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

Korábbi kezelés

• 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.

Más betegség

• 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.

Laborértékek

• 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.

Egyéb

• Presence of severe iron overload.
• Pregnancy, plan to be pregnant during study or breastfeeding in a postpartum female.

• 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.