NCT05757245RecruitingPhase 1
A Study Evaluating the Safety and Efficacy of the GMCN-508A Drug Product in Transfusion-dependent α-Thalassemia Participants
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)
Source: ClinicalTrials.gov · Last updated 18 Apr 2023 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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 \[autologous CD34+ hematopoietic stem cells transduced with GMCN-508A lentiviral vector encoding the human α-globin gene\].
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 5 (estimated)
- First submitted
- 24 Feb 2023
- Start date
- 8 May 2023
- Primary completion date
- 1 Aug 2028
- Lead sponsor
- First Affiliated Hospital of Guangxi Medical University
- Diseases
- Thalassaemia
- NCT number
- NCT05757245
What you commit to
- Study type: Interventional · Phase 1
- Planned participants: 5
- From 8 May 2023 to the primary completion date, 1 Aug 2028
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
Other illness
- 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.
Lab values
- 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.
Other
- Presence of severe iron overload.
- Pregnancy, plan to be pregnant during study or breastfeeding in a postpartum female.
Who the study is looking for
- 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.
Age in the registry: 5 years to 35 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| The affiliated hospital of guangxi medical university Coordinator of this site? Confirm its status | Nanning | China | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.