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Tests treatment safety and results for Leber Congenital Amaurosis
Official title Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
ClinicalTrials.gov ID: NCT05906953
What this study is testing
What is HG004?
HG004 is an investigational medicine, being studied as a potential treatment for leber congenital amaurosis.
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
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 to 50
You may be able to join if
- 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.
You likely can't join if
- Pre-existing eye conditions that would preclude the planned surgery or interfere with interpretation of study endpoints or complications of surgery...
- 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.
See the full eligibility criteria
- 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.
- 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...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Sacramento, California, United States
- Houston, Texas, United States
- Shanghai, Shanghai Municipality, China
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 - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 years to 50 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Sacramento, California, United States; Houston, Texas, United States; Shanghai, Shanghai Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.