Clinical Trial to Study the Safety and Tolerability of Memantin Mepha® in Sickle Cell Disease Patients (MemSID)
Primary Purpose
Sickle Cell Disease
Status
Completed
Phase
Phase 2
Locations
Switzerland
Study Type
Interventional
Intervention
Memantinhydrochlorid
Sponsored by
About this trial
This is an interventional treatment trial for Sickle Cell Disease
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
Sites / Locations
- University Hospital Zürich
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Memantin arm
Arm Description
Memantinhydrochlorid, administered once per day during 12 month. Dosage is from 0-20 mg.
Outcomes
Primary Outcome Measures
Number of participants with treatment related adverse events as assessed by CTCAE version 4.0
Secondary Outcome Measures
Haemoglobin in g/l
2 years before screening until end of study.
Erythrocytes in T/l
2 years before screening until end of study.
Reticulocytes in G/l
2 years before screening until end of study.
Haptoglobin in g/l
2 years before screening until end of study.
Billirubin in micmol/l
2 years before screening until end of study.
LDH (Lactat dehydrogenase) in U/l
2 years before screening until end of study.
Ferritin in micg/l
2 years before screening until end of study.
Transferrin saturation in %
2 years before screening until end of study.
Number of days in hospital
2 years before screening until end of study.
Number of days with inability to work
2 years before screening until end of study.
Number of transfusions
2 years before screening until end of study.
Number of days taken antibiotics
2 years before screening until end of study.
Amount of pain medication in mg or ml
2 years before screening until end of study.
Pain scale
Scores range from 0 (no pain) to 10 (worst possible pain).
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02615847
Brief Title
Clinical Trial to Study the Safety and Tolerability of Memantin Mepha® in Sickle Cell Disease Patients
Acronym
MemSID
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
2. Study Status
Record Verification Date
July 2017
Overall Recruitment Status
Completed
Study Start Date
August 2015 (undefined)
Primary Completion Date
March 31, 2017 (Actual)
Study Completion Date
July 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Zurich
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
9 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Memantin arm
Arm Type
Experimental
Arm Description
Memantinhydrochlorid, administered once per day during 12 month. Dosage is from 0-20 mg.
Intervention Type
Drug
Intervention Name(s)
Memantinhydrochlorid
Other Intervention Name(s)
Memantin Mepha
Intervention Description
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.
Primary Outcome Measure Information:
Title
Number of participants with treatment related adverse events as assessed by CTCAE version 4.0
Time Frame
14 month
Secondary Outcome Measure Information:
Title
Haemoglobin in g/l
Description
2 years before screening until end of study.
Time Frame
38 month
Title
Erythrocytes in T/l
Description
2 years before screening until end of study.
Time Frame
38 month
Title
Reticulocytes in G/l
Description
2 years before screening until end of study.
Time Frame
38 month
Title
Haptoglobin in g/l
Description
2 years before screening until end of study.
Time Frame
38 month
Title
Billirubin in micmol/l
Description
2 years before screening until end of study.
Time Frame
38 month
Title
LDH (Lactat dehydrogenase) in U/l
Description
2 years before screening until end of study.
Time Frame
38 month
Title
Ferritin in micg/l
Description
2 years before screening until end of study.
Time Frame
38 month
Title
Transferrin saturation in %
Description
2 years before screening until end of study.
Time Frame
38 month
Title
Number of days in hospital
Description
2 years before screening until end of study.
Time Frame
38 month
Title
Number of days with inability to work
Description
2 years before screening until end of study.
Time Frame
38 month
Title
Number of transfusions
Description
2 years before screening until end of study.
Time Frame
38 month
Title
Number of days taken antibiotics
Description
2 years before screening until end of study.
Time Frame
38 month
Title
Amount of pain medication in mg or ml
Description
2 years before screening until end of study.
Time Frame
38 month
Title
Pain scale
Description
Scores range from 0 (no pain) to 10 (worst possible pain).
Time Frame
38 month
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jeroen Goede, MD
Organizational Affiliation
Universitätsspital Zürich
Official's Role
Study Chair
Facility Information:
Facility Name
University Hospital Zürich
City
Zürich
ZIP/Postal Code
8091
Country
Switzerland
12. IPD Sharing Statement
Learn more about this trial
Clinical Trial to Study the Safety and Tolerability of Memantin Mepha® in Sickle Cell Disease Patients
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