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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Review the details below, then apply to join this clinical trial.
At a Glance
Age
3 – 14
Sex
Any
Study type
Observational
Participants needed
100 (estimated)
Sponsor
Institute of Hematology & Blood Diseases Hospital, China · Other
Who this trial is looking for
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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…
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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