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Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells

NCT06041620 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2023-10-17

No results posted yet for this study

Summary

This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.

Conditions

  • Thalassemia, Beta
  • Thalassemia Major

Interventions

BIOLOGICAL

VGB-Ex01

CRISPR-Cas12b editing hematopoietic stem cells

Sponsors & Collaborators

  • Shanghai Vitalgen BioPharma Co., Ltd.

    collaborator INDUSTRY

  • Institute of Hematology & Blood Diseases Hospital, China

    lead OTHER

Principal Investigators

  • Jun Shi, PhD · Institute of Hematology & Blood Diseases Hospital, China

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-08-31
Primary Completion
2025-12-31
Completion
2026-06-30

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06041620 on ClinicalTrials.gov