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
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for children with transfusion-dependent beta-thalassemia who had gene therapy, children with the same disease on lifelong supportive therapy, and healthy children.
- Age
- 3 to 14 years
- Sex
- Boys and girls can join. Comparison children have the same sex as their paired case.
- Disease stage
- Transfusion-dependent beta-thalassemia (TDT).
- Earlier treatment
- Gene therapy group: must have had gene therapy. Supportive therapy group: must not have had gene therapy or a stem cell transplant from a donor.
Main reasons for not taking part
- Compound alpha-thalassemia
- Uncontrolled infection by fungus, bacteria or virus
- History of solid cancer, myeloproliferative disease or immune deficiency disease
- Diagnosed mental illness
- Unable or unwilling to give written informed consent
Read by machine on 12 November 2026.
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, ChinaContact: Jun Shi, PhDTaking 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?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial 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.