EUCTR2019-004733-17-ES进行中（未招募）1 期
Effect of plasma exchange by albumin replacement in Adrenomyeloneuropathy: unicentric, single arm, proof of concept trial
Aurora Pujol Onofre0 个研究点目标入组 5 人开始时间: 2019年12月20日最近更新:
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 5
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. Men of 18 to 65 years old, inclusive
- •2. Elevated plasma VLCFA and gene mutation identified
- •3. Clinical signs of AMN with at least pyramidal signs in the lower limbs and difficulties to run
- •4. Presence of motor deficit according to the EDSS scale
- •5. Ability to perform the 2MWT
- •6. Normal brain MRI or brain MRI showing the following abnormalities that can be observed in AMN patients without cerebral form of X-ALD, obtained in the 6 months prior to screening:
- •abnormal hyperintensity of pyramidal tract fibers in the brain stem on FLAIR or T2 sequence
- •abnormal hyperintensity of pyramidal tract fibers in the internal capsules on FLAIR or T2 sequence
- •cerebellar atrophy
- •moderate cortical atrophy
- •7. Appropriate steroid replacement if adrenal insufficiency is present
- •8. Signed Informed consent
- •9. Affiliated to the Spanish Public Health System
- •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 5
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Any contraindication for plasma exchange due to behavioral disorders or abnormal coagulation parameters, such for example:
- •- Hypocalcemia (Ca++ < 8.7 mg/dl)
- •- Thrombocytopenia (< 100.000/µl)
- •- Fibrinogen < 1.5 g/l
- •- Prothrombin time (Quick) p< 60% versus control (INR > 1.5)
- •- Beta-blocker treatment and bradycardia < 55/min
- •- Treatment with ACIs (increased risk of allergic reactions)
- •2. Hemoglobin < 10 g/dl
- •3. Difficult venous access precluding plasma exchange
- •4. A history of frequent adverse reactions (serious or otherwise) to blood products
- •5. Hipersensibility to albumin o allergies to any of the components of Albunorm® 5%
- •6. Plasma creatine > 2 mg/dl
- •7. Uncontrolled high blood pressure (systolic blood pressure of 160 mmHg or higher and/or diastolic blood pressure of 100 mmHg or higher despite regular treatment during the last 3 months)
- •8. Liver cirrhosis or any liver problem with GPT > 2.5 x ULN, or bilirubin > 2 mg/dl
- •9. Heart diseases as evidenced by myocardial infarction, severe or unstable angina, or heart failure in the past 12 months
- •10. Gadolinium enhancement on T1 sequence of any abnormal hypersignal of white matter, including myelinated pyramidal tracts, visible at brain MRI on FLAIR sequences
- •11. Significant peripheral edema (2+ or more on the Assessment Chart for Pitting Edema) of the extremities of any etiology
- •12. Any evolutive malignancy during the last five years or any condition complicating adherence to the study protocol
- •13. Smokers (one pack/ day or more for at least 20 years), current or former
- •14. Any psychiatric disease
- •15. Present participation to another therapeutic clinical trial for X-ALD, or the receipt of any other investigational drug in the three months prior to the start of the study
- •16. Patients being treated with anticoagulants or antiplatelet therapy
- •17. Not easily contactable by the investigator in case of emergency or not capable to call the investigator
研究者
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