NCT05991336Recruiting
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Growth and Development, Health-related Quality of Life of Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Source: ClinicalTrials.gov · Last updated 4 Nov 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 100 (estimated)
- First submitted
- 27 Jul 2023
- Start date
- 5 Jun 2023
- Primary completion date
- 31 Dec 2027
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, China
- Diseases
- Thalassaemia
- NCT number
- NCT05991336
What you commit to
- Study type: Observational
- Planned participants: 100
- From 5 Jun 2023 to the primary completion date, 31 Dec 2027
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Subjects who received gene therapy or allogeneic hematopoietic stem cell transplantation during the study
Other illness
- Uncontrolled systemic fungal, bacterial, or viral infection
- History of malignant solid tumors, myeloproliferative or immunodeficiency diseases
- Diagnosed with mental illness
Other
- 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
Who the study is looking for
- 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.
Age in the registry: 3 years to 14 years · Sex: All · Accepts healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Regenerative Medicine Center Coordinator of this site? Confirm its status | Tianjin | China | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.