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.
調査の概要
詳細な説明
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.
研究の種類
入学 (実際)
段階
- フェーズ2
連絡先と場所
研究場所
-
-
-
Zürich、スイス、8091
- University Hospital Zurich
-
-
参加基準
適格基準
就学可能な年齢
健康ボランティアの受け入れ
受講資格のある性別
説明
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
研究計画
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：処理
- 割り当て：なし
- 介入モデル：単一グループの割り当て
- マスキング：なし（オープンラベル）
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
実験的：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.
他の名前：
|
この研究は何を測定していますか？
主要な結果の測定
結果測定 |
時間枠 |
|---|---|
|
Number of participants with treatment related adverse events as assessed by CTCAE version 4.0
時間枠：14 month
|
14 month
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Haemoglobin in g/l
時間枠：38 month
|
2 years before screening until end of study.
|
38 month
|
|
Erythrocytes in T/l
時間枠：38 month
|
2 years before screening until end of study.
|
38 month
|
|
Reticulocytes in G/l
時間枠：38 month
|
2 years before screening until end of study.
|
38 month
|
|
Haptoglobin in g/l
時間枠：38 month
|
2 years before screening until end of study.
|
38 month
|
|
Billirubin in micmol/l
時間枠：38 month
|
2 years before screening until end of study.
|
38 month
|
|
LDH (Lactat dehydrogenase) in U/l
時間枠：38 month
|
2 years before screening until end of study.
|
38 month
|
|
Ferritin in micg/l
時間枠：38 month
|
2 years before screening until end of study.
|
38 month
|
|
Transferrin saturation in %
時間枠：38 month
|
2 years before screening until end of study.
|
38 month
|
|
Number of days in hospital
時間枠：38 month
|
2 years before screening until end of study.
|
38 month
|
|
Number of days with inability to work
時間枠：38 month
|
2 years before screening until end of study.
|
38 month
|
|
Number of transfusions
時間枠：38 month
|
2 years before screening until end of study.
|
38 month
|
|
Number of days taken antibiotics
時間枠：38 month
|
2 years before screening until end of study.
|
38 month
|
|
Amount of pain medication in mg or ml
時間枠：38 month
|
2 years before screening until end of study.
|
38 month
|
|
Pain scale
時間枠：38 month
|
Scores range from 0 (no pain) to 10 (worst possible pain).
|
38 month
|
協力者と研究者
スポンサー
捜査官
- スタディチェア：Jeroen Goede, MD、Universitätsspital Zürich
研究記録日
主要日程の研究
研究開始
一次修了 (実際)
研究の完了 (実際)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (見積もり)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- HbSS-MemSID
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