Transfüzyona Bağımlı α-Talasemi Katılımcılarında GMCN-508A İlaç Ürününün Güvenliğini ve Etkinliğini Değerlendiren Bir Çalışma

Sade dilde özet

Bu Faz 1 (Phase 1), randomize olmayan, açık etiketli, tek merkezli ve tek dozluk çalışma, transfüzyona bağımlı α-talasemi (transfusion-dependent α-thalassemia) olan en fazla 5 katılımcıda GMCN-508A'yı inceliyor. GMCN-508A, insan α-globin genini taşıyan bir lentiviral vektör (lentiviral vector) ile değiştirilmiş, hastanın kendi CD34+ kan yapıcı kök hücrelerinden (autologous hematopoietic stem cells) oluşur ve otolog kök hücre nakli (autologous hematopoietic stem cell transplantation, HSCT) ile verilir. Amaç, güvenlik ve etkinliği değerlendirmektir; katılımcıların 5-35 yaş arasında olması ve transfüzyona bağımlı α-talasemi klinik tanısı bulunması gerekmektedir.

Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.

Deney bilgileri

Faz

Faz 1 (Phase 1)

Durum

Katılımcı alıyor

Sponsor

First Affiliated Hospital of Guangxi Medical University

Müdahale

GMCN-508A Drug Product

Başlangıç

08.05.2023

Tahmini bitiş

31.12.2030

Ülkeler

China

İlgili genler

Merkezler

• Nanning, China

Uygunluk kriterleri

Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.

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.