Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Recruiting now
Conditions studied: Transfusion-dependent Beta-Thalassemia, Gene Therapy
In brief
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.
Key facts
- Study ID
- NCT05991336
- Run by
- Institute of Hematology & Blood Diseases Hospital, China
- People needed
- 100
- Starts
- 2023-06-05
- Expected to finish
- 2029-12-31
- Last updated by the study team
- 2023-08-14
Who can join
Age: 3 and older, up to 14. Sex: any. Healthy volunteers: accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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
Where it is running
- Regenerative Medicine Center — Tianjin, Tianjin Municipality, China (enrolling)
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.