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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06064565
Other study ID # HG00401
Secondary ID
Status Recruiting
Phase Early Phase 1
First received
Last updated
Start date January 10, 2023
Est. completion date December 30, 2024

Study information

Verified date September 2023
Source HuidaGene Therapeutics Co., Ltd.
Contact Director Study
Phone +86 021-25076143
Email HG00401@huidagene.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical 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 mutations in RPE65 gene.


Recruitment information / eligibility

Status Recruiting
Enrollment 9
Est. completion date December 30, 2024
Est. primary completion date January 30, 2024
Accepts healthy volunteers No
Gender All
Age group 8 Years to 50 Years
Eligibility Inclusion Criteria: - Male or females between 8 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 = 20/160 or visual field less than 20 degrees in the eye to be injected. - Acceptable hematology, clinical chemistry, and urine laboratory parameters. Exclusion Criteria: - OCT examination determined that the outer nuclear layer was not visible in the planned injection area (Bleb) in the study eye. - 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 other condition that would not allow the potential subject to complete follow-up examinations during the study and would, in the opinion of the investigator, make the potential subject unsuitable for the study.

Study Design


Related Conditions & MeSH terms


Intervention

Genetic:
HG004
Method of Administration: Once unilateral subretinal injection; The duration of the study is about 60 weeks for each subject including a 8-week screening period, enrollment/baseline visit, treatment visit, and 52 weeks follow-up period.

Locations

Country Name City State
China Xinhua Hospital affiliated with Shanghai Jiao Tong University School of Medicine Shanghai Shanghai

Sponsors (2)

Lead Sponsor Collaborator
HuidaGene Therapeutics Co., Ltd. Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Country where clinical trial is conducted

China, 

Outcome

Type Measure Description Time frame Safety issue
Primary Incidence and severity of ocular and systemic adverse events Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs) Time Frame: 26 weeks
See also
  Status Clinical Trial Phase
Completed NCT01496040 - Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 Phase 1/Phase 2
Completed NCT02970266 - Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families. N/A
Completed NCT00516477 - Safety Study in Subjects With Leber Congenital Amaurosis Phase 1
Active, not recruiting NCT03920007 - Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D Phase 1/Phase 2
Recruiting NCT05906953 - Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR) Phase 1/Phase 2
Active, not recruiting NCT00999609 - Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis Phase 3
Active, not recruiting NCT01208389 - Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 Phase 1/Phase 2
Terminated NCT03913130 - Extension Study to Study PQ-110-001 (NCT03140969) Phase 1/Phase 2
Recruiting NCT04855045 - An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene. Phase 2/Phase 3
Recruiting NCT02435940 - Inherited Retinal Degenerative Disease Registry
Completed NCT00749957 - Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis Phase 1/Phase 2
Recruiting NCT01793168 - Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Not yet recruiting NCT04731883 - RPE65 Gene Therapy (LX101)for Leber's Congenital Amaurosis N/A
Recruiting NCT05203939 - Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis Phase 1/Phase 2
Recruiting NCT06088992 - Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT) Early Phase 1
Completed NCT00821340 - Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations Phase 1
Active, not recruiting NCT03913143 - A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) Phase 2/Phase 3
Completed NCT02781480 - Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) Phase 1/Phase 2
Completed NCT02714816 - Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65