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

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Using a monoclonal antibody to prevent graft-versus-host disease in thalassemia patients after stem cell transplantation

  This study is testing if a new antibody can help prevent graft-versus-host disease in thalassemia patients who are receiving stem cell transplants.

  Recruiting at 5 sites Phase 2 First Affiliated Hospital of Guangxi Medical University · Maoming, Guangdong and 4 other locations · NCT06657391

• Gene-modified stem cell therapy for transfusion-dependent beta-thalassemia

  A small, early-phase study will test a one-time infusion of a patient’s own genetically edited stem cells to see if it reduces transfusion needs in adults and children with transfusion-dependent beta-thalassemia.

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• Mitapivat for pediatric transfusion-dependent thalassemia (α- or β-TDT)

  This pediatric trial tests whether mitapivat can reduce or lessen the need for transfusions in children with transfusion-dependent alpha- or beta-thalassemia.

  Recruiting at 4 sites Phase 3 Agios Pharmaceuticals, Inc. · Atlanta, Georgia and 3 other locations · NCT07506863

• Treatment for Beta-Thalassemia using KL003 Cell Injection

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  Recruiting at 2 sites Phase 1/2 Kanglin Biotechnology (Hangzhou) Co., Ltd. · Shanghai, Shanghai Municipality and 1 other locations · NCT06280378

• Comparing growth and development in children with beta-thalassemia after gene therapy

  This study looks at how gene therapy affects the growth and development of children with beta-thalassemia compared to those getting regular treatments and healthy kids.

  Recruiting Institute of Hematology & Blood Diseases Hospital, China · Tianjin, Tianjin Municipality · NCT05991336

• Evaluating a new gene therapy for transfusion-dependent beta-thalassemia

  This study is testing a new gene therapy to see if it can help people with transfusion-dependent beta-thalassemia reduce or stop their need for regular blood transfusions.

  Recruiting Early Phase 1 First Affiliated Hospital of Guangxi Medical University · Nanning, Guangxi · NCT05762510

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.