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Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy

NCT05991336 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 100

Last updated 2023-08-14

No results posted yet for this study

Summary

The investigate will conduct a cohort study to compare the growth and development, metabolism, lifestyle behavior, and health-related quality of life among three groups: children with transfusion-dependent β-thalassemia (TDT) who have received gene therapy, TDT children with lifelong supportive therapy and healthy children.

Conditions

Interventions

GENETIC

Gene therapy

Autologous edited hematopoietic stem cell transplantation

Sponsors & Collaborators

  • Institute of Hematology & Blood Diseases Hospital, China

    lead OTHER

Eligibility

Min Age
3 Years
Max Age
14 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2023-06-05
Primary Completion
2028-12-31
Completion
2029-12-31

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 NCT05991336 on ClinicalTrials.gov