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.
Visão geral do estudo
Status
Condições
Intervenção / Tratamento
Descrição detalhada
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.
Tipo de estudo
Inscrição (Real)
Estágio
- Fase 2
Contactos e Locais
Locais de estudo
-
-
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Zürich, Suíça, 8091
- University Hospital Zurich
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Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Descrição
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
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: N / D
- Modelo Intervencional: Atribuição de grupo único
- Mascaramento: Nenhum (rótulo aberto)
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
|
Experimental: 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.
Outros nomes:
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O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Prazo |
|---|---|
|
Number of participants with treatment related adverse events as assessed by CTCAE version 4.0
Prazo: 14 month
|
14 month
|
Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Haemoglobin in g/l
Prazo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Erythrocytes in T/l
Prazo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Reticulocytes in G/l
Prazo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Haptoglobin in g/l
Prazo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Billirubin in micmol/l
Prazo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
LDH (Lactat dehydrogenase) in U/l
Prazo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Ferritin in micg/l
Prazo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Transferrin saturation in %
Prazo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Number of days in hospital
Prazo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Number of days with inability to work
Prazo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Number of transfusions
Prazo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Number of days taken antibiotics
Prazo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Amount of pain medication in mg or ml
Prazo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Pain scale
Prazo: 38 month
|
Scores range from 0 (no pain) to 10 (worst possible pain).
|
38 month
|
Colaboradores e Investigadores
Patrocinador
Investigadores
- Cadeira de estudo: Jeroen Goede, MD, UniversitätsSpital Zürich
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo
Conclusão Primária (Real)
Conclusão do estudo (Real)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Estimativa)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- HbSS-MemSID
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