Evaluating GMCN-508A for treating transfusion-dependent α-thalassemia

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 study is testing a new treatment using a patient's own stem cells to see if it can help young people with transfusion-dependent α-thalassemia live healthier lives.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 5 (estimated)
• Ages: 5 Years to 35 Years
• Sex: All
• Sponsor: First Affiliated Hospital of Guangxi Medical University (Academic / other)
• Locations: 1 site (Nanning, Guangxi)
• Trial ID: NCT05757245 on ClinicalTrials.gov

What this trial studies

This phase 1 clinical trial is a non-randomized, open-label study involving up to 5 participants aged 5 to 35 years with transfusion-dependent α-thalassemia. It aims to assess the safety and efficacy of autologous hematopoietic stem cell transplantation using the GMCN-508A drug product, which involves transducing the patient's own CD34+ hematopoietic stem cells with a lentiviral vector encoding the human α-globin gene. Participants will be monitored for a duration of 5 years to evaluate treatment outcomes.

Who should consider this trial

Good fit: Ideal candidates for this study are individuals aged 5 to 35 years who are diagnosed with transfusion-dependent α-thalassemia and meet specific performance and treatment criteria.

Not a fit: Patients with mild α-thalassemia or other specified hemoglobin disorders may not benefit from this treatment.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce or eliminate the need for blood transfusions in patients with α-thalassemia.

How similar studies have performed: While this approach is novel, similar gene therapy strategies have shown promise in treating other hemoglobinopathies, indicating potential for success.

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.

Where this trial is running

Nanning, Guangxi

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

Conditions Transfusion-dependent α-Thalassemia; GMCN-508A; gene therapy; Human Hematopoietic Stem Cell