Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy

Institute of Hematology & Blood Diseases Hospital, China

Status: Enrolling

Conditions: Gene Therapy; Transfusion-dependent Beta-Thalassemia

Treatments: Genetic: Gene therapy

Study type: Observational

Funder types: Other

Identifiers

NCT05991336

GDH-001

Details and patient eligibility

About

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.

Enrollment: 100 estimated patients

Sex: All

Ages: 3 to 14 years old

Volunteers: Accepts Healthy Volunteers

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

Trial design

100 participants in 3 patient groups

Gene therapy group

Description:

TDT Children who have received gene therapy between 3\~14 years old.

Treatment:

Genetic: Gene therapy

Supportive therapy group

Description:

TDT Children who received lifelong treatment with blood transfusions and iron chelation. They were selected based on the age and sex of the gene therapy group.

Healthy children group

Description:

Healthy children were selected based on the age and sex of the gene therapy group.