Trial document




drksid header

  DRKS00005271

Trial Description

start of 1:1-Block title

Title

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

end of 1:1-Block title
start of 1:1-Block acronym

Trial Acronym

FACEG

end of 1:1-Block acronym
start of 1:1-Block url

URL of the Trial

[---]*

end of 1:1-Block url
start of 1:1-Block public summary

Brief Summary in Lay Language

The trial is designed to investigate the efficiacy of a treatment with sustained-release 4-aminopyridine (Fampyra®) in comparison with placebo on the gait variablity in patients with cerebellar gait disorders.

end of 1:1-Block public summary
start of 1:1-Block scientific synopsis

Brief Summary in Scientific Language

Each Subject will be treated consecutively in a randomised crossover design with sustained release 4-aminopyridine (Fampyra®) and placebo for 12 weeks each. The first 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:
Proof of efficiacy of 4-AP-R on the gait variability at maximum walking speed 14 days after start of study treatment.

end of 1:1-Block scientific synopsis
start of 1:1-Block organizational data

Organizational Data

  •   DRKS00005271
  •   2013/10/15
  •   2013/01/11
  •   yes
  •   Approved
  •   001/13, Ethik-Kommission der Medizinischen Fakultät der Ludwig-Maximilians-Universität München
end of 1:1-Block organizational data
start of 1:n-Block secondary IDs

Secondary IDs

  •   2012-005312-26 
  •   EUCTR2012-005312-26-DE  (EU-CTR)
end of 1:n-Block secondary IDs
start of 1:N-Block indications

Health Condition or Problem studied

  •   G11.8 -  Other hereditary ataxias
end of 1:N-Block indications
start of 1:N-Block interventions

Interventions/Observational Groups

  •   sustained-release 4-aminopyridine (Fampyra®), 10 mg, oral, bid (1-0-1) for 12 weeks followed by a 4 weeks washout period
  •   Corresponding placebo, oral, bid (1-0-1) for 12 weeks
end of 1:N-Block interventions
start of 1:1-Block design

Characteristics

  •   Interventional
  •   [---]*
  •   Randomized controlled trial
  •   Blinded
  •   patient/subject, investigator/therapist, assessor
  •   Placebo
  •   Treatment
  •   Crossover
  •   III
  •   Yes
end of 1:1-Block design
start of 1:1-Block primary endpoint

Primary Outcome

Logarithmized gait variability at maximum walking speed (CVmax [%]) 14 days after start of study treatment.


Change in logarithmized individual preferred walking speed (G_pref) 14 days after start of study treatment compared to baseline.

end of 1:1-Block primary endpoint
start of 1:1-Block secondary endpoint

Secondary Outcome

Logarithmized gait variability at maximum walking speed at the end of each 3-month treatment period and after an additional 1-month follow-up period.

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.

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.

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.


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.

end of 1:1-Block secondary endpoint
start of 1:n-Block recruitment countries

Countries of Recruitment

  •   Germany
end of 1:n-Block recruitment countries
start of 1:n-Block recruitment locations

Locations of Recruitment

  • University Medical Center 
end of 1:n-Block recruitment locations
start of 1:1-Block recruitment

Recruitment

  •   Actual
  •   2013/06/19
  •   75
  •   Monocenter trial
  •   National
end of 1:1-Block recruitment
start of 1:1-Block inclusion criteria

Inclusion Criteria

  •   Both, male and female
  •   18   Years
  •   80   Years
end of 1:1-Block inclusion criteria
start of 1:1-Block inclusion criteria add

Additional 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

end of 1:1-Block inclusion criteria add
start of 1:1-Block exclusion criteria

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

end of 1:1-Block exclusion criteria
start of 1:n-Block addresses

Addresses

  • start of 1:1-Block address primary-sponsor
    • Klinikum der Universität München, Campus Großhadern
    • Marchioninistraße 15
    • 81377  München
    • Germany
    end of 1:1-Block address primary-sponsor
    start of 1:1-Block address contact primary-sponsor
    end of 1:1-Block address contact primary-sponsor
  • start of 1:1-Block address scientific-contact
    • Dept. of Neurology and German Center for Vertigo and Balance Disorders Ludwig-Maximilians-University Munich Klinikum Großhadern
    • Ms.  Dr. med.  Carolin  Muth 
    • Marchioninistr. 15
    • 81377   Munich
    • Germany
    end of 1:1-Block address scientific-contact
    start of 1:1-Block address contact scientific-contact
    end of 1:1-Block address contact scientific-contact
  • start of 1:1-Block address public-contact
    • Dept. of Neurology and German Center for Vertigo and Balance Disorders Ludwig-Maximillians-University Munich Klinikum Großhadern
    • Mr.  Dr. med.  Carolin  Muth 
    • Marchioninistr. 15
    • 81377  Munich
    • Germany
    end of 1:1-Block address public-contact
    start of 1:1-Block address contact public-contact
    end of 1:1-Block address contact public-contact
end of 1:n-Block addresses
start of 1:n-Block material support

Sources of Monetary or Material Support

  • start of 1:1-Block address materialSupport
    • Biogen Idec GmbH
    • Carl-Zeiss-Ring 6
    • 85737  Ismaning
    • Germany
    end of 1:1-Block address materialSupport
    start of 1:1-Block address contact materialSupport
    end of 1:1-Block address contact materialSupport
end of 1:n-Block material support
start of 1:1-Block state

Status

  •   Recruiting complete, follow-up complete
  •   2016/10/31
end of 1:1-Block state
start of 1:n-Block publications

Trial Publications, Results and other Documents

  • [---]*
end of 1:n-Block publications
* This entry means the parameter is not applicable or has not been set.