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Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector

NCT03351829 · Status: UNKNOWN · Phase: NA · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2017-11-30

No results posted yet for this study

Summary

This is a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s). The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.

Conditions

Interventions

GENETIC

Gene-modified autologous stem cells

1 infusion for 5x10\^6\~1x10\^7 gene-modified cells; or more infusions depending on the circumstances

Sponsors & Collaborators

  • Shenzhen Geno-Immune Medical Institute

    lead OTHER

Principal Investigators

  • Lung-Ji Chang, PhD · Shenzhen Geno-Immune Medical Institute

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-12-01
Primary Completion
2019-01-01
Completion
2020-12-31

Countries

  • China

Study Locations

More Related Trials

Entities

Diseases

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 NCT03351829 on ClinicalTrials.gov