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Gene Therapy

Gene Therapy for Leber Congenital Amaurosis (STAR Trial)

Phase 1 & 2

Recruiting

Research Sponsored by HuidaGene Therapeutics Co., Ltd.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male or females between 6 and 50 years of age at the time of signing the informed consent form

Ability to perform tests of visual and retinal function

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 52 weeks

Awards & highlights

STAR Trial Summary

This trial tests if a gene therapy is safe and effective for a rare eye disease caused by a gene mutation.

Who is the study for?

This trial is for individuals aged 6-50 with Leber Congenital Amaurosis due to RPE65 mutations, who can perform visual tests and have a visual acuity of ≤ 20/80 or field less than 20 degrees. They must have normal blood, chemistry, and urine lab results. Those with certain pre-existing eye conditions, recent eye surgery, prior gene therapy, or diseases affecting ocular function cannot participate.Check my eligibility

What is being tested?

The STAR trial is testing the safety and effectiveness of HG004 as a gene therapy for treating blindness caused by specific genetic mutations in patients with Leber Congenital Amaurosis. The focus is on whether this treatment can improve vision in affected individuals.See study design

What are the potential side effects?

While the side effects are not explicitly listed here, gene therapies like HG004 may cause immune reactions, changes at the injection site (like swelling or redness), potential damage to nearby cells or tissues from surgical procedures involved in delivery.

STAR Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 6 and 50 years old.

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I can perform tests for eye and vision health.

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I have been diagnosed with LCA due to RPE65 mutations.

STAR Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 52 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~52 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Incidence of ocular and non-ocular adverse events

Secondary outcome measures

Change from baseline in Best Corrected Visual Acuity (BCVA) of letters based on the Early Treatment Diabetic Retionpathy Study (ETDRS) chart

Change from baseline in visual fields of full-field stimulus threshold test in log cd.s/m2.

STAR Trial Design

1Treatment groups

Experimental Treatment

Group I: HG004Experimental Treatment1 Intervention

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

HuidaGene Therapeutics Co., Ltd.Lead Sponsor

4 Previous Clinical Trials

36 Total Patients Enrolled

2 Trials studying Leber Congenital Amaurosis

18 Patients Enrolled for Leber Congenital Amaurosis

Cholgene Therapeutics, Inc.UNKNOWN

Study DirectorStudy DirectorHuidaGene Therapeutics Co., Ltd.

1,211 Previous Clinical Trials

489,252 Total Patients Enrolled

Media Library

HG004 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05906953 — Phase 1 & 2

Leber Congenital Amaurosis Research Study Groups: HG004

Leber Congenital Amaurosis Clinical Trial 2023: HG004 Highlights & Side Effects. Trial Name: NCT05906953 — Phase 1 & 2

HG004 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05906953 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the eligibility criterion for this experiment limited to adults?

"This experiment has determined that its target demographic is between 6 and 50 years old. Furthermore, there are 9 studies for minors and 23 trials for elderly patients over 65."

Answered by AI

Are there any vacancies for individuals to join this investigation?

"Per the information presented on clinicaltrials.gov, recruitment for this medical study has been concluded since its posting date of September 1st 2023 and last edit on August 20th 2023. However, there are currently 25 other trials that are actively enrolling participants."

Answered by AI

Is it feasible for me to partake in this experiment?

"This medical study seeks to enrol 20 individuals between the ages of 6 and 50 suffering from leber congenital amaurosis. Applicants must meet various criteria, including being male or female as evidenced by an informed consent form, holding a molecular diagnosis due to RPE65 mutations, having visual acuity not exceeding 20/80 or a field of vision under 20 degrees in the eye that will be injected, displaying acceptable hematology and clinical chemistry results, along with passing laboratory urine tests."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)

2023. "Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05906953.