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Tests treatment safety and results for Transfusion-dependent α-Thalassemia
Official title A Study Evaluating the Safety and Efficacy of the GMCN-508A Drug Product in Transfusion-dependent α-Thalassemia Participants
ClinicalTrials.gov ID: NCT05757245
What this study is testing
What is GMCN-508A Drug Product?
GMCN-508A Drug Product is an investigational medicine, being studied as a potential treatment for transfusion-dependent α-thalassemia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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].
- Phase 1: an early, usually small safety study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 5 to 35
You may be able to join 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...
- 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...
- Karnofsky Level of Performance (KPS) score or Lansky Level of Performance (LPS) score ≥70.
- people were determined to undergo autologous hematopoietic stem cell transplantation and conditioning procedure by the principle investigator.
You likely can't join if
- Diagnosed with mild α-thalassemia, Hb Bart's edema, ATRx α-thalassemia, hemoglobin S/β-thalassemia, myelodysplastic subtype anemia, or with HbE...
- 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.
See the full eligibility criteria
- 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.
- people were determined to undergo autologous hematopoietic stem cell transplantation and conditioning procedure by the principle investigator.
- people were willing to comply with the protocol.
- Fertile people are willing to take effective contraceptive measures during the study.
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Nanning, Guangxi, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 5 years to 35 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Nanning, Guangxi, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.