Comparsion of the therapy with Fampyra and Acemit in patients with episodic ataxia type 2

Phase 1

• Conditions: Familial episodic ataxia (EA) represents a genetically and phenotypically diverse group of rare autosomal dominant hereditary disorders characterized by episodes of imbalance and incoordination that are variably associated with interictal neurological signs and typically triggered by physical exertion and emotional stress. Episodic ataxia type 2 (EA2) with associated interictal nystagmus and other ocular motor abnormalities is the most common and the best characterized of all EA syndromes.; MedDRA version: 18.1Level: LLTClassification code 10067447Term: Episodic ataxiaSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2013-000107-17-DE
• Lead Sponsor: Hospital of the University of Munich, Grosshadern

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-04-05
• Last Update Posted: 31 October 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Subjects will only be randomized in the study if they meet all of the following criteria:

1. Written informed consent of the subject
2. Patients (male or female) aged 18 years or older
3. Genetically confirmed episodic ataxia type 2 or a familial history of ataxia with clinical picture of episodic ataxia type 2 starting in childhood
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

Subjects will not be randomized 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. Known hypersensitivity to aminopyridine and/or acetazolamide/sulfonamide
5. 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), severe arterial hypertension (grade III according to the guidelines of the German society of cardiology, 2008)
6. Recently occurred stroke (within the last 3 months)
7. Epileptic seizure currently or in the past
8. Asthma (severity = grade III)
9. Obstructive lung disease (e.g. pulmonary emphysema)
10. Liver insufficiency defined as GOT/GPT/total bilirubin > 3 x upper range
(e.g. cirrhosis of the liver)
11. Milde or severe renal failure (Creatinine Clearance = 80ml/min)
12. Addison's disease
13. Unadjusted thyroid dysfunction
14. Acute gastric and intestinal ulcer
15. Known hyperchloremic acidosis
16. Depressed sodium and/or potassium blood serum levels
17. Chronic noncongestive glaucoma
18. Hypercalcaemia
19. Articular gout
20. Known African anaemia
21. Diabetes mellitus type I/II
22. Other acute, serious illness of the subject
23. Subject is unable to understand the scope, significance and consequences of this clinical trial and is unable to comply with the study design
24. Previous participation in this clinical trial or participation in any clinical trial taking an investigational medicinal product within 30 days prior to written informed consent for this clinical trial

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified