Comparing growth and development in children with beta-thalassemia after gene therapy
Growth and Development, Health-related Quality of Life of Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
This study looks at how gene therapy affects the growth and development of children with beta-thalassemia compared to those getting regular treatments and healthy kids.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 100 (estimated) |
| Ages | 3 Years to 14 Years |
| Sex | All |
| Sponsor | Institute of Hematology & Blood Diseases Hospital, China Academic / other |
| Locations | 1 site (Tianjin, Tianjin Municipality) |
| Trial ID | NCT05991336 on ClinicalTrials.gov |
What this trial studies
This observational cohort study aims to evaluate the growth, development, metabolism, lifestyle behaviors, and health-related quality of life in children aged 3-14 years with transfusion-dependent beta-thalassemia (TDT). The study will compare three groups: children who have received gene therapy, those receiving lifelong supportive therapy, and healthy children. Participants will be required to provide informed consent, and the study will focus on understanding the long-term outcomes of gene therapy in this population.
Who should consider this trial
Good fit: Ideal candidates include children aged 3-14 years with transfusion-dependent beta-thalassemia who have received gene therapy or are undergoing supportive therapy.
Not a fit: Patients with compound alpha-thalassemia, uncontrolled infections, or a history of malignancies may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide insights into the effectiveness of gene therapy in improving the quality of life and health outcomes for children with transfusion-dependent beta-thalassemia.
How similar studies have performed: While gene therapy for beta-thalassemia is a novel approach, similar studies have shown promising results in improving patient outcomes.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 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 Criteria: * 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 this trial is running
Tianjin, Tianjin Municipality
- Regenerative Medicine Center — Tianjin, Tianjin Municipality, China (Recruiting)
Study contacts
- Study coordinator: Jun Shi, PhD
- Email: shijun@ihcams.ac.cn
- Phone: 2223900913
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.