Trial document

PLEASE NOTE:
This study has been imported from ClinicalTrials.gov without additional data checks.

drksid header

DRKS-ID: DRKS00005837

Trial Description

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Title

Patient Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS)

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Trial Acronym

EFACTS

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URL of the Trial

[---]*

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Brief Summary in Lay Language

This is a multi-centre, multi-national, prospective, observational study of Friedreich's
Ataxia (FRDA) with a control group to:

- obtain natural history data on individuals affected by FRDA

- relate clinical assessments and results from proteomic analyses

- expedite identification and recruitment of participants for clinical trials

- develop and validate sensitive and reliable outcome measures for detecting onset and
change over the natural course of FRDA which may also be potential outcome measures for
use in future clinical trials and clinical care

- plan for future research studies

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Brief Summary in Scientific Language

The EFACTS patient registry integrates prospectively and systematically collected clinical
research data (e.g. clinical tests, demographical characteristics) with access to biological
specimens (e.g. blood, urine) obtained from individuals with genetically confirmed FRDA and
unrelated control research participants.

The EFACTS patient registry is a 4-year study and eligible subjects are assessed at annual
study visits on the clinical symptoms and signs of the disease. At each study visit, general
clinical, motor function, cognitive, and Quality of Life assessments are administered. In
addition, participants are providing bio samples for the purposes of genetic testing and for
research to identify valuable biomarkers of FRDA. Biological specimens and clinical data are
made available to qualified scientists within the EFACTS network whose projects are reviewed
and approved by the EFACTS Steering Committee. Research projects should aim to advance
scientific knowledge towards establishing clinically effective treatments that delay onset
and/or slow the progression of the disease.

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Organizational Data

DRKS-ID: DRKS00005837
Date of Registration in DRKS: 2015/01/19
Date of Registration in Partner Registry or other Primary Registry: 2014/02/19
Investigator Sponsored/Initiated Trial (IST/IIT): yes
Ethics Approval/Approval of the Ethics Committee: [---]*
(leading) Ethics Committee No.: [---]*

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Secondary IDs

Primary Registry-ID: NCT02069509 (ClinicalTrials.gov)
Sponsor-ID: HEALTH-F2-2010- 242193 (European Friedreich's Ataxia Consortium for Translational Studies)

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Health Condition or Problem studied

Free text: Friedreich's Ataxia
ICD10: G11.1 - Early-onset cerebellar ataxia

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Interventions/Observational Groups

[---]*

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Characteristics

Study Type: Non-interventional
Study Type Non-Interventional: Observational study
Allocation: [---]*
Blinding: [---]*
Who is blinded: [---]*
Control: [---]*
Purpose: [---]*
Assignment: [---]*
Phase: N/A
Off-label Drug use: [---]*

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Primary Outcome

- Disease progression as assessed by clinical examination; time frame: Participants are followed for a max. of 4 years with annual assessments

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Secondary Outcome

[---]*

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Countries of Recruitment

AT: Austria
BE: Belgium
FR: France
DE: Germany
IT: Italy
ES: Spain
UK: United Kingdom

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Locations of Recruitment

University Hospital Aachen, Dept. of Neurology, Aachen
University Hospital Bonn, Dept. of Neurology, Bonn
Philipps University Marburg, Dept. of Neurology, Marburg
University of Munich, Dept. of Neurology, Munich
University of Tübingen, Dept. of Neurodegenerative Diseases, Hertie-Institute for Clinical Brain Research, Tübingen

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Recruitment

Planned/Actual: [---]*
(Anticipated or Actual) Date of First Enrollment: 2010/05/31
Target Sample Size: 650
Monocenter/Multicenter trial: Multicenter trial
National/International: International

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Inclusion Criteria

Gender: Both, male and female
Minimum Age: no minimum age
Maximum Age: no maximum age

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Additional Inclusion Criteria

Inclusion Criteria:

- Genetic diagnosis of FRDA

- For control research participants: genetically confirmed absence of FRDA

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Exclusion Criteria

[---]*

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Addresses

Primary Sponsor
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- European Friedreich's Ataxia Consortium for Translational Studies
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- Telephone: [---]*
- Fax: [---]*
- E-mail: [---]*
- URL: [---]*
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Contact for Scientific Queries
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- University Hospital Aachen, Dept. of Neurology
- Jörg B. Schulz
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- Telephone: [---]*
- Fax: [---]*
- E-mail: [---]*
- URL: [---]*
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Contact for Public Queries
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- Jörg B. Schulz, MD
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- Telephone: 0049-241-80-89600
- Fax: [---]*
- E-mail: jschulz at ukaachen.de
- URL: [---]*
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Sources of Monetary or Material Support

[---]*
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- Bitte wenden Sie sich an den Sponsor / Please refer to primary sponsor
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- Telephone: [---]*
- Fax: [---]*
- E-mail: [---]*
- URL: [---]*
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Status

Recruitment Status: Recruiting ongoing
Study Closing (LPLV): [---]*

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Trial Publications, Results and other Documents

Further trial documents: EFACTS Registry

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The parameters in ClinicalTrials.gov and DRKS are not identical. Therefore the data import from ClinicalTrials.gov required adjustments. For full details please see the DRKS FAQs .

Translation on version: 1
Last processed date by ClinicalTrials.gov: 2014/02/23

* This entry means the parameter is not applicable or has not been set.