NL-OMON54928尚未招募不适用
Screening and Lead-in Observational Protocol to Determine Potential Patient Eligibility for Inclusion in AAV Gene Therapy Clinical Trials in Haemophilia B - ECLIPSE
Freeline Therapeutics0 个研究点目标入组 6 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 6
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- 12 至 99（—）
入选标准
- •1) Male participants >= 16 years of age.
- •2) Able to give full informed consent/assent (according to local regulations)
- •and/or obtain full informed consent from the participant*s legally acceptable
- •representative (as appropriate) and able to understand and comply with all
- •requirements of the study, including diary completion.
- •3) Interested in participation in future gene therapy clinical studies.
- •4) Subjects with Haemophilia B with known severe or moderately severe FIX
- •deficiency (<=2% of normal circulating FIX activity) for which the subject is
- •a) Continuous routine FIX prophylaxis*, OR
- •b) On demand FIX treatment*
- •5) If receiving prophylaxis, participant has been on stable** and adequate¶
- •prophylaxis for at least 2 months prior to enrolment
- •*Continuous routine prophylaxis is defined as the intent of treating with an a
- •priori defined frequency of infusions (e.g., twice weekly, once every two
- •weeks, etc.) as documented in the medical records3.
- •* Enrollment of on demand patients is competitive up to a maximum of 10
- •** Stable denotes subject has been on prophylaxis with no change in either the
- •FIX product used, the dose administered or the regimen in the 2 months prior to
- •¶ Adequate prophylaxis for purposes of this protocol means an annualised
- •bleeding rate (ABR) of <=8 in the year preceding enrolment.
排除标准
- •1) Documented evidence of liver fibrosis and/or liver dysfunction (including,
- •but not limited to, persistently elevated alanine aminotransaminase, aspartate
- •aminotransferase, and/or billirubin > 1.5 x upper limit of normal).
- •2) Prior treatment with a gene transfer medicinal product.
- •3) Known presence or history of neutralising anti-human FIX antibodies
- •(inhibitors).
- •4) Previously established serological evidence of HIV-1.
- •5) Documented 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.
- •6) Participants at high risk of thromboembolic events (history of arterial or
- •venous thromboembolism [e.g. deep vein thrombosis, pulmonary embolism,
- •non-haemorrhagic stroke, arterial embolus] and those with acquired
- •thrombophilia. Participants with a history of atrial fibrillation).
- •7) Known coagulation disorder other than Haemophilia B.
- •8) Known history of an allergic reaction or anaphylaxis to Factor IX products
- •or known uncontrolled allergic conditions.
- •9) Known history of allergy to corticosteroids or to tacrolimus or any other
- •10) Known medical condition that would require chronic administration of
- •corticosteroids (excluding topical formulations).
- •11) History of alcohol or drug dependence.
- •12) Planned surgical procedure within the next 12 months requiring prophylactic
- •FIX treatment.
- •13) Known active severe infection (including documented COVID-19 infection), or
- •any other significant concurrent, uncontrolled medical condition evaluated by
- •the investigator to interfere with adherence to the protocol procedures or with
- •tolerance to gene therapy in a future treatment study including, but not
- •limited to, renal, hepatic, cardiovascular, ophthalmological, haematological,
- •immunological, gastrointestinal, endocrine, pulmonary, neurological, cerebral
- •or psychiatric disease, malignancy or any other psychological disorder.
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