Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
NCT ID: NCT05991336
Last Updated: 2023-08-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
100 participants
OBSERVATIONAL
2023-06-05
2029-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Gene therapy group
TDT Children who have received gene therapy between 3\~14 years old.
Gene therapy
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.
No interventions assigned to this group
Healthy children group
Healthy children were selected based on the age and sex of the gene therapy group.
No interventions assigned to this group
Interventions
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Gene therapy
Autologous edited hematopoietic stem cell transplantation
Eligibility Criteria
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Inclusion Criteria
* 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
* 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
3 Years
14 Years
ALL
Yes
Sponsors
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Institute of Hematology & Blood Diseases Hospital, China
OTHER
Responsible Party
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Jun Shi
Director, Regenerative Medicine Clinic Center
Locations
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Regenerative Medicine Center
Tianjin, Tianjin Municipality, China
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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GDH-001
Identifier Type: -
Identifier Source: org_study_id
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