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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

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Enrollment

100 participants

Start Date

Jun 5, 2023

Study Type

OBSERVATIONAL

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.

Eligibility

Min Age: 3 YearsMax Age: 14 Years

Inclusion Criteria14

  • 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 Criteria10

  • 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

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Interventions

GENETICGene therapy

Autologous edited hematopoietic stem cell transplantation

Locations(1)

Regenerative Medicine Center

Tianjin, Tianjin Municipality, China

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NCT05991336