An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy(LX101) in Patients With LCA

NCT06024057 | Not Yet Recruiting

• 1 other identifier: SHGH-LX101-EX
• Study Type: interventional
• Target: 3
• Locations: 0 countries
• Sites: N/A

Brief Summary

To evaluate the scaling clinical trail of AAV2-RPE65 gene therapy agent (LX101) in patients with congenital amaurosis (LCA).

Conditions

To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)

Outcome Measures

Primary Outcomes (1)

• Local and systemic safety assessment

  Eye and non-eye adverse events

  52（+4）weeks

Study Arms (1)

LX101

EXPERIMENTAL

Drug: LX101

Interventions

LX101 DRUG

LX101 (Containing recombinant human adeno-associated virus serotype 2, AAV2-RPE65 encoding human RPE65 gene).Specification: 0.2mL/bottle.Route of administration: subretinal injection, injection volume 0.3 mL/ eye

LX101

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \) Participants in the main study "Exploratory clinical study evaluating AAV2-RPE65 gene therapy formulation (LX101) in patients with congenital amaurosis (LCA) 2) Willingness to participate in the extended study, subject and/or their guardian signing a written informed consent and complying with a long-term follow-up protocol

You may not qualify if:

• Have any of the following eye conditions:
• Subjects with SAE in the "Exploratory clinical Study Evaluating AAV2-RPE65 gene therapy agent (LX101) in patients with congenital Amaurosis (LCA)
• pre-existing eye conditions (such as glaucoma, cornea, or severe lens opacity) that the investigator determines may interfere with planned surgery or with interpretation of the study endpoint;
• Screening subjects who had undergone any internal eye surgery in the six months prior to the visit;
• Inability or unwillingness to meet the requirements of the study;
• Have any of the following systemic conditions:
• \) Known allergy to the drug planned for use in the study; 7) Have a history of surgical operations within 1 month before enrollment, and/or present unhealed wounds, ulcers, fractures, etc.; Present infectious disease requiring oral, intramuscular or intravenous administration; 8) Baseline laboratory values for complex systemic diseases or clinically significant abnormalities; 9) Complex systemic diseases include diseases in which the disease itself or the treatment of the disease can alter eye function. For example, the treatment of malignant tumors can affect central nervous system function (e.g., orbital radiation therapy; Leukemia with central/optic nerve involvement); 10) Subjects with a history of immune-damaging diseases due to the possibility of opportunistic infection (e.g., CMV retinitis); Subjects with diabetes or sickle cell disease will be excluded if they have any manifestations of advanced retinopathy, such as macular edema or proliferative changes. Subjects with juvenile rheumatoid arthritis have an increased risk of infection due to poor postoperative wound healing; 11) Participants who cannot discontinue Viagra (Sildenafil) or related compounds used to treat erectile dysfunction during the study period; 12) Subjects who cannot discontinue hydroxychloroquine, Chloroquine, meralide, or any related retinal toxic compound during the study; 13) Any other conditions in which the potential subject is not allowed to complete a follow-up examination during the study, and the investigator believes that there are conditions that make the potential subject unfit for the study.
• Fertile subjects with any of the following conditions:
• \) Those who do not use effective contraceptive measures; 15) Pregnant and lactating women.

Sponsors & Collaborators

• Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine: lead

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assessor

Study Record Dates

• First Submitted: August 29, 2023
• First Posted: September 5, 2023
• Study Start: September 1, 2023
• Primary Completion (Estimated): September 30, 2028
• Study Completion (Estimated): September 30, 2038
• Last Updated: September 5, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will not share