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

Recruiting

• Conditions: Gene Therapy; Transfusion-dependent Beta-Thalassemia
• Interventions: Genetic: Gene therapy

• Registration Number: NCT05991336
• Lead Sponsor: Institute of Hematology & Blood Diseases Hospital, China

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2023-06-05
• Study First Submitted: 2023-07-27
• Status Verified: 2023-08
• Study First Submitted QC: 2023-08-06
• Last Update Submitted: 2023-08-06
• Study First Posted: 2023-08-14
• Last Update Posted: 2023-08-14
• Primary Completion: 2028-12-31
• Study Completion: 2029-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 100

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Gene therapy group	Gene therapy	TDT Children who have received gene therapy between 3\~14 years old.

Primary Outcome Measures

Name	Time	Method
Growth in pediatric subjects will be assessed by height.	Up to 5 years.	Height in meters.
Growth in pediatric subjects will be assessed by interocular distance.	Up to 5 years.	Interocular distance in centimeters.
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.
Growth in pediatric subjects will be assessed by weight.	Up to 5 years.	Weight in kilograms.
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.
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 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.
Lifestyle behaviors will be assessed by general questioning.	Up to 5 years.

Trial Locations

Locations (1)

Regenerative Medicine Center; 🇨🇳 Tianjin, Tianjin, China