Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients with Bietti Crystalline Dystrophy

Last updated: November 19, 2024
Sponsor: Shanghai Vitalgen BioPharma Co., Ltd.
Overall Status: Active - Not Recruiting

Phase

3

Condition

Retinitis Pigmentosa

Retina

Treatment

VGR-R01

Clinical Study ID

NCT06699108
VGR-R01-301
  • Ages 18-69
  • All Genders

Study Summary

This is a phase 3 study to evaluate the efficacy and safety of VGR-R01 in subjects with Bietti Crystalline Dystrophy.

This is a multicenter, randomized controlled study which will enroll 45 subjects.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  1. Able to provide informed consent and comply with requirements of the study;

  2. ≥18 years and <70 years of age;

  3. Confirmed diagnosis of Bietti Crystalline Dystrophy and molecular diagnosis ofCYP4V2 mutations (homozygotes or compound heterozygotes);

  4. Hand Motion ≤ BCVA ≤ 60 ETDRS letters in the study eye;

Exclusion

Key Exclusion Criteria:

  1. Have insufficient viable retinal photoreceptor cells based on investigator'sdecision;

  2. Have current ocular or periocular infections, or endophthalmitis;

  3. Have any significant ocular disease/disorder other than BCD, including age-relatedmacular degeneration, diabetic retinopathy, optic neuropathy, significant lensopacity, glaucoma, uveitis, retinal detachment, etc;

  4. Have intraocular surgery history except cataract surgery in the study eye;

  5. Have or potentially require of systemic medications that may cause eye injure;

  6. Have contraindications for corticosteroids or immunosuppressant;

  7. Unwilling or unable to have the planned follow-up;

  8. Abnormal coagulation function or other clinically significant abnormal laboratoryresults;

  9. Have malignancies or history of malignancies;

  10. History of immunodeficiency (acquired or congenital); Other protocol definedInclusion/Exclusion criteria may apply.

Study Design

Total Participants: 45
Treatment Group(s): 1
Primary Treatment: VGR-R01
Phase: 3
Study Start date:
December 01, 2024
Estimated Completion Date:
June 30, 2027

Study Description

VGR-R01 is a novel AAV vector carrying the human CYP4v2 coding sequence. This study is intended to assess the efficacy of VGR-R01 in subjects with Bietti crystalline dystrophy (BCD) based on the change from baseline in best corrected visual acuity (BCVA) of the study eyes.

30 subjects will be enrolled in the intervention group and will receive monocular administration of VGR-R01, and 15 subjects in the control group will not receive any intervention. After completion of the trial, the untreated eyes will receive administration of VGR-R01 in subsequent clinical trial.

Connect with a study center

  • Shanghai Vitalgen Biopharma Co.,Ltd.

    Shanghai, Shanghai
    China

    Site Not Available

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