Skip to main content
OpenTrials
Recruiting

NCT Number: NCT05991336

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

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Transfusion-dependent Beta-Thalassemia Gene Therapy

Age range

3 year–14 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Gene therapy

Genetic

Autologous edited hematopoietic stem cell transplantation

Primary outcomes

  1. Growth in pediatric subjects will be assessed by height.

    Time frame: Up to 5 years.

    Height in meters.

  2. Growth in pediatric subjects will be assessed by weight.

    Time frame: Up to 5 years.

    Weight in kilograms.

  3. Growth in pediatric subjects will be assessed by the head and chest circumference.

    Time frame: Up to 5 years.

    Head and chest circumference in centimeters.

  4. Growth in pediatric subjects will be assessed by interocular distance.

    Time frame: Up to 5 years.

    Interocular distance in centimeters.

  5. Growth in pediatric subjects will be assessed by bone density.

    Time frame: 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).

  6. The pubertal status will be measured using by Tanner scale.

    Time frame: 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.

  7. Hormonal levels will be measured by clinical examination.

    Time frame: 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.

  8. Quality of life will be measured using by Pediatric Quality of Life Inventory (PedsQL) at baseline and the end of the study.

    Time frame: 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.

  9. Lifestyle behaviors will be assessed by general questioning.

    Time frame: Up to 5 years.

Study contacts

Contact information is provided by the study sponsor or research team.

Jingyu Zhao, MPH

CONTACT

[email protected]

13752253515

Jun Shi, PhD

CONTACT

[email protected]

2223900913

Sponsors and collaborators

Lead sponsor

Institute of Hematology & Blood Diseases Hospital, China

Other

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2028
Study completion
2029
First posted
Aug 14, 2023
Registry last updated
Aug 14, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.