Trials / Recruiting
RecruitingNCT05991336
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
- Status
- Recruiting
- Phase
- —
- Study type
- Observational
- Enrollment
- 100 (estimated)
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China · Academic / Other
- Sex
- All
- Age
- 3 Years – 14 Years
- Healthy volunteers
- Accepted
Summary
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
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | Gene therapy | Autologous edited hematopoietic stem cell transplantation |
Timeline
- Start date
- 2023-06-05
- Primary completion
- 2028-12-31
- Completion
- 2029-12-31
- First posted
- 2023-08-14
- Last updated
- 2023-08-14
Locations
1 site across 1 country: China
Source: ClinicalTrials.gov record NCT05991336. Inclusion in this directory is not an endorsement.