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.
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Descripción detallada
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 estudio
Inscripción (Actual)
Fase
- Fase 2
Contactos y Ubicaciones
Ubicaciones de estudio
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Zürich, Suiza, 8091
- University Hospital Zurich
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Descripción
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
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: N / A
- Modelo Intervencionista: Asignación de un solo grupo
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
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Experimental: Memantin arm
Memantinhydrochlorid, administered once per day during 12 month.
Dosage is from 0-20 mg.
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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.
Otros nombres:
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Periodo de tiempo |
|---|---|
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Number of participants with treatment related adverse events as assessed by CTCAE version 4.0
Periodo de tiempo: 14 month
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14 month
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Haemoglobin in g/l
Periodo de tiempo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Erythrocytes in T/l
Periodo de tiempo: 38 month
|
2 years before screening until end of study.
|
38 month
|
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Reticulocytes in G/l
Periodo de tiempo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Haptoglobin in g/l
Periodo de tiempo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Billirubin in micmol/l
Periodo de tiempo: 38 month
|
2 years before screening until end of study.
|
38 month
|
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LDH (Lactat dehydrogenase) in U/l
Periodo de tiempo: 38 month
|
2 years before screening until end of study.
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38 month
|
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Ferritin in micg/l
Periodo de tiempo: 38 month
|
2 years before screening until end of study.
|
38 month
|
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Transferrin saturation in %
Periodo de tiempo: 38 month
|
2 years before screening until end of study.
|
38 month
|
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Number of days in hospital
Periodo de tiempo: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Number of days with inability to work
Periodo de tiempo: 38 month
|
2 years before screening until end of study.
|
38 month
|
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Number of transfusions
Periodo de tiempo: 38 month
|
2 years before screening until end of study.
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38 month
|
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Number of days taken antibiotics
Periodo de tiempo: 38 month
|
2 years before screening until end of study.
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38 month
|
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Amount of pain medication in mg or ml
Periodo de tiempo: 38 month
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2 years before screening until end of study.
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38 month
|
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Pain scale
Periodo de tiempo: 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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Colaboradores e Investigadores
Patrocinador
Investigadores
- Silla de estudio: Jeroen Goede, MD, UniversitätsSpital Zürich
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio
Finalización primaria (Actual)
Finalización del estudio (Actual)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Estimar)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
Otros números de identificación del estudio
- HbSS-MemSID
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Ensayos clínicos sobre Anemia drepanocítica
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Adelphi Values LLCBlueprint Medicines CorporationTerminadoLeucemia de mastocitos (LCM) | Mastocitosis Sistémica Agresiva (ASM) | SM w Asoc Clonal Hema Non-mast Cell Linage Disease (SM-AHNMD) | Mastocitosis sistémica latente (MSS) | Mastocitosis Sistémica Indolente (ISM) Subgrupo ISM Completamente ReclutadoEstados Unidos