Clinical Study of Super Transplantation in the Treatment of Severe β-thalassemia

NCT06734520 | Recruiting DMC

• 1 other identifier: 2024217A01
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 2

Brief Summary

This is a single-arm, open label, phase 1 study in subjects with beta-thalassemia. This study will evaluate the safety and efficacy of preconditioning-free super-transplantation on beta-thalassemia pediatric patients.

Conditions

Thalassemia Majors (Beta-Thalassemia Major); Haplo-identical Donors

Outcome Measures

Primary Outcomes (1)

• chimeric status

  Detecting the chimerism in recipients' peripheral blood and bone marrow.Chimerism is generally measured in percentages(%)

  1 year after transplantation.

Study Arms (1)

Severe β -thalassemia

EXPERIMENTAL

Children with β-thalassemia major received super transplantation, and chimerism tests were performed at 3 months, 6 months, 9 months, and 1 year after transplantation to evaluate the safety and effectiveness.

Drug: super-transplantation in 3 severe β -thalassemia pediatric patients.

Interventions

super-transplantation in 3 severe β -thalassemia pediatric patients. DRUG

Haplo-identical donors are mobilized with G-CSF and PBMCs that contain CD34+ stem cells and CD3+ lymphocytes will be extracted by blood cell separators. Then the patients will be infused with the PBMC

Severe β -thalassemia

Eligibility Criteria

• Age: 7 Years - 10 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Diagnosis of severe β -thalassemia
• Age between 7-10 years old, male and female; Weight \< 40kg
• The patient has or does not have an HLA-compatible or semi-compatible donor, but unconditional transplantation or refusal of blood stem cell transplantation; Patients with thalassemia gene therapy without conditions or refusal;
• There are fully compatible or incompatible HLA donors, and the physical examination meets the donor conditions;
• The patient and family members agree to receive hypertransplant therapy and sign a written informed consent prior to the transplant trial.

You may not qualify if:

• Mental patients;
• Participants in other drug clinical trials within the past 1 month;
• There are no suitable HLA-incompatible donors.
• Other researchers decide that it is not suitable to participate in this researcher.

Sponsors & Collaborators

• Guangzhou Women and Children's Medical Center: lead

Study Sites (2)

Guangzhou Women and Children's Medical Center Affiliated to Guangzhou Medical University

Guangzhou, Guangdong, 51000, China

ACTIVE NOT RECRUITING

Guangzhou Women and Children's Medical Center Affiliated to Guangzhou Medical University

Guangzhou, Guangdong, China

RECRUITING

Related Publications (3)

• Ali S, Mumtaz S, Shakir HA, Khan M, Tahir HM, Mumtaz S, Mughal TA, Hassan A, Kazmi SAR, Sadia, Irfan M, Khan MA. Current status of beta-thalassemia and its treatment strategies. Mol Genet Genomic Med. 2021 Dec;9(12):e1788. doi: 10.1002/mgg3.1788. Epub 2021 Nov 5.

  PMID: 34738740 BACKGROUND

• Shafique F, Ali S, Almansouri T, Van Eeden F, Shafi N, Khalid M, Khawaja S, Andleeb S, Hassan MU. Thalassemia, a human blood disorder. Braz J Biol. 2021 Sep 3;83:e246062. doi: 10.1590/1519-6984.246062. eCollection 2021.

  PMID: 34495151 BACKGROUND

• Dever DP, Bak RO, Reinisch A, Camarena J, Washington G, Nicolas CE, Pavel-Dinu M, Saxena N, Wilkens AB, Mantri S, Uchida N, Hendel A, Narla A, Majeti R, Weinberg KI, Porteus MH. CRISPR/Cas9 beta-globin gene targeting in human haematopoietic stem cells. Nature. 2016 Nov 17;539(7629):384-389. doi: 10.1038/nature20134. Epub 2016 Nov 7.

  PMID: 27820943 BACKGROUND

MeSH Terms

Conditions

beta-Thalassemia

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Hua Jiang, PHD

  Guangzhou Medical University Affiliated Women's and Children's Medical Center

  STUDY DIRECTOR

Central Study Contacts

Hua Jiang, Doctor

CONTACT

86-020-38076501; jiang_hua18@sina.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 16, 2024
• First Posted: December 16, 2024
• Study Start: October 1, 2024
• Primary Completion (Estimated): July 1, 2029
• Study Completion (Estimated): July 1, 2029
• Last Updated: December 16, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: The start date is October 2024 and the end date is July 2029
• Access Criteria: The sponsors and collaborators are able to access the IPD and supporting information, IPD relies on team collaboration , as well as continuous improvement in innovation and efficiency. Participants will share project information, including the project's objectives, statistical datas, and clinical study report.

Locations

CN (2)