Safety and Efficacy of CRISPR/Cas9 mRNA Instantaneous Gene Editing Therapy to Treat Refractory Viral Keratitis
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|ClinicalTrials.gov Identifier: NCT04560790|
Recruitment Status : Completed
First Posted : September 23, 2020
Last Update Posted : August 24, 2022
|Condition or disease||Intervention/treatment||Phase|
|Viral Keratitis Blindness Eye Herpes Simplex Virus Infection Cornea||Drug: BD111 Adult single group Dose||Not Applicable|
This is an open-label, single ascending dose study of BD111 in adult (ages 18 to 70) participants with refractory herpetic viral keratitis. Approximately 6 participants will be enrolled.BD111 is a novel gene editing product designed to clear Herpes simplex virus type I (HSV-1) that results in herpetic stromal keratitis in both acute and recurrent infection models which is the leading factor for infectious blindness.
The follow-up period was 360 days, and the patients will be followed up 3±1 days, 7±2 days, 30±7 days, 90±14 days, 180±21 days, and 360+31 days after treatment.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||3 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Single group target value: Since there is no similar products approved on the market and there is no similar comparative treatment methods, the single group target value method is adopted|
|Masking:||None (Open Label)|
|Official Title:||CRISPR/Cas9 mRNA Instantaneous Gene Editing Therapy Assisted Corneal Transplantation in the Treatment of Refractory Viral Keratitis|
|Actual Study Start Date :||November 4, 2020|
|Actual Primary Completion Date :||July 5, 2022|
|Actual Study Completion Date :||July 5, 2022|
Experimental: BD111 Adults single group Dose
Administered by corneal injection surgery. Dosage form:injection solution. Dose:200uL. Frequency of administration: one time injection.
Drug: BD111 Adult single group Dose
3-6 Participants will receive a single group dose administered via corneal injection in the study eye.
- Effective clearance of HSV-1 genome [ Time Frame: 12 months ]Judge HSV-1 genome clearance effective according to DNA sequencing results by methods of Plaque assay,Elisa,PCR etc.
- Rate of reblindness in 3 participants with Refractory HSV Keratitis [ Time Frame: 12 months ]180 days after corneal surgical, calculate rate of reblindness of treated eye in 3 participants.
- HSV-1 virus testing outcome of the intervention eye [ Time Frame: 12 months ]Herpes virus content before and after treatment were determinated by methods of plaque assay, ELISA, PCR etc. Compare the viral content changes with baseline.
- Corneal graft survival time [ Time Frame: 12 months ]Observe the survival time of grafted cornea in participants, whether the grafted cornea is transparency or opacity.
- Visual improvement compared with baseline [ Time Frame: 12 months ]Judge the visual recovery progress according to visual examination results on day 3±1，7±2，30±7，90±14，180±21，360±31.
- Concentration of dose limiting toxicities [ Time Frame: 12 months ]Observe and record AE,SAE incidence of dose limiting toxicities related with BD111 administration.
- Concentration of maximum tolerated dose [ Time Frame: 12 months ]Observe and record AE,SAE at maximum tolerated dose when occurs dose limiting toxicities.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04560790
|Eye & Ent Hospital of Fudan University|
|Shanghai, Shanghai, China, 200000|
|Principal Investigator:||Yujia Cai, PhD||Shanghai BDgene Co., Ltd.|