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

Gene Therapy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05991336
• Other study ID #: GDH-001
• Status: Recruiting
• Start date: June 5, 2023
• Est. completion date: December 31, 2029

Study information

• Verified date: August 2023
• Source: Institute of Hematology & Blood Diseases Hospital
• Contact: Jun Shi, PhD
• Phone: 2223900913
• Email: shijun[@]ihcams.ac.cn
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 100
• Est. completion date: December 31, 2029
• Est. primary completion date: December 31, 2028
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 3 Years to 14 Years

Eligibility

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 ß-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 a-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

Related Conditions & MeSH terms

• beta-Thalassemia
• Gene Therapy
• Thalassemia
• Transfusion-dependent Beta-Thalassemia

Intervention

Genetic:
• Gene therapy
Autologous edited hematopoietic stem cell transplantation

Locations

Country	Name	City	State
China	Regenerative Medicine Center	Tianjin	Tianjin

Sponsors (1)

Lead Sponsor	Collaborator
Institute of Hematology & Blood Diseases Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Growth in pediatric subjects will be assessed by height.	Height in meters.	Up to 5 years.
Primary	Growth in pediatric subjects will be assessed by weight.	Weight in kilograms.	Up to 5 years.
Primary	Growth in pediatric subjects will be assessed by the head and chest circumference.	Head and chest circumference in centimeters.	Up to 5 years.
Primary	Growth in pediatric subjects will be assessed by interocular distance.	Interocular distance in centimeters.	Up to 5 years.
Primary	Growth in pediatric subjects will be assessed by bone density.	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).	Up to 5 years.
Primary	The pubertal status will be measured using by Tanner scale.	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.	Up to 5 years.
Primary	Hormonal levels will be measured by clinical examination.	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.	Up to 5 years.
Primary	Quality of life will be measured using by Pediatric Quality of Life Inventory (PedsQL) at baseline and the end of the study.	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.	Up to 5 years.
Primary	Lifestyle behaviors will be assessed by general questioning.		Up to 5 years.