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

Recruiting now · Phase 1

Conditions studied: Transfusion-dependent α-Thalassemia

In brief

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 \[autologous CD34+ hematopoietic stem cells transduced with GMCN-508A lentiviral vector encoding the human α-globin gene\].

Key facts

Study ID

NCT05757245

Run by

First Affiliated Hospital of Guangxi Medical University

People needed

5

Starts

2023-05-08

Expected to finish

2030-12-31

Last updated by the study team

2023-04-18

Who can join

Age: 5 and older, up to 35. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• The affiliated hospital of guangxi medical university — Nanning, Guangxi, China (enrolling)

Full record on ClinicalTrials.gov

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