Clinical Trial to Study the Safety and Tolerability of Memantin Mepha® in Sickle Cell Disease Patients (MemSID)
A Phase II, Open Label, Single Center Trial to Study the Safety and Tolerability of Memantin Mepha® as Supportive Long Term Treatment in Symptomatic Sickle Cell Disease
Symptomatic sickle cell disease is worldwide the most frequent cause for hereditary hemolytic anemia with recurrent pain crisis. Hemolysis, vaso- occlusive and pain crises are hallmarks of this disease and are causative for an important socio-economic burden worldwide, especially in Africa.
Aside from allogenic stem cell transplantation, which is rarely available and very expensive, at present there is no curative treatment for patients with sickle cell disease (SCD). The current standard of care includes treatment with hydroxycarbamide and symptomatic care such as transfusions, antibiotic/analgesic treatment.
This study has the aim to study the safety and tolerability of Memantin in patients with sickle cell disease.
研究概览
详细说明
During the study participants will be asked if medical occurrences (AEs) happened and laboratory analysis (haematology, coagulation, chemistry) and urine samples will be taken. In addition at each visit a physical examination and measurement of vital signs will be performed.
In addition the number of hospital days and emergency consultations, the impact on working ability (the number of days with inability to work, changes in iv%), the amount and type of analgesic medication, the amount of RBC transfusions, the number of days that antibiotics prescribed and haematology (at local and external laboratory) and chemistry laboratory parameters will be assessed. For the impact on work and social life a questionnaire of quality of life will be filled out monthly by the patient.
At screening and at the end of the study SCD specific assessments will be performed, which include cardiologic examination (ECG, ECHO), abdominal sonography, ophthalmological examination, lung function testing and neuroangiologic examination.
研究类型
注册 (实际的)
阶段
- 阶段2
联系人和位置
学习地点
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-
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Zürich、瑞士、8091
- University Hospital Zurich
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参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Inclusion Criteria:
Documented symptomatic sickle cell disease (HbSS or HbS/beta thalassemia)
- Age 18 years or older
- Able and willing to provide written informed consent and to comply with the study protocol procedures
- Willing to use two effective methods of contraception during study treatment until 6 months after stop of study treatment. Effective contraception methods are considered oral, injectable, implantative contraceptives or intrauterine contraceptive devices combined with the use of condom.
Exclusion Criteria:
History of transfusion during last three months before Screening
- Patients with active bacterial, viral or fungal infection requiring systemic treatment
- Patients with known infection with human immunodeficiency virus (HIV) of human T cell leukaemia virus 1 (HTLV-1)
- Inadequate renal function: creatinine clearance < 30ml/min
- Inadequate liver function: NCICTC Grade 3 liver function tests (AST, ALT > 5x upper limit of normal (ULN))
- History of malignancy
- Women who are pregnant or breast feeding
- Previous known mental disorder or known family history of psychiatric diseases
- Known epileptic disease
- The receipt of any investigational product within 30 days prior to this trial
学习计划
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：不适用
- 介入模型：单组作业
- 屏蔽：无（打开标签）
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
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实验性的：Memantin arm
Memantinhydrochlorid, administered once per day during 12 month.
Dosage is from 0-20 mg.
|
Memantin Mepha® Lactabs (memantinehydrochlorid) will be provided as 5 mg, 10 mg, 15 mg and 20 mg tablets, packed in blister. The study drug will be taken once a day per os, during 12 month.
其他名称：
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研究衡量的是什么？
主要结果指标
结果测量 |
大体时间 |
|---|---|
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Number of participants with treatment related adverse events as assessed by CTCAE version 4.0
大体时间：14 month
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14 month
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Haemoglobin in g/l
大体时间：38 month
|
2 years before screening until end of study.
|
38 month
|
|
Erythrocytes in T/l
大体时间：38 month
|
2 years before screening until end of study.
|
38 month
|
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Reticulocytes in G/l
大体时间：38 month
|
2 years before screening until end of study.
|
38 month
|
|
Haptoglobin in g/l
大体时间：38 month
|
2 years before screening until end of study.
|
38 month
|
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Billirubin in micmol/l
大体时间：38 month
|
2 years before screening until end of study.
|
38 month
|
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LDH (Lactat dehydrogenase) in U/l
大体时间：38 month
|
2 years before screening until end of study.
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38 month
|
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Ferritin in micg/l
大体时间：38 month
|
2 years before screening until end of study.
|
38 month
|
|
Transferrin saturation in %
大体时间：38 month
|
2 years before screening until end of study.
|
38 month
|
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Number of days in hospital
大体时间：38 month
|
2 years before screening until end of study.
|
38 month
|
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Number of days with inability to work
大体时间：38 month
|
2 years before screening until end of study.
|
38 month
|
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Number of transfusions
大体时间：38 month
|
2 years before screening until end of study.
|
38 month
|
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Number of days taken antibiotics
大体时间：38 month
|
2 years before screening until end of study.
|
38 month
|
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Amount of pain medication in mg or ml
大体时间：38 month
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2 years before screening until end of study.
|
38 month
|
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Pain scale
大体时间：38 month
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Scores range from 0 (no pain) to 10 (worst possible pain).
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38 month
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合作者和调查者
调查人员
- 学习椅：Jeroen Goede, MD、Universitätsspital Zürich
研究记录日期
研究主要日期
学习开始
初级完成 (实际的)
研究完成 (实际的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (估计)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
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