A Study Evaluating the Safety and Efficacy of the GMCN-508A Drug Product in Transfusion-dependent α-Thalassemia Participants
A Phase 1 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With Transfusion-dependent α-Thalassemia by Transplantation of Autologous CD34+ Cells Transduced Ex Vivo With a Lentiviral Vector (GMCN-508A Drug Product)
First Affiliated Hospital of Guangxi Medical University
5 participants
May 8, 2023
INTERVENTIONAL
Conditions
Summary
This is a non-randomized, open label, single-site, single-dose, phase 1 study in up to 5 participants (between 5 and 35 years of age, inclusive) with Transfusion-dependent α-thalassemia. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using GMCN-508A Drug Product \[autologous CD34+ hematopoietic stem cells transduced with GMCN-508A lentiviral vector encoding the human α-globin gene\].
Eligibility
Inclusion Criteria7
- 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.
- Ages 5 to 35, no gender limitation.
- 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.
- Karnofsky Level of Performance (KPS) score or Lansky Level of Performance (LPS) score ≥70.
- Subjects were determined to undergo autologous hematopoietic stem cell transplantation and conditioning procedure by the principle investigator.
- Subjects were willing to comply with the protocol.
- Fertile Subjects are willing to take effective contraceptive measures during the study.
Exclusion Criteria13
- 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.
- Uncorreted Bleeding disorders with frequent bleeding (eg, menorrhagia, epistaxis, coagulation disorders).
- Bacterial, fungal, parasitic or viral infection as determined by the investigator to be clinically significant.
- Presence of severe iron overload.
- Any prior or current malignancy, myeloproliferative disorders or immunodeficiency disorders.
- Any major medical disease, laboratory test abnormality or mental illness that would render the participant ineligible for the study.
- Immediate family member with a known Familial Cancer Syndrome.
- Prior receipt of gene therapy, allogeneic bone marrow transplantation or allogeneic hematopoietic stem cell transplantation.
- Participation in another clinical study with an investigational drug 3 months prior to Screening.
- Pregnancy, plan to be pregnant during study or breastfeeding in a postpartum female.
- Known hypersensitivity to any ingredients or excipients of the test drug.
- Eligible for allogeneic bone marrow transplantation or allogeneic hematopoietic stem cell transplantation with a known and available donor.
- Any other condition that would render the participant ineligible for the study, as determined by the investigator.
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Interventions
Transplant of autologous hematopoietic stem and progenitor cells transduced with GMCN-508A lentiviral vector.
Locations(1)
View Full Details on ClinicalTrials.gov
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NCT05757245