cerebellar gait disorder, cerebellar ataxia, sensorimotor adaption and ocular motor disorders, postural sway, dysarthria MedDRA version: 19.0 Level: PT Classification code 10008025 Term: Cerebellar ataxia System Organ Class: 10029205 - Nervous system disorders
Conditions
Sponsors
Hospital of the University of Munich, Grosshadern
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects will only be included in the study if they meet all of the following criteria: 1. Patients male or female, aged between 18 and 80 years 2. Clinical evaluated diagnosis of cerebellar ataxia with at least 2 points in the scale for the assessment and rating of ataxia (SARA) 3. Written informed consent of the subject 4. Subjects, with the ability to follow study instructions and likely to attend and complete all required visits (Compliance). Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Subjects will not be included in the study if any of the following criteria applies: 1. Weight of 40 kg or less 2. Pregnancy or breast-feeding 3. Concurrend treatment with inhibitors of organic cation transporter 2 (OCT2), e.g. cimetidine 4. Cardiovascular diseases e.g. recent heart attack (within the last 3 months), cardiac arrhythmia (QTc interval > 500 ms, atrial fibrillation, AV block grade = II), unstable angina pectoris (chest tightness due to impaired blood flow to the heart), severe heart failure (NYHA class IV) 5. Recently occurred stroke (within the last 3 months) 6. Epileptic seizure currently or in the past 7. Severe arterial hypertension (grade III according to the guidelines of the German society of cardiology, 2008) 8. Liver insufficiency (e.g. cirrhosis of the liver) 9. Asthma (severity = grade III) 10. Milde or severe renal failure (Creatinine Clearance = 80ml/min) 11. Unadjusted thyroid dysfunction 12. Acute gastric and intestinal ulcer 13. Other acute, serious illness of the subject 14. Subject is unable to understand the nature, scope, significance and consequences of this clinical trial. 15. Subject is unable to comply with the study design 16. Previous participation in this clinical trial or simultaneous participation in another clinical trial or participation in any clinical trial involving an investigational medicinal product within 30 days prior to written informed consent for this clinical trial Note to Exclusion Criterion 10. Mild or severe renal failure (Creatinine Clearance = 80ml/min): If Creatinine Clearance at Screening Visit is > 80ml/min a 24 hour urin sample will be taken (collection start at day of Screening Visit) for re-assessment of Creatinine Clearance.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Primary objective: 1) To demonstrate the effectiveness of sustained released 4-aminopyridine regarding the reduction of the gait variability (improvement of gait) at maximum walking speed compared to placebo. 2) To demonstrate the effectiveness of sustained released 4-aminopyridine regarding the increase of individual preferred walking speed compared to placebo. ;Secondary Objective: Secondary objective: 1) Comparison of the gait variability at maximum walking speed at the end of the 12-week treatment phase under sustained released 4-aminopyridine or placebo (long term effect). 2) Analysis of the difference in the (relative) change of the individual preferred walking speed at the end of the 12-week treatment phase versus pre-treatment (baseline) under sustained released 4-aminopyridine or placebo (long term effect). 3) Quantitative description or comparison of the changes in various ataxia-, mobility- and QoL-scores within the two treatment groups compared to baseline value (after 14 days, 12 weeks, or follow-up visit). 4) Comparison of the number of falls under sustained released 4-aminopyridine or placebo. 5) Study the frequency of (S)AEs under sustained released 4-aminopyridine compared to placebo. ;Primary end point(s): Primary end point: 1) Logarithmized gait variability at maximum walking speed (CVmax [%]) 2) Logarithmized individual preferred walking speed (G_pref) ;Timepoint(s) of evaluation of this end point: 14 days after beginnig of a 12-week treatment phase (Visit 2 and Visit 5) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary end point: 1) Logarithmized gait variability at maximum walking speed 2) Change of the logarithmized individual preferred walking speed [m/sec] 3) Number of falls analysed via a standarized fall diary 4) Changes in ataxia-score (SCAFI) and in mobility- and QoL-scores (FES, ABC, BDI-II, EQ-5D-5L, FSS) ;Timepoint(s) of evaluation of this end point: Regarding end points 1 & 2: At the end of each 12-week treatment phase (Visit 3 and Visit 6) and after an additional 1-month follow-up period. Regarding end points 3: Messured during each 12-week treatment phase and after an additional 1-month follow-up period. Regarding end points 4: 14 days after beginning of each treatment phase (Visit 2 and Visit 5) or rather at the end of each 12-week treatment phase (Visit 3 and Visit 6) and after an additional 1-month follow-up period. | — |
Countries
Germany
Contacts
Public ContactStudienzentrale DSGZ
Hospital of the University of Munich
Outcome results
None listed