Growth and Development, Health-related Quality of Life of Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- Growth in pediatric subjects will be assessed by height.
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 3 Years 至 14 Years（Child）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •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.
排除标准
- •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
结局指标
主要结局
Growth in pediatric subjects will be assessed by height.
时间窗: Up to 5 years.
Height in meters.
Growth in pediatric subjects will be assessed by interocular distance.
时间窗: Up to 5 years.
Interocular distance in centimeters.
Hormonal levels will be measured by clinical examination.
时间窗: 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.
时间窗: 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.
时间窗: 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.
时间窗: Up to 5 years.
Head and chest circumference in centimeters.
Growth in pediatric subjects will be assessed by bone density.
时间窗: 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.
时间窗: 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.
时间窗: Up to 5 years.
次要结局
未报告次要终点
研究者
Jun Shi
Director, Regenerative Medicine Clinic Center
Institute of Hematology & Blood Diseases Hospital, China