- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05991336
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
August 6, 2023 updated by: Jun Shi, Institute of Hematology & Blood Diseases Hospital
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.
Study Overview
Status
Recruiting
Intervention / Treatment
Study Type
Observational
Enrollment (Estimated)
100
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Jun Shi, PhD
- Phone Number: 2223900913
- Email: shijun@ihcams.ac.cn
Study Contact Backup
- Name: Jingyu Zhao, MPH
- Phone Number: 13752253515
- Email: zhaojingyu@ihcams.ac.cn
Study Locations
-
-
Tianjin
-
Tianjin, Tianjin, China
- Recruiting
- Regenerative Medicine Center
-
Contact:
- Jun Shi, PhD
- Phone Number: (86)2223900913
- Email: shijun@ihcams.ac.cn
-
Contact:
- Jingyu Zhao, MPH
- Phone Number: (86)13752253515
- Email: zhaojingyu@ihcams.ac.cn
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
Accepts Healthy Volunteers
Yes
Sampling Method
Non-Probability Sample
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.
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
Gene therapy group
TDT Children who have received gene therapy between 3~14 years old.
|
Autologous edited hematopoietic stem cell transplantation
|
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 the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Growth in pediatric subjects will be assessed by height.
Time Frame: Up to 5 years.
|
Height in meters.
|
Up to 5 years.
|
Growth in pediatric subjects will be assessed by weight.
Time Frame: Up to 5 years.
|
Weight in kilograms.
|
Up to 5 years.
|
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.
|
Up to 5 years.
|
Growth in pediatric subjects will be assessed by interocular distance.
Time Frame: Up to 5 years.
|
Interocular distance in centimeters.
|
Up to 5 years.
|
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).
|
Up to 5 years.
|
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.
|
Up to 5 years.
|
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.
|
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.
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.
|
Up to 5 years.
|
Lifestyle behaviors will be assessed by general questioning.
Time Frame: Up to 5 years.
|
Up to 5 years.
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
June 5, 2023
Primary Completion (Estimated)
December 31, 2028
Study Completion (Estimated)
December 31, 2029
Study Registration Dates
First Submitted
July 27, 2023
First Submitted That Met QC Criteria
August 6, 2023
First Posted (Actual)
August 14, 2023
Study Record Updates
Last Update Posted (Actual)
August 14, 2023
Last Update Submitted That Met QC Criteria
August 6, 2023
Last Verified
August 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- GDH-001
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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