Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT06931912

Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia

There are nearly 300,000 patients with severe or intermediate thalassemia in China. Growth retardation is the most significant health issue for children and adolescents with transfusion-dependent thalassemia (TDT), placing a substantial economic burden on their families and a serious social strain on the labor force. Investigating the growth and development of these children and adolescents, and establishing targeted intervention plans, holds significant social value for public health practice.

1. To screen and identify pediatric patients with growth problems by conducting growth and development assessments in high-incidence areas of China, including physical development, endocrine function, nutritional status, brain function and lifestyle behaviors. 2. Implement the MENBS clinical interventions for pediatric patients with growth problems, concentrating on the following areas:

* Monitor: Continuously monitor health-related indicators through regular follow-up. * Education: Provide health education to improve the cognition of patients and their families. * Nutrition: Assess patients' nutritional risks and develop personalized diet plans. * Behavior: Recommend appropriate exercise plans to promote physical development. * Support: Conduct home visits, offer free clinics and establish a support network. 3. Repeat growth assessment for pediatric patients with growth problems after 1-year clinical interventions. 4. Evaluate the effectiveness of MENBS interventions by comparing changes in growth and development indicators.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Notify Me

Key information

Conditions

Thalassemia Intermedia Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Endocrine System Diseases Genetic Diseases, Inborn Gonadal Disorders Growth Delay Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Puberty, Delayed Thalassemia Thalassemia Major beta-Thalassemia

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Regenerative Medicine Center and Red Blood Cell Disorders Center

Tianjin, Tianjin Municipality, China

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Subjects diagnosed with transfusion-dependent thalassemia (TDT)
  • Male or female age ≤19 years
  • Subjects who are willing and able to provide written informed consent

Exclusion criteria

  • Subjects with clinically significant thyroid dysfunction
  • Subjects received allo-HSCT or gene therapy

Treatment and study plan

MENBS clinical interventions

Behavioral
  • Monitor: Continuously monitor health-related indicators through regular follow-up.
  • Education: Provide health education to improve cognition of patients and their families.
  • Nutrition: Assess patients' nutritional risks and develop personalized diet plans.
  • Behavior: Recommend appropriate exercise plans to promote physical development.
  • Support: Conduct home visits, offer free clinics and establish a support network.

Primary outcomes

  1. Bone age

    Time frame: Up to 1 year

    one age was assessed from left hand-wrist radiographs according to the Chinese Hand-Wrist Bone Development Standard (China 05).

  2. Height-for-age (m)

    Time frame: Up to 1 yearUp to 1 year

    It is assessed using the 'Growth Standard for Children under 7 Years of Age' and the 'Standard for Height Level Classification among Children and Adolescents Aged 7-18 Years' issued by the National Health Commission

  3. Body mass index-for-age (BMI-for-age, kg/m^2)

    Time frame: Up to 1 yearUp to 1 year

    It is assessed using the 'Growth Standard for Children under 7 Years of Age' and the 'Dietary Guidelines for Chinese Residents'.

  4. Endocrine function

    Time frame: Up to 1 year

    Endocrine function such as hormonal levels (IGF-1, GH, etc.) will be measured by clinical examination

  5. Nutritional status

    Time frame: Up to 1 year

    Nutritional status will be measured by clinical examination, such as concentration of Vitamin D and Zinc

  6. Intelligence quotient (IQ)

    Time frame: Up to 1 year

    IQ will be measured by scores obtained from Raven's Progressive Matrices. Raw scores are converted into percentile ranks or IQ scores based on age-group norms:

    • ≥95th percentile (IQ ~125+): Very high intelligence.
    • 75th-94th percentile (IQ ~110-124): Above average.
    • 25th-74th percentile (IQ ~90-109): Average range (most common).
    • 5th-24th percentile (IQ ~80-89): Below average.
    • <5th percentile (IQ <80): Potential intellectual disability.

  7. Brain function

    Time frame: Up to 1 year

    Brain function will be measured by Functional Near-infrared Spectroscopy (fNIRS) to record brain activations

Sponsors and collaborators

Lead sponsor

Institute of Hematology & Blood Diseases Hospital, China

Other

Registry information

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Apr 17, 2025
Registry last updated
Jul 24, 2026

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.