Sustained-release 4-aminopyridine (Fampyra®) in the treatment of cerebellar ataxia

Phase 3

• Conditions: G11.8; Other hereditary ataxias

• Registration Number: DRKS00005271
• Lead Sponsor: Klinikum der Universität München, Campus Großhadern

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-10-15
• Study Completion: 2016-10-31
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Complete
• Sex: All
• Target Recruitment: 75

Inclusion Criteria

Patients male or female, aged between 18 and 80 years

- Clinical evaluated diagnosis of cerebellar ataxia with at least 2 points in the subscore gait of the scale for the assessment and rating of ataxia (SARA)

- Written informed consent of the subject

- Subjects, with the ability to follow study instructions and likely to attend and complete all required visits

Exclusion Criteria

- Body weight 40 kg or less

- Pregnancy or breast-feeding

- Treatment with inhibitors of organic cation transporter 2 (OCT2), e.g. cimetidine

- 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, severe heart failure (NYHA class IV)

- Recently occurred stroke (within the last 3 months)

- Epileptic seizure currently or in the past

- Asthma (severity = grade III according to the Leitlinien des Programm für Nationale Versorgungsleitlinien)

- Mild or severe renal failure (Creatinine Clearance = 80ml/min)

- Unadjusted thyroid dysfunction

- Acute gastric and intestinal ulcer

- Other acute, serious illness of the subject

- Subject is unable to understand the scope, significance and consequences of this clinical trial and is unable to comply with the study design

- Previous participation in this clinical trial or participation in any clinical trial taking an investigational medicinal product within 30 days prior to participation in this clinical trial

Study & Design

• Study Type: interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
ogarithmized gait variability at maximum walking speed (CVmax [%]) 14 days after start of study treatment.<br><br><br>Change in logarithmized individual preferred walking speed (G_pref) 14 days after start of study treatment compared to baseline.

Secondary Outcome Measures

Name	Time	Method
ogarithmized gait variability at maximum walking speed at the end of each 3-month treatment period and after an additional 1-month follow-up period.<br><br>Change of the logarithmized individual preferred walking speed [m/sec] at the end of each 3-month treatment period and after an additional 1-month follow-up period.<br><br>Number of falls analysed via a standarized fall diary measured during each 3-month treatment period and after an additional 1-month follow-up period.<br><br>Changes in ataxia-score (SCAFI) 14 days after beginning of each treatment phase or at the end of each 3-month treatment period and after an additional 1-month follow-up period.<br><br><br>Changes in mobility- and QoL-scores (FES, ABC, BDI-II, EQ-5D-5L, FSS) 14 days after beginning of each treatment phase or at the end of each 3-month treatment period and after an additional 1-month follow-up period.