Transfusion-dependent Beta-ThalassemiaNCT05991336
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
An observational study of Transfusion-dependent Beta-Thalassemia and Gene Therapy, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
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… Read more about this study
- Who is it for?
- Ages 3 to 14 yearsHealthy volunteers can take part
- Conditions the study is for
- Transfusion-dependent Beta-Thalassemia, Gene Therapy
- What is being tested?
- Gene therapy
- How many people
- 100 participants
- Where
- 1 siteChina
- Study dates
- 5 Jun 2023to 31 Dec 2028
- Phase
- Observational
- 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 3 to 14 years
- All sexes
- Healthy volunteers accepted
- Child
Study population: Participants are divided into three groups: TDT children who have received gene therapy, TDT children who have received lifelong supportive therapy, and healthy children.
Who may be able to join
- 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
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- 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.
Who cannot join
- 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 :
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Regenerative Medicine Center
Tianjin, Tianjin Municipality
Jun Shi, PhD, (86)2223900913, shijun@ihcams.ac.cn
Jingyu Zhao, MPH, (86)13752253515, zhaojingyu@ihcams.ac.cn
About this study
Official title: Growth and Development, Health-related Quality of Life of 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.
- Being tested
- Gene therapy
- Keywords
- Transfusion-dependent Beta-Thalassemia, gene therapy, cohort study, case-control
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gene therapyGenetic
Autologous edited hematopoietic stem cell transplantation
| Study group | Role | What they receive |
|---|---|---|
| Gene therapy group | TDT Children who have received gene therapy between 3~14 years old. Receives: Genetic: Gene therapy | |
| Supportive therapy group | TDT Children who received lifelong treatment with blood transfusions and iron chelation. They were selected based on the age and sex of the gene therapy group. | |
| Healthy children group | Healthy children were selected based on the age and sex of the gene therapy group. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Growth in pediatric subjects will be assessed by height.
Height in meters.
Measured: Up to 5 years.
Growth in pediatric subjects will be assessed by weight.
Weight in kilograms.
Measured: Up to 5 years.
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).
Measured: Up to 5 years.
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.
Measured: Up to 5 years.
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.
Measured: Up to 5 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Aug 2023
- Study start5 Jun 2023
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transfusion-dependent Beta-ThalassemiaStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jun Shi, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05991336
- Sponsor's protocol ID
- QTJC2023054
- Status last verified
- Nov 2026
- First submitted
- 27 Jul 2023
- Last updated
- 4 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Growth and Development, Health-related Quality of Life of Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
- ClinicalTrials.gov ID
- NCT05991336
- Acronym / sponsor ID
- QTJC2023054
- Keywords
- Transfusion-dependent Beta-Thalassemia, gene therapy, cohort study, case-control
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 5 Jun 2023 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 14 Aug 2023 (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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Gene therapy group | – | Genetic: Gene therapyTDT Children who have received gene therapy between 3~14 years old. |
| Supportive therapy group | – | –TDT Children who received lifelong treatment with blood transfusions and iron chelation. They were selected based on the age and sex of the gene therapy group. |
| Healthy children group | – | –Healthy children were selected based on the age and sex of the gene therapy group. |
Who may qualify
- Ages
- 3 to 14 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
- Study population
- Participants are divided into three groups: TDT children who have received gene therapy, TDT children who have received lifelong supportive therapy, and healthy children.
Key inclusion criteria (14 listed)
- 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
Key exclusion criteria (10 listed)
- 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
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 |
|---|---|---|
| Gene therapyAutologous edited hematopoietic stem cell transplantation | Genetic | Gene therapy group |
Primary outcome
Growth in pediatric subjects will be assessed by height.
Time frame: Up to 5 years.
Height in meters.
Growth in pediatric subjects will be assessed by weight.
Time frame: Up to 5 years.
Weight in kilograms.
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).
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.
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.
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 4 Nov 2026.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Transfusion-dependent Beta-Thalassemia and Gene Therapy, 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 14 inclusion and 10 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: looking for participants now, as of the sponsor's last update on 4 Nov 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?
Contact Jun Shi, PhD (2223900913, shijun@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Growth in pediatric subjects will be assessed by height. (time frame: Up to 5 years.). The study lists 5 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 4 Nov 2026.