History of Changes for Study: NCT05906953
A Safety and Efficacy Study of HG004 in Subjects With Leber Congenital Amaurosis
Latest version (submitted November 9, 2023) on
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Study Record Versions
Version A B Submitted Date Changes
1 June 13, 2023 None (earliest Version on record)
2 August 20, 2023 Contacts/Locations and Study Status
3 September 28, 2023 Conditions, Oversight and Study Status
4 October 26, 2023 Recruitment Status, Study Status, Study Identification and Contacts/Locations
5 October 30, 2023 Study Identification and Study Status
6 November 7, 2023 Study Status and Contacts/Locations
7 November 9, 2023 Study Status and Study Identification
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Study NCT05906953
Submitted Date:  June 13, 2023 (v1)

Open or close this module Study Identification
Unique Protocol ID: HG00402
Brief Title: A Safety and Efficacy Study of HG004 in Subjects With Leber Congenital Amaurosis
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)
Secondary IDs:
Open or close this module Study Status
Record Verification: May 2023
Overall Status: Not yet recruiting
Study Start: September 2023
Primary Completion: December 2025 [Anticipated]
Study Completion: December 2025 [Anticipated]
First Submitted: May 9, 2023
First Submitted that
Met QC Criteria:
June 13, 2023
First Posted: June 18, 2023 [Actual]
Last Update Submitted that
Met QC Criteria:
June 13, 2023
Last Update Posted: June 18, 2023 [Actual]
Open or close this module Sponsor/Collaborators
Sponsor: HuidaGene Therapeutics Co., Ltd.
Responsible Party: Sponsor
Collaborators: Cholgene Therapeutics, Inc.
Open or close this module Oversight
U.S. FDA-regulated Drug: Yes
U.S. FDA-regulated Device: No
Data Monitoring:
Open or close this module Study Description
Brief 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.
Detailed Description:
Open or close this module Conditions
Conditions: Leber Congenital Amaurosis
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 1/Phase 2
Interventional Study Model: Single Group Assignment
Number of Arms: 1
Masking: None (Open Label)
Allocation: N/A
Enrollment: 20 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Experimental: HG004 Drug: HG004
Low dose Medium dose High dose
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Incidence of ocular and non-ocular adverse events
[ Time Frame: 52 weeks ]

Secondary Outcome Measures:
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 ]

Open or close this module Eligibility
Minimum Age: 6 Years
Maximum Age: 50 Years
Sex: All
Gender Based:
Accepts Healthy Volunteers: No

Inclusion 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.

Exclusion Criteria:

  • 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.
Open or close this module Contacts/Locations
Central Contact Person: Study Director
Telephone: +862125076143
Study Officials: Study Director
Study Director
HuidaGene Therapeutics Co., Ltd.
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Available IPD/Information:

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