Following growth and development in children with beta-thalassemia after gene therapy
Compares growth, development and quality of life in children with beta-thalassemia after gene therapy.
Plain-language title generated from the registry record
Registry title: Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Key facts
- Where
- Tianjin, ChinaSee the site
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 3 and 14 years old?
Source: NCT05991336, who can take part
Where in China
- Regenerative Medicine CenterTianjin, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Institute of Hematology & Blood Diseases Hospital, China
- Trial code
- NCT05991336
- Record updated
- 4 November 2026
- Original description
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.Read full original descriptionShow less
Growth and Development, Health-related Quality of Life of Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
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.
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: Subjects who received gene therapy or allogeneic hematopoietic stem cell transplantation during the study; Uncontrolled systemic fungal, bacterial, or viral infection; History of malignant solid tumors, myeloproliferative or immunodeficiency diseases; Diagnosed with mental illness; Diagnosis of compound α-thalassemia; 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; The subject is seriously non-compliant with the study requirements, such as missing 2 consecutive visits; Subject lost to follow-up.