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FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector

NCT03351868 · Status: UNKNOWN · Phase: NA · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2019-09-19

No results posted yet for this study

Summary

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

Conditions

  • Fanconi Anemia

Interventions

GENETIC

Gene-modified autologous stem cells

Infusion for 5x10\^6\~1x10\^7 per kilogram of body weight of gene-modified cells; or more infusions depending on the circumstances

Sponsors & Collaborators

  • Shenzhen Geno-Immune Medical Institute

    lead OTHER

Principal Investigators

  • Lung-Ji Chang, Ph.D · Shenzhen Geno-Immune Medical Institute

  • Xiao-Dong Shi, M.D./Ph. D · Capital Institute of Pediatrics affiliated Children's hospital

  • Jie Zheng, M.D./Ph. D · Beijing Children's Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
20 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-12-01
Primary Completion
2020-12-31
Completion
2021-12-31

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 NCT03351868 on ClinicalTrials.gov