Clinical trial NCT02615847: Clinical Trial to Study the Safety and Tolerability of Memantin Mepha® in Sickle Cell Disease Patients
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Sickle Cell Disease
Lead sponsor: University of Zurich
Sponsor class: OTHER
First posted: 2015-11-26
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Number of participants with treatment related adverse events as assessed by CTCAE version 4.0
timeFrame: 14 month
Secondary outcomes:
measure: Haemoglobin in g/l
timeFrame: 38 month
measure: Erythrocytes in T/l
timeFrame: 38 month
measure: Reticulocytes in G/l
timeFrame: 38 month
measure: Haptoglobin in g/l
timeFrame: 38 month
measure: Billirubin in micmol/l
timeFrame: 38 month
measure: LDH (Lactat dehydrogenase) in U/l
timeFrame: 38 month
measure: Ferritin in micg/l
timeFrame: 38 month
measure: Transferrin saturation in %
timeFrame: 38 month
measure: Number of days in hospital
timeFrame: 38 month
measure: Number of days with inability to work
timeFrame: 38 month
measure: Number of transfusions
timeFrame: 38 month
measure: Number of days taken antibiotics
timeFrame: 38 month
measure: Amount of pain medication in mg or ml
timeFrame: 38 month
measure: Pain scale
timeFrame: 38 month
Other outcomes:
Locations:
city: Zürich
country: Switzerland
facility: University Hospital Zürich
Study officials:
affiliation: Universitätsspital Zürich
name: Jeroen Goede, MD
role: STUDY_CHAIR
References: