Gene Therapy Study for Transfusion-Dependent Alpha-Thalassemia in Young People
Tests a gene-modified stem cell treatment in people aged 5 to 35 with alpha-thalassemia.
Plain-language title generated from the registry record
Registry title: A Study Evaluating the Safety and Efficacy of the GMCN-508A Drug Product in Transfusion-dependent α-Thalassemia Participants
Key facts
- Where
- Nanning, ChinaSee the site
- Phase
- Phase 1
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 5 and 35 years old?
Source: NCT05757245, who can take part
Where in China
- The affiliated hospital of guangxi medical universityNanning, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- First Affiliated Hospital of Guangxi Medical University
- Trial code
- NCT05757245
- Record updated
- 18 April 2023
- Original description
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].Read full original descriptionShow less
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)
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].
Inclusion: 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: Any prior or current malignancy, myeloproliferative disorders or immunodeficiency disorders; 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; Eligible for allogeneic bone marrow transplantation or allogeneic hematopoietic stem cell transplantation with a known and available donor; 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; Immediate family member with a known Familial Cancer Syndrome; Known hypersensitivity to any ingredients or excipients of the test drug; Any other condition that would render the participant ineligible for the study, as determined by the investigator; 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; Any major medical disease, laboratory test abnormality or mental illness that would render the participant ineligible for the study; Presence of severe iron overload; Pregnancy, plan to be pregnant during study or breastfeeding in a postpartum female.