Leber Congenital AmaurosisPhase 1/2NCT05906953
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
A Phase 1/2 study testing HG004 in people with Leber Congenital Amaurosis and Inherited Retinal Diseases Caused by RPE65 Mutations, run by HuidaGene Therapeutics Co., Ltd. at 3 sites in 2 countries.
Trial summary
The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene. Read more about this study
- Who is it for?
- Ages 6 to 50 years
- Conditions the study is for
- Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations
- What is being tested?
- HG004
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 31 Oct 2023to Dec 2025
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or females between 6 and 50 years of age at the time of signing the informed consent form.
- Willing to adhere to protocol as evidenced by written informed consent or parental permission and subject assent.
- Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) and molecular diagnosis of LCA due to RPE65 mutations.
- Ability to perform tests of visual and retinal function.
- Visual acuity of ≤ 20/80 or visual field less than 20 degrees in the eye to be injected.
- Acceptable hematology, clinical chemistry, and urine laboratory parameters.
Who cannot join
- Pre-existing eye conditions that would preclude the planned surgery or interfere with interpretation of study endpoints or complications of surgery (e.g., glaucoma requiring upcoming surgery, corneal or significant lenticular opacities).
- Presence of epiretinal membrane by OCT.
- Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
- Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter ocular function.
- Prior ocular surgery within six months.
- Prior gene therapy or oligonucleotide therapy treatments.
- Any condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
United States2 sites, 2 recruiting
- Recruiting
Research Site
Houston, Texas, 77707
Study Director, HG00402@huidagene.com
- Recruiting
Research Site
Sacramento, California, 95817
Study Director, HG00402@huidagene.com
China1 site, 1 recruiting
- Recruiting
Research Site
Shanghai, Shanghai Municipality
Study Director, HG00402@huidagene.com
About this study
Official title: A Phase 1/2, Open-label, Multi-national, Multiple-cohort, Dose-escalation Study to Evaluate the Safety, Tolerability, and Efficacy of HG004 Gene Therapy in Subjects with RPE65-associated Leber Congenital Amaurosis Type 2 (LCA2)
The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene.
- Being tested
- HG004
- Keywords
- IRD, LCA, LCA2
- Medical subject headings
- Leber Congenital Amaurosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HG004Drug
Low dose Medium dose High dose
| Study group | Role | What they receive |
|---|---|---|
| HG004 | Experimental | Not described Receives: Drug: HG004 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of ocular and non-ocular adverse events
Measured: 52 weeks
Secondary outcomes (2)
Change from baseline in Best Corrected Visual Acuity (BCVA) of letters based on the Early Treatment Diabetic Retionpathy Study (ETDRS) chart
Measured: 52 weeks
Change from baseline in visual fields of full-field stimulus threshold test in log cd.s/m2.
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 posted18 Jun 2023
- Study start31 Oct 2023
- Primary completionDec 2025 (estimated)
- Study completionDec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leber Congenital AmaurosisStudies 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
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 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
- NCT05906953
- Sponsor's protocol ID
- HG00402
- Status last verified
- Sep 2024
- First submitted
- 9 May 2023
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Open-label, Multi-national, Multiple-cohort, Dose-escalation Study to Evaluate the Safety, Tolerability, and Efficacy of HG004 Gene Therapy in Subjects with RPE65-associated Leber Congenital Amaurosis Type 2 (LCA2)
- ClinicalTrials.gov ID
- NCT05906953
- Acronym / sponsor ID
- HG00402
- Keywords
- IRD, LCA, LCA2
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- HuidaGene Therapeutics Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 31 Oct 2023 (actual)
- Primary completion
- Dec 2025 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 18 Jun 2023 (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.
The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HG004 | Experimental | Drug: HG004 |
Who may qualify
- Ages
- 6 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Male or females between 6 and 50 years of age at the time of signing the informed consent form.
- Willing to adhere to protocol as evidenced by written informed consent or parental permission and subject assent.
- Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) and molecular diagnosis of LCA due to RPE65 mutations.
- Ability to perform tests of visual and retinal function.
- Visual acuity of ≤ 20/80 or visual field less than 20 degrees in the eye to be injected.
Key exclusion criteria (7 listed)
- Pre-existing eye conditions that would preclude the planned surgery or interfere with interpretation of study endpoints or complications of surgery (e.g., glaucoma requiring upcoming surgery, corneal or significant lenticular opacities).
- Presence of epiretinal membrane by OCT.
- Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
- Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter ocular function.
- Prior ocular surgery within six months.
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 |
|---|---|---|
| HG004Low dose Medium dose High dose | Drug | HG004 |
Primary outcome
Incidence of ocular and non-ocular adverse events
Time frame: 52 weeks
2 secondary outcomes
Change from baseline in Best Corrected Visual Acuity (BCVA) of letters based on the Early Treatment Diabetic Retionpathy Study (ETDRS) chart
Time frame: 52 weeks
Change from baseline in visual fields of full-field stimulus threshold test in log cd.s/m2.
Time frame: 52 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Study Director study director
HuidaGene Therapeutics Co., Ltd.
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing HG004 in people with Leber Congenital Amaurosis and Inherited Retinal Diseases Caused by RPE65 Mutations, run by HuidaGene Therapeutics Co., Ltd. at 3 sites in 2 countries.
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 7 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: status not confirmed, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Study Director (732-318-9873, HG00402@huidagene.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of ocular and non-ocular adverse events (time frame: 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 19 Sep 2024.