Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy

Plain-language summary

This study follows growth and development-related outcomes after gene therapy in children with transfusion-dependent beta-thalassemia. It enrolls children who have received gene therapy, along with children on supportive therapy and healthy children (ages 3-14) as comparison groups. It is an observational study with no assigned phase and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Recruiting

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Intervention

Gene therapy

Start date

6/5/2023

Estimated completion

12/31/2029

Countries

China

Related genes

Locations

• Tianjin, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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