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

Primary Purpose

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

Status
Not yet recruiting
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
LX101
Sponsored by
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: 1) 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 Exclusion Criteria: 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: 6) 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: 14) Those who do not use effective contraceptive measures; 15) Pregnant and lactating women.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    LX101

    Arm Description

    Outcomes

    Primary Outcome Measures

    Local and systemic safety assessment
    Eye and non-eye adverse events

    Secondary Outcome Measures

    Full Information

    First Posted
    August 29, 2023
    Last Updated
    August 29, 2023
    Sponsor
    Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
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    1. Study Identification

    Unique Protocol Identification Number
    NCT06024057
    Brief Title
    An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy(LX101) in Patients With LCA
    Official Title
    An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy (LX101) in Patients With Leber's Congenital Amaurosis (LCA)
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    August 2023
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    September 1, 2023 (Anticipated)
    Primary Completion Date
    September 30, 2028 (Anticipated)
    Study Completion Date
    September 30, 2038 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No

    5. Study Description

    Brief Summary
    To evaluate the scaling clinical trail of AAV2-RPE65 gene therapy agent (LX101) in patients with congenital amaurosis (LCA).
    Detailed Description
    This study evaluated the overall safety and initial efficacy of RPE65 mutant congenital amaurosis (RPE65-LCA) in subjects treated with a single, subretinal injection of LX101 (containing recombinant human adeno-associated virus serotype 2, AAV2-RPE65) in the second eye 1 year after treatment.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Not Applicable
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    3 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    LX101
    Arm Type
    Experimental
    Intervention Type
    Drug
    Intervention Name(s)
    LX101
    Intervention Description
    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
    Primary Outcome Measure Information:
    Title
    Local and systemic safety assessment
    Description
    Eye and non-eye adverse events
    Time Frame
    52(+4)weeks

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: 1) 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 Exclusion Criteria: 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: 6) 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: 14) Those who do not use effective contraceptive measures; 15) Pregnant and lactating women.

    12. IPD Sharing Statement

    Plan to Share IPD
    No

    Learn more about this trial

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

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