Actively Recruiting
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2023-08-14
100
Participants Needed
1
Research Sites
343 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female children aged 3 to 14 years
- Children with transfusion-dependent beta-thalassemia (TDT) who have received gene therapy
- Children with TDT without history of gene therapy or allogeneic hematopoietic stem cell transplantation, matched by age and gender
- Healthy children matched by age and gender
- Subjects willing and able to provide written informed consent
You will not qualify if you...
- Diagnosis of compound alpha-thalassemia
- Uncontrolled systemic fungal, bacterial, or viral infection
- History of malignant solid tumors, myeloproliferative or immunodeficiency diseases
- Diagnosed with mental illness
- Considered ineligible by the investigator for other reasons
- Subjects unwilling or unable to continue participation
- Subjects receiving gene therapy or allogeneic hematopoietic stem cell transplantation during the study
- Subjects seriously non-compliant with study requirements
- Subjects lost to follow-up
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Regenerative Medicine Center
Tianjin, Tianjin Municipality, China
Actively Recruiting
Research Team
J
Jun Shi, PhD
CONTACT
J
Jingyu Zhao, MPH
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
3
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