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

Sponsor

Institute of Hematology & Blood Diseases Hospital (Other)

Overall Status

Recruiting

CT.gov ID

NCT05991336

Collaborator

(none)

100

Enrollment

Location

78.9

Anticipated Duration (Months)

1.3

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Transfusion-dependent Beta-Thalassemia Gene Therapy	Genetic: Gene therapy

Study Design

Study Type:

Observational

Anticipated Enrollment :

100 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Growth and Development, Health-related Quality of Life of Children With Transfusion-dependent Beta-thalassemia After Gene Therapy

Actual Study Start Date :

Jun 5, 2023

Anticipated Primary Completion Date :

Dec 31, 2028

Anticipated Study Completion Date :

Dec 31, 2029

Arms and Interventions

Arm	Intervention/Treatment
Gene therapy group TDT Children who have received gene therapy between 3~14 years old.	Genetic: Gene therapy Autologous edited hematopoietic stem cell transplantation
Supportive therapy group 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 Healthy children were selected based on the age and sex of the gene therapy group.

Arm

Intervention/Treatment

Gene therapy group

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

Genetic: Gene therapy

Autologous edited hematopoietic stem cell transplantation

Supportive therapy group

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

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

Outcome Measures

Primary Outcome Measures

Growth in pediatric subjects will be assessed by height. [Up to 5 years.]
Height in meters.
Growth in pediatric subjects will be assessed by weight. [Up to 5 years.]
Weight in kilograms.
Growth in pediatric subjects will be assessed by the head and chest circumference. [Up to 5 years.]
Head and chest circumference in centimeters.
Growth in pediatric subjects will be assessed by interocular distance. [Up to 5 years.]
Interocular distance in centimeters.
Growth in pediatric subjects will be assessed by bone density. [Up to 5 years.]
Bone density measurement is measured by a procedure called densitometry, performed in the radiology medicine departments of hospitals or clinics. The result is the bone mineral density in grams per unit area (g/cm2).
The pubertal status will be measured using by Tanner scale. [Up to 5 years.]
The Tanner scale, a well-known technique used to define pubertal stage, is based on physical measurements of development based on external primary and secondary sex characteristics (from Tanner 1: pre-pubertal to Tanner 5: mature adolescent).It was developed by Marshall and Tanner while conducting a longitudinal study during the 1940s to the 1960s in England. Based on observational data, they developed separate scales for the development of external genitalia: phallus, scrotum, and testes volume in males; breasts in females; and pubic hair in both males and females.
Hormonal levels will be measured by clinical examination. [Up to 5 years.]
Hormonal levels included thyroid-stimulating hormone (TSH), serum triiodothyronine (T3), serum-free triiodothyronine (T4), growth hormone (GH, including IGF-1 and IGF-2), and sex hormone examination. Sex hormone examination is only for the following subjects: estradiol, FSH and LH for girls aged 9-18; testosterone, FSH and LH for boys aged 12-18.
Quality of life will be measured using by Pediatric Quality of Life Inventory (PedsQL) at baseline and the end of the study. [Up to 5 years.]
The Pediatric Quality of Life Inventory (PedsQL) is a valid, standardized, generic, and self-reporting assessment tool to measure health-related quality of life HRQOL for pediatrics and adolescents. PedsQL contain 23-items, and consists of the following: physical functioning, emotional functioning, social functioning, and school functioning. Each item is scored on a 5-point scale, scores are linearly transformed to a 0-100 scale in which high score means better condition.
Lifestyle behaviors will be assessed by general questioning. [Up to 5 years.]

Eligibility Criteria

Criteria

Ages Eligible for Study:

3 Years to 14 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Yes

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