Trial document




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  DRKS00005258

Trial Description

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Title

Pharmacological treatment of episodic ataxie type 2: Comparison of efficacy of 4-aminopyridine sustained release (Fampyra®) and acetazolamid (Acemit®) versus placebo

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Trial Acronym

EAT2TREAT

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URL of the Trial

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Brief Summary in Lay Language

This trial is designed to investigate the efficacy of treatment with sustained release fampridine or treatment with acetazolamide in comparison with placebo in reducing the attack frequency in patients with episodic ataxia type 2.

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Brief Summary in Scientific Language

Each Subject will be treated consecutively in a randomised crossover design with sustained release 4-Aminopyridin (Fampyra®), Acetazolamid (Acemit®) and Placebo for 12 weeks each. The first and the second treatment period will be followed by a four week washout period to avoid carry over effects. A follow up assessment will be performed for each subject four weeks after the last study drug intake.

Primary Objective:
Efficacy in reducing attack frequency during treatment with sustained release 4-Aminopyridin (4-AP-R), Acetazolamid (ACTZ) in comparison with placebo.

Quantitative comparison of efficacy of 4-AP-R versus ACTZ in reducing attack frequency.

Secondary Objective:
Quantitative description and comparison of median attack duration and strength during treatment with 4-AP-R versus ACTZ versus placebo, measured during the last 30 days within a 12 week treatment period.

It is planned to randomise a total of 30 patients with episodic ataxia type 2.

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Organizational Data

  •   DRKS00005258
  •   2013/08/29
  •   2013/04/05
  •   yes
  •   Approved
  •   163-13, Ethik-Kommission der Medizinischen Fakultät der Ludwig-Maximilians-Universität München
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Secondary IDs

  •   2013-000107-17 
  •   EUCTR2013-000107-17-DE  (EU Clinical Trials Register )
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Health Condition or Problem studied

  •   G11.8 -  Other hereditary ataxias
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Interventions/Observational Groups

  •   Fampyra (sustained release tablets 4-Aminopyridin) 10 mg, oral, bid for 12 weeks followed by 4 weeks wash out
  •   Acemit (Acetazolamid) 250 mg, oral, tid for 12 weeks, followed by 4 weeks wash out
  •   corresponding placebo in double dummy design, oral, bid or tid for 12 weeks
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Characteristics

  •   Interventional
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  •   Randomized controlled trial
  •   Blinded
  •   patient/subject, investigator/therapist, assessor
  •   Placebo, Active control (effective treament of control group)
  •   Treatment
  •   Crossover
  •   III
  •   Yes
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Primary Outcome

Number of attacks measured during the last 30 days within a 12 week treatment period

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Secondary Outcome

Median duration and strength of attacks during the last 30 days within a 12 week treatment period.

Coefficient of variability of maximal walking speed (CVmax): Alteration (relative change [log scale]) after 12 weeks compared to the beginning of a 12 week treatment period as well as 4 weeks after the last dose of study medication.

Quality of life (EQ-5D-5L, VDADL): absolute change after 12 weeks compared to the beginning of a 12 week treatment period as well as 4 weeks after the last dose of study medication.

Ataxia scale (SARA): absolute changes after 12 weeks compared to the beginning of a 12 week treatment period as well as 4 weeks after the last dose of study medication.

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Countries of Recruitment

  •   Germany
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Locations of Recruitment

  • University Medical Center 
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Recruitment

  •   Actual
  •   2013/07/26
  •   30
  •   Monocenter trial
  •   National
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Inclusion Criteria

  •   Both, male and female
  •   18   Years
  •   no maximum age
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Additional Inclusion Criteria

- Subject has provided written informed consent prior to any study specific procedure

- Female and male subjects ≥ 18 years of age

- Subjects with genetically confirmed EA2 or hereditary ataxia with onset in the childhood presenting clinically as EA2

- Women of childbearing potential must use an acceptable method of contraception before randomisation and throughout study participation

- Subject is able to follow the study instructions and likely to complete all required study visits


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Exclusion Criteria

- Body weight ≤ 40kg

- Female subjects who are pregnant, breastfeeding or contemplate pregnancy during study duration

- Subjects who are taking OCT2 inhibitors, e.g. Cimetidin

- Known hypersensitivity against aminopyridines and/or acetazolamide/sulfonamides

- Cardiovascular diseases, e.g. actue myocardial infarction (within the last 3 months), cardiac arrythmia (prolonged QTc interval > 500ms, atrial fibrillation, AV block ≥ II), unstable angina pectoris, severe heart failure (NYHA IV), arterial hypertension (grade III according to the guidlines of the German Association for Cardiology 2008)

- Stroke within the last 3 months

- Epileptic seizure (current or in the past)

- Asthma (≥ grade III according to the Nationale Versorgungsleitlinie)

- Obstructive pulmonary disease, e.g. pulmonary emphysema

- Hepatic insufficiency defined as AST/ALT/total bilirubin > 3 ULN

- Mild or severe renal insufficiency (creatinine clearance ≤ 80ml/min)

- Adrenocortical insufficiency

- Not adjusted thyroid disease

- Acute gastric/intestinal ulcer

- Hyperchloremic acidosis

- Decreased sodium/potassium blood serum level

- Glaucoma

- Hypercalciuria

- Gout

- Known sickle cell anemia

- Diabetes mellitus type I/II

- Other acute, severe disease

- Subject is not able to understand the extent, meaning and consequences of the trial and to follow the study schedule

- Subject previously participated in this clinical trial or was treated with another investigational drug 30 days prior to study participation

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Addresses

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    • Klinikum der Universität München, Campus Großhadern
    • Marchioninistraße 15
    • 81377  München
    • Germany
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    • Klinikum der LMU München - Großhadern Deutsches Schwindel- und Gleichgewichtszentrum (IFB)
    • Mr.  Professor  Michael  Strupp 
    • Marchioninistr. 15
    • 81377  München
    • Germany
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    • Klinikum der LMU München - Großhadern Deutsches Schwindel- und Gleichgewichtszentrum (IFB)
    • Studienzentrale 
    • Marchioninistr. 15
    • 81377  München
    • Germany
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Sources of Monetary or Material Support

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    • Bundesministerium für Bildung und Forschung Dienstsitz Bonn
    • Heinemannstr. 2
    • 53175  Bonn
    • Germany
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Status

  •   Recruiting complete, follow-up complete
  •   2016/10/06
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Trial Publications, Results and other Documents

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