Clinical trial NCT05991336: Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Official title: Growth and Development, Health-related Quality of Life of Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Transfusion-dependent Beta-Thalassemia
Gene Therapy
Keywords: Transfusion-dependent Beta-Thalassemia
gene therapy
cohort study
case-control
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
First posted: 2023-08-14
Enrollment: 100
Sex: ALL
Minimum age (years): 3
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants are divided into three groups: TDT children who have received gene therapy, TDT children who have received lifelong supportive therapy, and healthy children.

Brief 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 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

Primary outcomes:
measure: Growth in pediatric subjects will be assessed by height.
timeFrame: Up to 5 years.
measure: Growth in pediatric subjects will be assessed by weight.
timeFrame: Up to 5 years.
measure: Growth in pediatric subjects will be assessed by the head and chest circumference.
timeFrame: Up to 5 years.
measure: Growth in pediatric subjects will be assessed by interocular distance.
timeFrame: Up to 5 years.
measure: Growth in pediatric subjects will be assessed by bone density.
timeFrame: Up to 5 years.
measure: The pubertal status will be measured using by Tanner scale.
timeFrame: Up to 5 years.
measure: Hormonal levels will be measured by clinical examination.
timeFrame: Up to 5 years.
measure: Quality of life will be measured using by Pediatric Quality of Life Inventory (PedsQL) at baseline and the end of the study.
timeFrame: Up to 5 years.
measure: Lifestyle behaviors will be assessed by general questioning.
timeFrame: Up to 5 years.
Secondary outcomes:
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Regenerative Medicine Center
state: Tianjin
Study officials:
References: