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

Recruiting Observational Study
Transfusion-dependent Beta-Thalassemia Gene Therapy
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Healthy Volunteers Welcome You do not need to have the condition being studied to take part. No Study Drug Researchers observe your health over time — no experimental treatment is given.
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At a Glance
Age
3 – 14
Sex
Any
Study type
Observational
Participants needed
100 (estimated)
Sponsor
Institute of Hematology & Blood Diseases Hospital, China · Other
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About This Trial

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.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: Gene therapy group-inclusion * Male or female age of 3-14 years * TDT Children who have received gene therapy. * Subjects who are willing and able to provide written informed consent. Supportive therapy group-inclusion * Gender same as the matched case * Age similar to the ma…
Contacts

Jun Shi, PhD

2223900913

shijun@ihcams.ac.cn

CONTACT

Jingyu Zhao, MPH

13752253515

zhaojingyu@ihcams.ac.cn

CONTACT