Hemofili B Gen Terapisi İlacının Güvenliği ve Etkinliğinin Değerlendirilmesi

Sade dilde özet

Bu çalışma, hemofili B (hemophilia B) hastalığında BBM-H901 adlı bir gen terapisini tek doz damar içi enjeksiyon şeklinde inceleyerek güvenliğini ve etkinliğini değerlendirmektedir. Çalışmaya, düşük düzeyde FIX pıhtılaşma faktörü aktivitesine sahip, 18 yaş ve üzeri erkek hastalar alınmıştır. Faz 2/Faz 3 aşamasında yürütülmekte; şu an katılımcı almamakta ancak devam etmektedir (active, not recruiting).

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

Deney bilgileri

Faz

PHASE2/PHASE3

Durum

Devam ediyor, katılım kapalı

Sponsor

Shanghai Xinzhi BioMed Co., Ltd.

Müdahale

Single dose intravenous injection of BBM-H901

Başlangıç

30.12.2021

Tahmini bitiş

30.06.2028

Ülkeler

China

İlgili genler

Merkezler

• Hefei, China
• Beijing, China
• Guangzhou, China
• Shenzhen, China
• Tangshan, China
• Zhengzhou, China
• Taiyuan, China
• Tianjin, China
• Kunming, China

Uygunluk kriterleri

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

Inclusion Criteria of Phase 1/2/3:

1. Males ≥ 18 years of age;
2. Have hemophilia B with ≤2 IU/dL (≤2 %) endogenous FIX activity levels;
3. Have had ≥100 prior exposure days (EDs) to any recombinant and/or plasma-derived FIX protein products based on historical data from the subjects' records/histories;
4. Have had bleeding events and/or injected with FIX protein products (including recombination and plasma source) during the last 12 weeks documented in the subjects' medical records;
5. Have no prior history of hypersensitivity or anaphylaxis associated with any FIX or IV immunoglobulin administration;
6. Agree to use a reliable barrier contraception method from the beginning of signing the informed consent to 52 weeks after administration.

Exclusion Criteria of Phase 1/2/3:

1. Being positive for hepatitis B surface antigen (HBsAg) or hepatitis B virus-DNA (HBV-DNA). Being positive for hepatitis C virus antibody (HCV-Ab) or hepatitis C virus RNA (HCV-RNA). Subjects with medical history of hepatitis B or C can be regarded as negative only when 2 required samplings are conducted at least 3 months apart and both test results of indicators aforementioned are negative, i.e. subjects with natural clearance and anti-viral therapy clearance for hepatitis B or C are eligible;
2. Have potential liver diseases, such as previous diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy or liver fibrosis (fibrosis stage ≥ 3); nodules or cysts were found by B ultrasound, or elevated alpha-fetoprotein was detected by laboratory tests. Subjects who are not eligible for the study if the abnormalities are clinically significant regarding to the medical judgement of the investigator;
3. HIV positive patients;
4. Have participated in a previous gene therapy research trial before screening, or in a clinical study with an investigational drug within 5 half-life of the investigational product, whichever is longer;
5. Have alcohol or drug dependence, or cannot stop drinking throughout the study;
6. Any concurrent clinically significant major disease or condition that the investigator deems unsuitable for participation in the study.