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

Recruitingn/aAges 3 Years - 14 YearsAllInstitute of Hematology & Blood Diseases Hospital…
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In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

📋 Eligibility rules (as published)
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 matched case * Children with β-TDT * No history of gene therapy or allogeneic hematopoietic stem cell transplantation * Subjects who are willing and able to provide written informed consent. Healthy children group-inclusion * Gender same as the matched case * Age similar to the matched case * Subjects who are willing and able to provide written informed consent. Exclusion Criteria: * 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 * Patients considered to be ineligible for the study by the investigator for reasons other than the above Discontinuation of Study : * Subjects who are unwilling or unable to continue participating in the study (withdrawal of informed consent) may withdraw from the study * Subjects who received gene therapy or allogeneic hematopoietic stem cell transplantation during the study * The subject is seriously non-compliant with the study requirements, such as missing 2 consecutive visits * Subject lost to follow-up
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What it tests

  • Gene therapy
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Where

1 site
Regenerative Medicine Center
Tianjin, China
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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