Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Gene therapy.
- Who it may be relevant to
- Registry conditions: Transfusion-dependent Beta-Thalassemia, Gene Therapy. Basic parameters: 3 years — 14 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Growth and Development, Health-related Quality of Life of Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Overview
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.
Interventions
- Genetic Gene therapy
Autologous edited hematopoietic stem cell transplantation
Primary outcome measures
- Growth in pediatric subjects will be assessed by height. [Time frame: Up to 5 years.]
- Growth in pediatric subjects will be assessed by weight. [Time frame: Up to 5 years.]
- Growth in pediatric subjects will be assessed by the head and chest circumference. [Time frame: Up to 5 years.]
- Growth in pediatric subjects will be assessed by interocular distance. [Time frame: Up to 5 years.]
- Growth in pediatric subjects will be assessed by bone density. [Time frame: Up to 5 years.]
- The pubertal status will be measured using by Tanner scale. [Time frame: Up to 5 years.]
- Hormonal levels will be measured by clinical examination. [Time frame: Up to 5 years.]
- Quality of life will be measured using by Pediatric Quality of Life Inventory (PedsQL) at baseline and the end of the study. [Time frame: Up to 5 years.]
- Lifestyle behaviors will be assessed by general questioning. [Time frame: Up to 5 years.]
Eligibility 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: Yes
Study design
- Observational model
- Cohort
Study locations
China · 1 center
- Regenerative Medicine Center — Tianjin
Identifiers
NCT: NCT05991336 · GDH-001