Leber Congenital AmaurosisEarly Phase 1NCT06088992
Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)
A Early Phase 1 study testing HG004 in people with Leber Congenital Amaurosis, run by Xinhua Hospital, Shanghai Jiao Tong University School of Medicine at 1 site in China.
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 mutationsin RPE65 gene. Read more about this study
- Who is it for?
- Ages 8 to 50 years
- Conditions the study is for
- Leber Congenital Amaurosis
- What is being tested?
- HG004
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 10 Jan 2023to 30 Oct 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 8 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or females between 8 and 50 years of age at the time of signing theinformed consent form.
- Willing to adhere to protocol as evidenced by written informed consent orparental permission and subject assent.
- Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) andmolecular diagnosis of LCA due to RPE65 mutations.
- Ability to perform tests of visual and retinal function.
- Visual acuity of ≤ 20/160 or visual field less than 20 degrees in the eye to beinjected.
- Acceptable hematology, clinical chemistry, and urine laboratory parameters.
Who cannot join
- OCT examination determined that the outer nuclear layer was not visible inthe planned injection area (Bleb) in the study eye.
- Presence of epiretinal membrane by OCT.
- Complicating systemic diseases or clinically significant abnormal baselinelaboratory values.
- Complicating systemic diseases would include those in which the diseaseitself, or the treatment for the disease, can alter ocular function.
- Prior ocular surgery within six months.
- Prior gene therapy or oligonucleotide therapy treatments.
- Any other condition that would not allow the potential subject to completefollow-up examinations during the study and would, in the opinion of theinvestigator, make the potential subject unsuitable for the study.
Trial locations and contacts
1 site in 1 country.
China1 site
Xinhua Hospital affiliated with Shanghai Jiao Tong UniversitySchool of Medicine
Shanghai, Shanghai Municipality
About this study
Official title: An Investigator-Initiated Open-Label, Multiple-Dose Clinical Study to Evaluate the Safety,Tolerability, and Efficacy of Gene Therapy for 2Leber's Congenital Amaurosis with RPE65 Mutation (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 mutationsin RPE65 gene.
- Conditions
- Leber Congenital Amaurosis
- Being tested
- HG004
- 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.
HG004Genetic
Method of Administration: Once unilateralsubretinal injection; The duration of the study isabout 60 weeks for each subject including a 8-weekscreening period, enrollment/baseline visit,treatment visit, and 52 weeks follow-up period.
| Study group | Role | What they receive |
|---|---|---|
| HG004 | Experimental | Not described Receives: Genetic: HG004 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of ocular and systemic adverse events
Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)
Measured: 26 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2023
- Study start10 Jan 2023
- Primary completion30 Oct 2024 (estimated)
- Study completion30 Oct 2028 (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.
- More trials run by Xinhua Hospital, Shanghai Jiao Tong University School of MedicineThe sponsor's other registered studies.
- 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
- 9 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- HuidaGene Therapeutics Co., Ltd.
- Responsible party
- Principal investigator, Peiquan Zhao, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
- Investigators
- Peiquan Zhao, Principal investigator, Xinhua Hospital affiliated with Shanghai Jiao Tong UniversitySchool of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06088992
- Sponsor's protocol ID
- HG00401
- Status last verified
- Sep 2024
- First submitted
- 12 Oct 2023
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- 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
- An Investigator-Initiated Open-Label, Multiple-Dose Clinical Study to Evaluate the Safety,Tolerability, and Efficacy of Gene Therapy for 2Leber's Congenital Amaurosis with RPE65 Mutation (LCA2)
- ClinicalTrials.gov ID
- NCT06088992
- Acronym / sponsor ID
- HG00401
- Conditions
- Leber Congenital Amaurosis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Collaborators
- HuidaGene Therapeutics Co., Ltd.
- Study start
- 10 Jan 2023 (actual)
- Primary completion
- 30 Oct 2024 (estimated)
- Study completion
- 30 Oct 2028 (estimated)
- First posted
- 18 Oct 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 mutationsin 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 | Genetic: HG004 |
Who may qualify
- Ages
- 8 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Male or females between 8 and 50 years of age at the time of signing theinformed consent form.
- Willing to adhere to protocol as evidenced by written informed consent orparental permission and subject assent.
- Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) andmolecular diagnosis of LCA due to RPE65 mutations.
- Ability to perform tests of visual and retinal function.
- Visual acuity of ≤ 20/160 or visual field less than 20 degrees in the eye to beinjected.
Key exclusion criteria (7 listed)
- OCT examination determined that the outer nuclear layer was not visible inthe planned injection area (Bleb) in the study eye.
- Presence of epiretinal membrane by OCT.
- Complicating systemic diseases or clinically significant abnormal baselinelaboratory values.
- Complicating systemic diseases would include those in which the diseaseitself, 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 |
|---|---|---|
| HG004Method of Administration: Once unilateralsubretinal injection; The duration of the study isabout 60 weeks for each subject including a 8-weekscreening period, enrollment/baseline visit,treatment visit, and 52 weeks follow-up period. | Genetic | HG004 |
Primary outcome
Incidence and severity of ocular and systemic adverse events
Time frame: 26 weeks
Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)
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 19 Sep 2024.
- Individual participant dataSharing planned: No
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Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
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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.
Peiquan Zhao principal investigator
Xinhua Hospital affiliated with Shanghai Jiao Tong UniversitySchool of Medicine
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 HG004 in people with Leber Congenital Amaurosis, run by Xinhua Hospital, Shanghai Jiao Tong University School of Medicine 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 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: under way, not looking for participants, 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 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of ocular and systemic adverse events (time frame: 26 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.