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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Memantin arm | Experimental | Memantinhydrochlorid, administered once per day during 12 month. Dosage is from 0-20 mg. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Memantinhydrochlorid | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with treatment related adverse events as assessed by CTCAE version 4.0 | 14 month |
| Measure | Description | Time Frame |
|---|---|---|
| Haemoglobin in g/l | 2 years before screening until end of study. | 38 month |
| Erythrocytes in T/l | 2 years before screening until end of study. |
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Inclusion Criteria:
Documented symptomatic sickle cell disease (HbSS or HbS/beta thalassemia)
Exclusion Criteria:
History of transfusion during last three months before Screening
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| Name | Affiliation | Role |
|---|---|---|
| Jeroen Goede, MD | Universitätsspital Zürich | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University Hospital Zürich | Zurich | 8091 | Switzerland |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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|
| 38 month |
| Reticulocytes in G/l | 2 years before screening until end of study. | 38 month |
| Haptoglobin in g/l | 2 years before screening until end of study. | 38 month |
| Billirubin in micmol/l | 2 years before screening until end of study. | 38 month |
| LDH (Lactat dehydrogenase) in U/l | 2 years before screening until end of study. | 38 month |
| Ferritin in micg/l | 2 years before screening until end of study. | 38 month |
| Transferrin saturation in % | 2 years before screening until end of study. | 38 month |
| Number of days in hospital | 2 years before screening until end of study. | 38 month |
| Number of days with inability to work | 2 years before screening until end of study. | 38 month |
| Number of transfusions | 2 years before screening until end of study. | 38 month |
| Number of days taken antibiotics | 2 years before screening until end of study. | 38 month |
| Amount of pain medication in mg or ml | 2 years before screening until end of study. | 38 month |
| Pain scale | Scores range from 0 (no pain) to 10 (worst possible pain). | 38 month |
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |