. Because the gene is too large to fit into a single AAV (adeno-associated virus) vector, HG005 used two AAV vectors that work together in retinal cells to produce the full-length, functional ABCA4 protein. The goal of HG005 is to restore normal waste removal, protect retinal cells from further damage, and slow or stop vision loss.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HG005 | Experimental | Genetic: HG005The study will enroll up to 2 dose cohorts |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Patient ≥ 6 and ≤17 years at the time of signing informed consent, with clinical diagnosis of Stargardt disease;
- At least one ABCA4 allele on each chromosome;
- Both eyes must have well-defined macular atrophic lesions consistent with the diagnosis of Stargardt macular dystrophy.
- Meet visual acuity criteria based on ETDRS letter chart
- Subject must agree to contraception during the study.
Key exclusion criteria (9 listed)
- Presence of active intraocular inflammation or uveitis history in either eye;
- Presence of ocular or periocular infection history in either eye within 2 weeks prior to selection;
- History or presence of corneal dystrophy in the study eye;
- History of HIV or hepatitis A, B, or C infection;
- Previous treatment with any gene therapy or cell therapy (e.g., stem cell transplantation);
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HG005Once subretinal injection; The duration of the study is about 52 weeks for each subject, including a 4 weeks screening period, enrollment visit, treatment visit, and 56 weeks follow-up period. | Genetic | HG005 |
Primary outcome
Incidence and severity of systemic adverse events
Time frame: 26 & 52 weeks
Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)
3 secondary outcomes
Change of study eye versus control eye in Low Luminance Visual Acuity(LLVA)
Time frame: 52 weeks
Change of study eye versus control eye in Fundus Autofluorescence (FAF)
Time frame: 52 weeks
Change of study eye versus control eye in Optical Coherence Tomography (OCT)
Time frame: 52 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Aug 2025.
Related trials for Stargardt Disease Type 1 (STGD1)
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Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)
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A Phase 1, First-in-Human Study of CITY-RBP4 in Healthy Volunteers and STGD1 Participants
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Study Director study director
HuidaGene Therapeutics Co., Ltd.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing HG005 in people with Stargardt Disease Type 1 (STGD1), run by HuidaGene Therapeutics Co., Ltd. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 27 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Study Director (+86 021-25076143, HG00501@huidagene.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of systemic adverse events (time frame: 26 & 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Aug 2025.