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.
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Description détaillée
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.
Type d'étude
Inscription (Réel)
Phase
- Phase 2
Contacts et emplacements
Lieux d'étude
-
-
-
Zürich, Suisse, 8091
- University Hospital Zurich
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-
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
Accepte les volontaires sains
Sexes éligibles pour l'étude
La description
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 d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: N / A
- Modèle interventionnel: Affectation à un seul groupe
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
|---|---|
|
Expérimental: 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.
Autres noms:
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Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Délai |
|---|---|
|
Number of participants with treatment related adverse events as assessed by CTCAE version 4.0
Délai: 14 month
|
14 month
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Haemoglobin in g/l
Délai: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Erythrocytes in T/l
Délai: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Reticulocytes in G/l
Délai: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Haptoglobin in g/l
Délai: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Billirubin in micmol/l
Délai: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
LDH (Lactat dehydrogenase) in U/l
Délai: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Ferritin in micg/l
Délai: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Transferrin saturation in %
Délai: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Number of days in hospital
Délai: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Number of days with inability to work
Délai: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Number of transfusions
Délai: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Number of days taken antibiotics
Délai: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Amount of pain medication in mg or ml
Délai: 38 month
|
2 years before screening until end of study.
|
38 month
|
|
Pain scale
Délai: 38 month
|
Scores range from 0 (no pain) to 10 (worst possible pain).
|
38 month
|
Collaborateurs et enquêteurs
Parrainer
Les enquêteurs
- Chaise d'étude: Jeroen Goede, MD, UniversitätsSpital Zürich
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude
Achèvement primaire (Réel)
Achèvement de l'étude (Réel)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Estimation)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- HbSS-MemSID
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