EUCTR2017-000852-24-IT进行中（未招募）1 期
A Phase I/II, Open label, Multicentre, Ascending Single Dose, Safety Study of a Novel Adeno-associated Viral Vector (FLT180a) in Patients With Haemophilia B - A Factor IX Gene Therapy Study (FIX-GT)
IVERSITY COLLEGE LONDO0 个研究点目标入组 24 人开始时间: 2021年1月18日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 24
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- Male
入选标准
- •1.Adults males, = 18 years of age;
- •2.Confirmed diagnosis of HB defined as one of the following:
- •(a)Documented severe FIX deficiency with plasma FIX activity of <1% of normal or
- •(b)moderately severe FIX deficiency with plasma FIX activity level between =1% and =2% and a severe bleeding phenotype defined by one of the following:
- •(i) On prophylaxis for a history of bleeding, or
- •(ii) On-demand therapy with a history of 4 or more bleeding episodes/year on average over the past 3 years, or
- •(iii) Evidence of chronic haemophilic arthropathy (pain, joint destruction, and loss of range of motion).
- •3.Able to give full informed consent and able to comply with all requirements of the trial including 15-year long-term follow-up;
- •4.Willing to practice barrier contraception until at least two consecutive semen samples after vector administration are negative for vector sequences;
- •5.Lack of neutralising anti-AAV-S3 antibodies using an in-vivo transduction inhibition assay within 4 weeks of vector administration;
- •6.At least 150 exposure days to FIX concentrates.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 16
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 8
排除标准
- •1.Presence of neutralising anti-human FIX antibodies (inhibitor, determined by the
- •Bethesda inhibitor assay) at the time of enrolment or a previous history of FIX
- •2.Patients at high risk of thromboembolic events (high risk patients would include those
- •with a history of arterial or venous thromboembolism (e.g. deep vein thrombosis,
- •pulmonary embolism, non-haemorrhagic stroke, arterial embolus) and those with acquired
- •thrombophilia including conditions such as atrial fibrilation);
- •3.Use of investigational therapy for haemophilia within 30 days before enrolment;
- •4.Patients with active hepatitis B or C, and HBsAg or HCV RNA viral load positivity,
- •respectively, or currently on antiviral therapy for hepatitis B or C. (Negative viral
- •assays in 2 samples, collected at least 6 months apart, will be required to be
- •considered negative. Both natural clearers and those who have cleared HCV on antiviral
- •therapy are eligible.);
- •5. Serological evidence of HIV-1
- •6.Evidence of liver dysfunction (persistently elevated alanine aminotransaminase,
- •aspartate aminotransferase, bilirubin >1.5 x upper limit of normal);
- •7.Platelet count <50x109/L;
- •8.Uncontrolled glaucoma, diabetes mellitus, or hypertension;
- •9.Malignancy requiring treatment;
- •10.Patients with uncontrolled cardiac failure or unstable angina or myocardial infaction
- •in the past 6 months;
- •11.Poor performance status (World Health Organization score >1);
- •12.Prior treatment with any gene transfer medicinal product;
- •13.Known or suspected intolerance hypersensitivity or contraindicationto the
- •investigational product and non-investigational product or their excipients.
- •14.Planned major elective surgery prior to the end of trial.
- •15. Current or relevant history of a physical of psychiatric illness or any medical
- •condition that in the opinion of the investigator could affect the patients safety or
- •interfere with the study assessments.
- •16. CMV IgG postive patients who are CMV PCR positive at screening
研究者
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