Comparing growth and development in children with beta-thalassemia after gene therapy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational cohort study aims to evaluate the growth, development, metabolism, lifestyle behaviors, and health-related quality of life in children aged 3-14 years with transfusion-dependent beta-thalassemia (TDT). The study will compare three groups: children who have received gene therapy, those receiving lifelong supportive therapy, and healthy children. Participants will be required to provide informed consent, and the study will focus on understanding the long-term outcomes of gene therapy in this population.

Why it matters

Who can join: the full rules

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

Where it's running

• Regenerative Medicine Center
  Tianjin, Tianjin Municipality, China Recruiting

Study contacts

• Study coordinator: Jun Shi, PhD
• Email: shijun@ihcams.ac.cn
• Phone: 2223900913

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Gene-modified stem cell therapy for transfusion-dependent beta-thalassemia

  A small, early-phase study will test a one-time infusion of a patient’s own genetically edited stem cells to see if it reduces transfusion needs in adults and children with transfusion-dependent beta-thalassemia.

  Recruiting at 2 sites Phase 2 Fondazione Telethon · Rome, Lazio and 1 other locations · NCT07680803

• Mitapivat for pediatric transfusion-dependent thalassemia (α- or β-TDT)

  This pediatric trial tests whether mitapivat can reduce or lessen the need for transfusions in children with transfusion-dependent alpha- or beta-thalassemia.

  Recruiting at 4 sites Phase 3 Agios Pharmaceuticals, Inc. · Atlanta, Georgia and 3 other locations · NCT07506863

• KLN-1010 gene therapy for relapsed or refractory multiple myeloma

  This Phase 1 trial will try KLN-1010, an in vivo CAR‑T gene therapy, to see if it is safe and can be given at a tolerable dose for people with relapsed or refractory multiple myeloma who have had at least three prior therapies.

  Recruiting at 11 sites Phase 1 Kelonia Therapeutics, Inc. · Duarte, California and 10 other locations · NCT07075185

• Perceptual evaluation and rehabilitation program for congenital hearing loss

  This project will try a gamified, age‑adjusted software program to measure and train speech, music, and spatial hearing in people born with hearing loss who use hearing aids, cochlear implants, or gene therapy.

  Recruiting Eye & ENT Hospital of Fudan University · Shanghai · NCT07024524

• Treatment for Beta-Thalassemia using KL003 Cell Injection

  This study is testing a new gene therapy using KL003 cell injections to see if it can help young people with severe beta-thalassemia reduce their need for blood transfusions.

  Recruiting at 2 sites Phase 1/2 Kanglin Biotechnology (Hangzhou) Co., Ltd. · Shanghai, Shanghai Municipality and 1 other locations · NCT06280378

• Evaluating a new gene therapy for transfusion-dependent beta-thalassemia

  This study is testing a new gene therapy to see if it can help people with transfusion-dependent beta-thalassemia reduce or stop their need for regular blood transfusions.

  Recruiting Early Phase 1 First Affiliated Hospital of Guangxi Medical University · Nanning, Guangxi · NCT05762510

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.