Plasma Exchange With Albumin in AMN Patients
关键词
抽象
日期
| 最后验证: | 02/29/2020 |
| 首次提交: | 03/05/2020 |
| 提交的预估入学人数: | 03/09/2020 |
| 首次发布: | 03/10/2020 |
| 上次提交的更新: | 03/09/2020 |
| 最近更新发布: | 03/10/2020 |
| 实际学习开始日期: | 03/08/2020 |
| 预计主要完成日期: | 05/30/2021 |
| 预计完成日期: | 05/30/2021 |
状况或疾病
干预/治疗
Drug: Patients
相
手臂组
| 臂 | 干预/治疗 |
|---|---|
| Experimental: Patients Patients before and after the treatment | Drug: Patients plasma exchange with albumin, one per week for one month, then one per month for 5 months |
资格标准
| 有资格学习的年龄 | 18 Years 至 18 Years |
| 有资格学习的性别 | Male |
| 接受健康志愿者 | 是 |
| 标准 | Inclusion Criteria: 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 the 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 Exclusion Criteria: 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 |
结果
主要结果指标
1. Concentration of very long chain fatty acids [Change from baseline at 6 months]
次要成果指标
1. 2 Minute Walk Test [Months 0, 6 and 12]
2. 6 Minute Walk Test [Months 0, 6 and 12]
3. Timed Up and Go (TUG) test [Months 0, 6 and 12]
4. Time to walk 25 Feet (TW25) [Months 0, 6 and 12]
5. Expanded disability status scale (EDSS) [Months 0, 6 and 12]
6. Ashworth scale [Months 0, 6 and 12]
7. SF-Qualiveen (Short-form Qualiveen) [Months 0, 6 and 12]
