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Gene Therapy Trial Report

Summary

A Study Evaluating the Safety and Efficacy of the GMCN-508A Drug Product in Transfusion-dependent α-Thalassemia Participants


NCTID NCT05757245 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Transfusion-dependent Alpha-Thalassemia
Disease Ontology Term DOID:1099
Compound Name GMCN-508A
Sponsor First Affiliated Hospital of Guangxi Medical University
Funder Type Other
Recruitment Status
Enrollment Count 5 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant HBA
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Viral transduction
Vector Type LV
Editor Type none
Dose 1 Transduced CD34+ cells

Study Record Dates


Current Stage Phase1
Submit Date 2023-02-24
Completion Date 2030-12-31
Last Update 2023-04-18

Participation Criteria


Eligible Age 5 Years - 35 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. The subject himself/herself or one legal guardian/agent of the subject is required to fully understand the study and voluntarily sign a written informed consent. 2. Ages 5 to 35, no gender limitation. 3. The clinical diagnosis of Transfusion-dependent α-Thalassemia.Transfusion dependence was defined as ≥6 Units of transfusions of pRBCs for the prior 24 weeks without \>56 days of non-transfusion. 4. Karnofsky Level of Performance (KPS) score or Lansky Level of Performance (LPS) score ≥70. 5. Subjects were determined to undergo autologous hematopoietic stem cell transplantation and conditioning procedure by the principle investigator. 6. Subjects were willing to comply with the protocol. 7. Fertile Subjects are willing to take effective contraceptive measures during the study. Exclusion Criteria: 1. Diagnosed with mild α-thalassemia, Hb Bart's edema, ATRx α-thalassemia, hemoglobin S/β-thalassemia, myelodysplastic subtype anemia, or with HbE homozygous β gene mutation, or with any type of β-thalassemia Thalassemia. 2. Uncorreted Bleeding disorders with frequent bleeding (eg, menorrhagia, epistaxis, coagulation disorders). 3. Bacterial, fungal, parasitic or viral infection as determined by the investigator to be clinically significant. 4. Presence of severe iron overload. 5. Any prior or current malignancy, myeloproliferative disorders or immunodeficiency disorders. 6. Any major medical disease, laboratory test abnormality or mental illness that would render the participant ineligible for the study. 7. Immediate family member with a known Familial Cancer Syndrome. 8. Prior receipt of gene therapy, allogeneic bone marrow transplantation or allogeneic hematopoietic stem cell transplantation. 9. Participation in another clinical study with an investigational drug 3 months prior to Screening. 10. Pregnancy, plan to be pregnant during study or breastfeeding in a postpartum female. 11. Known hypersensitivity to any ingredients or excipients of the test drug. 12. Eligible for allogeneic bone marrow transplantation or allogeneic hematopoietic stem cell transplantation with a known and available donor. 13. Any other condition that would render the participant ineligible for the study, as determined by the investigator.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links


Resources/Links

No External Links Available.