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.

Study Overview

• Status: Completed
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Drug: Memantinhydrochlorid

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.

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Phase 2

Contacts and Locations

Study Locations

• Switzerland
  • Zürich, Switzerland, 8091
    • University Hospital Zurich

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Memantin arm; Memantinhydrochlorid, administered once per day during 12 month. Dosage is from 0-20 mg.	Drug: Memantinhydrochlorid; 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.; Other Names: Memantin Mepha

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of participants with treatment related adverse events as assessed by CTCAE version 4.0	14 month

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: University of Zurich
• Investigators: Study Chair: Jeroen Goede, MD, Universitatsspital Zurich

Study record dates

Study Major Dates

• Study Start: August 1, 2015
• Primary Completion (Actual): March 31, 2017
• Study Completion (Actual): July 1, 2017

Study Registration Dates

• First Submitted: September 7, 2015
• First Submitted That Met QC Criteria: November 24, 2015
• First Posted (Estimate): November 26, 2015

Study Record Updates

• Last Update Posted (Actual): August 7, 2017
• Last Update Submitted That Met QC Criteria: August 4, 2017
• Last Verified: July 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: HbSS-MemSID