Clinical Trial to Study the Safety and Tolerability of Memantin Mepha® in Sickle Cell Disease Patients

Phase 2

Completed

Brief Summary: 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.

Detailed Description: 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.

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
Memantin arm	Memantinhydrochlorid	Memantinhydrochlorid, administered once per day during 12 month. Dosage is from 0-20 mg.

Primary Outcome Measures

Name	Time	Method
Number of participants with treatment related adverse events as assessed by CTCAE version 4.0	14 month

Secondary Outcome Measures

Name	Time	Method
Haptoglobin in g/l	38 month	2 years before screening until end of study.
Billirubin in micmol/l	38 month	2 years before screening until end of study.
Haemoglobin in g/l	38 month	2 years before screening until end of study.
Ferritin in micg/l	38 month	2 years before screening until end of study.
Erythrocytes in T/l	38 month	2 years before screening until end of study.
Reticulocytes in G/l	38 month	2 years before screening until end of study.
Amount of pain medication in mg or ml	38 month	2 years before screening until end of study.
LDH (Lactat dehydrogenase) in U/l	38 month	2 years before screening until end of study.
Number of days taken antibiotics	38 month	2 years before screening until end of study.
Transferrin saturation in %	38 month	2 years before screening until end of study.
Number of days in hospital	38 month	2 years before screening until end of study.
Pain scale	38 month	Scores range from 0 (no pain) to 10 (worst possible pain).
Number of days with inability to work	38 month	2 years before screening until end of study.
Number of transfusions	38 month	2 years before screening until end of study.