Health TrialsbyHealthPanel

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

ConditionsLeber Congenital AmaurosisInherited Retinal Diseases Caused by RPE65 Mutations
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age6-50
SponsorHuidaGene Therapeutics Co., Ltd.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

  • HG004

    Drug

    Low dose Medium dose High dose

Treatment groups

20 Participants
are divided into 1 treatment group
Group A: HG004Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of ocular and non-ocular adverse events

Time frame
52 weeks

Secondary outcomes

1

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
2

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

Time frame
52 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

HuidaGene Therapeutics Co., Ltd.

Lead sponsor

Source ClinicalTrials.gov
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