Thalassemia IntermediaNCT06931912
Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia
A study testing MENBS clinical interventions in people with Thalassemia Intermedia and Thalassemia Major, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Thalassemia Intermedia, Thalassemia Major, Growth Delay
- What is being tested?
- MENBS clinical interventions
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 385 participants
- Where
- 1 siteChina
- Study dates
- 1 Dec 2024to 30 Nov 2026
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects diagnosed with transfusion-dependent thalassemia (TDT)
- Male or female age ≤19 years
- Subjects who are willing and able to provide written informed consent
Who cannot join
- Subjects with clinically significant thyroid dysfunction
- Subjects received allo-HSCT or gene therapy
Trial locations and contacts
1 site in 1 country.
China1 site
Regenerative Medicine Center and Red Blood Cell Disorders Center
Tianjin, Tianjin Municipality
About this study
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.
- 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.
- Implement the MENBS clinical interventions for pediatric patients with growth problems, concentrating on the following areas:
Read more
- 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.
- Repeat growth assessment for pediatric patients with growth problems after 1-year clinical interventions.
- Evaluate the effectiveness of MENBS interventions by comparing changes in growth and development indicators.
- Being tested
- MENBS clinical interventions
- Keywords
- Thalassemia, Children and adolescents, Growth development, Health Promotion, Clinical Management
- Medical subject headings
- beta-Thalassemia, Puberty, Delayed, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MENBS clinical interventionsBehavioural
- 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.
| Study group | Role | What they receive |
|---|---|---|
| Pediatric patients with growth problems | Experimental | We identify pediatric patients with growth problems by conducting growth and development assessments. Implement the MENBS clinical interventions for pediatric patients with growth problems. Receives: Behavioral: MENBS clinical interventions |
What is being measured
The primary outcome is the main question the study is designed to answer.
Bone age
one age was assessed from left hand-wrist radiographs according to the Chinese Hand-Wrist Bone Development Standard (China 05).
Measured: Up to 1 year
Height-for-age (m)
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
Measured: Up to 1 yearUp to 1 year
Body mass index-for-age (BMI-for-age, kg/m^2)
It is assessed using the 'Growth Standard for Children under 7 Years of Age' and the 'Dietary Guidelines for Chinese Residents'.
Measured: Up to 1 yearUp to 1 year
Endocrine function
Endocrine function such as hormonal levels (IGF-1, GH, etc.) will be measured by clinical examination
Measured: Up to 1 year
Nutritional status
Nutritional status will be measured by clinical examination, such as concentration of Vitamin D and Zinc
Measured: Up to 1 year
Intelligence quotient (IQ)
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.
Measured: Up to 1 year
Brain function
Brain function will be measured by Functional Near-infrared Spectroscopy (fNIRS) to record brain activations
Measured: Up to 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2025
- Study start1 Dec 2024
- Primary completion31 Oct 2026 (estimated)
- Study completion30 Nov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thalassemia IntermediaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 385 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06931912
- Sponsor's protocol ID
- CAMSCRF2024041
- Status last verified
- Jul 2026
- First submitted
- 7 Jan 2025
- Last updated
- 18 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia
- ClinicalTrials.gov ID
- NCT06931912
- Acronym / sponsor ID
- CAMSCRF2024041
- Keywords
- Thalassemia, Children and adolescents, Growth development, Health Promotion, Clinical Management
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 385 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 1 Dec 2024 (actual)
- Primary completion
- 31 Oct 2026 (estimated)
- Study completion
- 30 Nov 2026 (estimated)
- First posted
- 17 Apr 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
- 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.
- 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.
- Repeat growth assessment for pediatric patients with growth problems after 1-year clinical interventions.
- Evaluate the effectiveness of MENBS interventions by comparing changes in growth and development indicators.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pediatric patients with growth problems | Experimental | Behavioral: MENBS clinical interventionsWe identify pediatric patients with growth problems by conducting growth and development assessments. Implement the MENBS clinical interventions for pediatric patients with growth problems. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Subjects diagnosed with transfusion-dependent thalassemia (TDT)
- Male or female age ≤19 years
- Subjects who are willing and able to provide written informed consent
Key exclusion criteria (2 listed)
- Subjects with clinically significant thyroid dysfunction
- Subjects received allo-HSCT or gene therapy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MENBS clinical interventions* 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. | Behavioural | Pediatric patients with growth problems |
Primary outcome
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).
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
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'.
Endocrine function
Time frame: Up to 1 year
Endocrine function such as hormonal levels (IGF-1, GH, etc.) will be measured by clinical examination
Nutritional status
Time frame: Up to 1 year
Nutritional status will be measured by clinical examination, such as concentration of Vitamin D and Zinc
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.
Brain function
Time frame: Up to 1 year
Brain function will be measured by Functional Near-infrared Spectroscopy (fNIRS) to record brain activations
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2026.
- Individual participant dataSharing planned: No
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Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
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RecruitingPhase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
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A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing MENBS clinical interventions in people with Thalassemia Intermedia and Thalassemia Major, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 18 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Bone age (time frame: Up to 1 year). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Sep 2026.