Hemofili A Hastalarında Valoctocogene Roxaparvovec'in Etkililik ve Güvenliliğini Değerlendiren Tek Kollu Çalışma (BMN 270-301)

Sade dilde özet

Bu çalışma, hemofili A (hemophilia A) olan hastalarda, valoctocogene roxaparvovec adlı bir gen terapisinin etkililiğini ve güvenliliğini tek kollu olarak değerlendirmiştir. Çalışmaya, FVIII düzeyi ≤ 1 IU/dL olan ve en az 12 aydır önleyici FVIII replasman tedavisi alan, 18 yaş ve üzeri erkek hastalar alınmıştır. Çalışma 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

BioMarin Pharmaceutical

Müdahale

valoctocogene roxaparvovec

Başlangıç

19.12.2017

Tahmini bitiş

20.11.2024

Ülkeler

United States, Australia, Belgium, Brazil, France, Germany, Israel, Italy, South Africa, South Korea, Spain, Taiwan, United Kingdom

İlgili genler

Merkezler

• Los Angeles, United States
• Sacramento, United States
• San Diego, United States
• San Francisco, United States
• Aurora, United States
• Tampa, United States
• Chicago, United States
• Louisville, United States
• New Orleans, United States
• Ann Arbor, United States
• Detroit, United States
• Minneapolis, United States
• St Louis, United States
• Chapel Hill, United States
• Columbus, United States
• Adelaide, Australia
• Brisbane, Australia
• Melbourne, Australia
• Perth, Australia
• Sydney, Australia
• Leuven, Belgium
• Campinas, Brazil
• Curitiba, Brazil
• Rio de Janeiro, Brazil
• São Paulo, Brazil
• Vitória, Brazil
• Lille, France
• Marseille, France
• Berlin, Germany
• Bonn, Germany
• Ramat Gan, Israel
• Milan, Italy
• Johannesburg, South Africa
• Seoul, South Korea
• A Coruña, Spain
• Seville, Spain
• Changhua, Taiwan
• Kaohsiung City, Taiwan
• Taichung, Taiwan
• Taipei, Taiwan

+9

Uygunluk kriterleri

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

Inclusion Criteria:

1. Males ≥ 18 years of age with hemophilia A and residual FVIII levels ≤ 1 IU/dL as evidenced by medical history, at the time of signing the informed consent.
2. Must have been on prophylactic FVIII replacement therapy for at least 12 months prior to study entry.
3. Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure days (EDs).
4. No previous documented history of a detectable FVIII inhibitor, and results from a Bethesda assay or Bethesda assay with Nijmegen modification of less than 0.6 Bethesda Units (BU) on 2 consecutive occasions at least one week apart within the past 12 months.

Exclusion Criteria:

1. Detectable pre-existing antibodies to the adeno-associated virus 5 (AAV5) capsid.
2. Any evidence of active infection or any immunosuppressive disorder, except for HIV infection
3. Any evidence of active infection or any immunosuppressive disorder, including HIV infection (effective as of Protocol Amendment 3)
4. Significant liver dysfunction.
5. Prior liver biopsy showing significant fibrosis.
6. Evidence of any bleeding disorder not related to hemophilia A.
7. Platelet count of \< 100 x 10\^9/L.
8. Creatinine ≥ 1.5 mg/dL.
9. Liver cirrhosis of any etiology as assessed by liver ultrasound.
10. Chronic or active hepatitis B.
11. Active Hepatitis C.
12. Active malignancy, except non-melanoma skin cancer.
13. History of hepatic malignancy.
14. History of arterial or venous thromboembolic events.
15. Known inherited or acquired thrombophilia, including conditions associated with increased thromboembolic risk, such as atrial fibrillation.