Status:
RECRUITING
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Lead Sponsor:
Institute of Hematology & Blood Diseases Hospital, China
Conditions:
Transfusion-dependent Beta-Thalassemia
Gene Therapy
Eligibility:
All Genders
3-14 years
Brief 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-depe...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
June 5 2023
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 31 2029
Estimated Enrollment :
100 Patients enrolled
Trial Details
Trial ID
NCT05991336
Start Date
June 5 2023
End Date
December 31 2029
Last Update
August 14 2023
Active Locations (1)
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1
Regenerative Medicine Center
Tianjin, Tianjin Municipality, China