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

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age3-14
SponsorInstitute of Hematology & Blood Diseases Hospital, China

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.

Eligibility criteria

Qualifiers

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.

Gender same as the matched case

Disqualifiers

Diagnosis of compound α-thalassemia

Uncontrolled systemic fungal, bacterial, or viral infection

History of malignant solid tumors, myeloproliferative or immunodeficiency diseases

Diagnosed with mental illness

Trial design

Treatments tested in this trial

  • Gene therapy

Treatment groups

100 Participants
are divided into 3 treatment groups