Clincosm LogoSign in Get a demo

The Gene Replacement Therapy by rAAV2/8-hCYP4V2

Sponsor
Beijing Tongren Hospital (Other)
Overall Status
Recruiting
CT.gov ID
NCT04722107
Collaborator
(none)
3
Enrollment
1
Location
1
Arm
32.3
Anticipated Duration (Months)
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Primary Objectives: To evaluate the safety of The Gene Replacement Therapy by rAAV2/8-hCYP4V2 Following a Single Subretinal Administration in The Patients of Bietti's Crystalline Dystrophy (BCD).

Secondary Objectives: To explore the efficacy of The Gene Replacement Therapy by rAAV2/8-hCYP4V2.

Condition or Disease Intervention/Treatment Phase
  • Drug: Gene Replacement Therapy by rAAV2/8-hCYP4V2
 Early Phase 1

Detailed Description

A Safety Study of The Gene Replacement Therapy by rAAV2/8-hCYP4V2 Following a Single Subretinal Administration in The Patients of Bietti's Crystalline Dystrophy (BCD)

Study Design

Study Type:
Interventional
Anticipated Enrollment :
3 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Safety Study of The Gene Replacement Therapy by rAAV2/8-hCYP4V2 Following a Single Subretinal Administration in The Patients of Bietti's Crystalline Dystrophy (BCD)
Actual Study Start Date :
Apr 21, 2021
Anticipated Primary Completion Date :
Jun 30, 2023
Anticipated Study Completion Date :
Dec 30, 2023

Arms and Interventions

Arm Intervention/Treatment
Experimental: Single arm

Single arm

Drug: Gene Replacement Therapy by rAAV2/8-hCYP4V2
Gene Replacement Therapy by rAAV2/8-hCYP4V2

Outcome Measures

Primary Outcome Measures

  1. the safety of The Gene Replacement Therapy by rAAV2/8-hCYP4V2 [In15 months]

    Incidence of AE

Secondary Outcome Measures

  1. the efficacy of The Gene Replacement Therapy by rAAV2/8-hCYP4V2. [In15 months]

    Changes of functional indexes, behavioral indexes and morphological indexes

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
    1. Are 18 years and older;
    1. Are diagnosed of Bietti's crystalline dystrophy (BCD);
    1. Molecular diagnosis confirmed due to CYP4V2 mutations (two pathogenic mutation);
    1. Are met with the target eye inclusion criterion;
    1. Agree to take effective contraceptive measures from the beginning of the study to 1 year after the administration;
    1. Are voluntarily participated in the study and signed the informed consent form.
Exclusion Criteria:
    1. Have insufficient viable retinal photoreceptor cells. Retinal photoreceptor cells are less than 1D , or areas of retina with thickness measurements less than 100 µm；
    1. Existing or pre-existing of choroidal neovascular (CNV) lesions that were secondary to BCD, or other eye conditions interfering with the surgery or the interpretation of the clinical endpoint, in the investigators' opinion;
    1. Prior use of medicines which may affect the experimental observation within the 6 months before screening (e.g. Lucentis, Avastin Eylea, Conbercept, Triamcinolone acetonide, Corticosteroids)；
    1. Prior intraocular surgery in the target eye (e.g. PDT, pars plana vitrectomy, periocular vascular bypass surgery), or requirement of intraocular surgery during the clinical study (e.g. cataract surgery, laser therapy of retina);
    1. Use of or potentially require of systemic medications that may cause eye damage (e.g. psoralen, risselinic acid, tamoxifen)；
    1. Known hypersensitivity to any ingredient of clinical trial medicine, or allergies (with an allergy history to two or more kinds of medicines or foods);
    1. Abnormal physical examination, vital signs, laboratory examination (e.g. blood routine, urine routine, blood biochemistry, coagulation function, immunologic test, pregnacy test), or other related indicators which have a clinical significance considered by the investigators；
    1. Have any medical conditions or medical history which may have an effect on the safety or the intracorporal process of drugs, especially medical history of angiocarpy, liver, kidney, internal secretion, digestive tract, lung, nerve, blood, tumor, immune or metabolic disorders, which have a clinical significance considered by the investigators；
    1. Participation in any medicine or medical device clinical trials within 3 months prior to enrollment;
    1. Neutralizing antibodies to rAAV> 1:1000 by immunologic test;
    1. For females in pregnancy or lactation period;
    1. Any other conditions which leads the investigator to determine the participant is unsuitable for this study.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Beijing Tongren Hospital Beijing Beijing China 100730

Sponsors and Collaborators

  • Beijing Tongren Hospital

Investigators

  • Principal Investigator: wenbin Wei, Doctor, Vice President of Beijing Tongren Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Beijing Tongren Hospital
ClinicalTrials.gov Identifier:
NCT04722107
Other Study ID Numbers:
  • ZVS101e-01
First Posted:
Jan 25, 2021
Last Update Posted:
May 10, 2021
Last Verified:
Jan 1, 2021
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Product Manufactured in and Exported from the U.S.:
No
Additional relevant MeSH terms:
Corneal Dystrophies, Hereditary
Retinal Diseases

Study Results

No Results Posted as of May 10, 2021