β0/β0 Genotipi Olmayan Transfüzyona Bağımlı β-Talasemi Olan Katılımcılarda LentiGlobin® BB305 İlacının Etkililiğini ve Güvenliğini Değerlendiren Çalışma

Sade dilde özet

Bu çalışma, β0/β0 genotipi taşımayan, transfüzyona bağımlı β-talasemi (transfusion-dependent β-thalassemia) hastalarında LentiGlobin BB305 adlı bir gen terapisinin etkinliğini ve güvenliğini değerlendirmektedir. Çalışmaya, 50 yaş ve altında olan, kök hücre nakline uygun ve HBB geninde iki β0 mutasyonu bulunmayan hastalar alınmıştır. Faz 3 aşamasında yürütülmüş ve tamamlanmıştır.

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

Deney bilgileri

Faz

Faz 3 (Phase 3)

Durum

Tamamlandı

Sponsor

Genetix Biotherapeutics Inc.

Müdahale

LentiGlobin BB305 Drug Product

Başlangıç

08.08.2016

Tahmini bitiş

31.03.2022

Ülkeler

United States, France, Germany, Italy, Thailand, United Kingdom

İlgili genler

Merkezler

• Oakland, United States
• Chicago, United States
• Philadelphia, United States
• Marseille, France
• Hanover, Germany
• Rome, Italy
• Bangkok, Thailand
• London, United Kingdom

Uygunluk kriterleri

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

Inclusion Criteria:

* Participants \<= 50 years of age at the time of consent or assent (as applicable), and able to provide written consent (adults, or legal guardians, as applicable) or assent (adolescents or children). Provided that the Data Monitoring Committee (DMC) has approved enrolling participants younger than 5 years of age, participants younger than 5 years of age may be enrolled if they weigh a minimum of 6 kilograms (kg) and are reasonably anticipated to be able to provide at least the minimum number of cells required to initiate the manufacturing process.
* Diagnosis of TDT with a history of at least 100 milliliter per kilogram per year (mL/kg/year) of pRBCs in the 2 years preceding enrollment (all participants), or be managed under standard thalassemia guidelines with \>= 8 transfusions of pRBCs per year in the 2 years preceding enrollment (participants \>= 12 years).
* Clinically stable and eligible to undergo (HSCT).
* Treated and followed for at least the past 2 years in a specialized center that maintained detailed medical records, including transfusion history.

Exclusion Criteria:

* Presence of a mutation characterized as β0 mutation at both alleles of the β-globin gene HBB.
* Positive for presence of human immunodeficiency virus type 1 or 2 (HIV-1 and HIV-2), hepatitis B virus (HBV), or hepatitis C (HCV).
* A white blood cell (WBC) count less than (\<) 3×10\^9/Liter (L), and/or platelet count \< 100×10\^9/L not related to hypersplenism.
* Uncorrected bleeding disorder.
* Any prior or current malignancy.
* Immediate family member with a known Familial Cancer Syndrome.
* Prior HSCT.
* Advanced liver disease.
* A cardiac T2\* \< 10 ms by MRI.
* Any other evidence of severe iron overload that, in the Investigator's opinion, warrants exclusion.
* Participation in another clinical study with an investigational drug within 30 days of Screening.
* Any other condition that would render the participant ineligible for HSCT, as determined by the attending transplant physician or investigator.
* Prior receipt of gene therapy.
* Pregnancy or breastfeeding in a postpartum female or absence of adequate contraception for fertile participant.
* A known and available Human leukocyte antigen (HLA) matched family donor.
* Any contraindications to the use of granulocyte colony stimulating factor (G-CSF) and plerixafor during the mobilization of hematopoietic stem cells and any contraindications to the use of busulfan and any other medicinal products required during the myeloablative conditioning, including hypersensitivity to the active substances or to any of the excipients.