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Efficacy of Epoetin Alfa in Patients With Friedreich's Ataxia

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ClinicalTrials.gov Identifier: NCT00631202
Recruitment Status : Completed
First Posted : March 7, 2008
Last Update Posted : May 27, 2010
Sponsor:
Information provided by:
Federico II University

Tracking Information
First Submitted Date  ICMJE February 28, 2008
First Posted Date  ICMJE March 7, 2008
Last Update Posted Date May 27, 2010
Study Start Date  ICMJE February 2008
Actual Primary Completion Date December 2008   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 12, 2009)
Primary endpoint will be the frataxin level in PBMCs from patients at different timing from a single Epoetin alfa administration. [ Time Frame: 0, 24, 48, 96 hours; 7, 15, 30, 60 days ]
Original Primary Outcome Measures  ICMJE
 (submitted: February 28, 2008)
Primary endpoint will be the frataxin level in PBMCs from patients at different timing from a single rhu-EPO administration. [ Time Frame: 0, 24, 48, 96h, 7, 15, 30, 60 days ]
Change History Complete list of historical versions of study NCT00631202 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 12, 2009)
  • Echocardiography: Strain and strain rate after EPO administration at the highest study dose [ Time Frame: 0, 30 days ]
  • Safety laboratory parameters, adverse events and tolerability [ Time Frame: 0, 7, 15, 30, 60 days ]
  • International cooperative ataxia rating scale (ICARS). [ Time Frame: 0, 7, 30 days ]
Original Secondary Outcome Measures  ICMJE
 (submitted: February 28, 2008)
  • Echocardiography: Strain and strain rate after rhu-EPO administration at the highest study dose [ Time Frame: 0, 30 days ]
  • safety laboratory parameters, adverse events and tollerability [ Time Frame: 0, 7, 15, 30, 60 days ]
  • International cooperative ataxia rating scale (ICARS), 9-hole peg test (9-hpt) and the 25-feet timed walk will be used. They will explore rhu-EPO safety on gait, stance, limb ataxia, dysarthria, oculomotor disorders, and hand function. [ Time Frame: 0, 7, 30 days ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy of Epoetin Alfa in Patients With Friedreich's Ataxia
Official Title  ICMJE Single-Center, Open-Label, Sequential Trial to Test the Efficacy, Safety and Tolerability of Epoetin Alfa in Patients With Friedreich's Ataxia
Brief Summary

Friedreich's ataxia is a rare genetic disorder characterized by severe neurological disability and cardiomyopathy. Friedreich's ataxia is the consequence of frataxin deficiency. Although several drugs have been proposed, there is no available treatment. It was recently demonstrated that erythropoietin can increase the intracellular levels of frataxin in an in-vitro model.

The present project is aimed at testing the possible therapeutic approach of erythropoietin, which is an already available and commercialized drug. The investigators will perform both in-vitro and in-vivo tests, in order to asses its efficacy and safety in patients. The results will be useful to plan further clinical trials.

Detailed Description Friedreich ataxia (FRDA) is an inherited recessive disorder characterized by progressive neurological disability. FRDA is the consequence of frataxin deficiency. Although several drugs have been proposed for FRDA, there is no available treatment. Recently it was shown that recombinant human erythropoietin (rhu-EPO) administration increases frataxin expression in cultured human lymphocytes of FRDA patients. It is therefore of primary importance to test extensively rhu-EPO's ability in increasing frataxin levels in-vitro and in-vivo. In addition rhu-EPO is an already available and commercialized drug approved for the treatment of anaemia associated with chronic renal disease, heart failure and cancer. Towards this overall purpose, we will perform an acute clinical trial in FRDA patients with rhu-EPO and will assess its effect in-vivo on frataxin expression. In addition, rhu-EPO's safety in FRDA patients based on laboratory parameters and neurological indexes will be tested. The results will be useful to gain new insight in the role of rhu-EPO in FRDA, and in the future, it may be useful to plan further clinical trials.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Friedreich's Ataxia
Intervention  ICMJE Drug: Epoetin alfa
Patients that will satisfy all inclusion/exclusion criteria will be sequentially treated with three single Epoetin alfa administrations. The first time the dose will be 600U/KG BW s.c. in a single administration. The outcome measures will be assessed. A washout period of 1 month will be necessary to eliminate any carry-over effect. A second administration of 1200U/KG BW s.c. will be performed. Outcome measures will be again assessed.
Other Name: Eprex 40.000 IU
Study Arms  ICMJE Experimental: I
Treatment arm
Intervention: Drug: Epoetin alfa
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Estimated Enrollment  ICMJE
 (submitted: February 28, 2008)
10
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE June 2009
Actual Primary Completion Date December 2008   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Molecular diagnosis of FA based on a homozygous GAA expansion within the FRDA with a triplet repeat sequence in the pathological range.
  • Age >18, <50 years

Exclusion Criteria:

  • Failure to meet one of the inclusion criteria
  • Patients in treatment with Idebenone
  • Wheelchair bound patients
  • Significant renal, hepatic or haematological disease
  • Positive history for arterial or venous thrombosis
  • Acute diseases that might interfere with the study
  • Positive history for arterial hypertension
  • Present or programmed pregnancy
  • Known hypersensitivity to study drug
  • Other unacceptable concomitant medications (in particular agents thought to have a neuroprotective potential as tocopherol, amantadine, memantine, free radical scavengers).
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 50 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Italy
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00631202
Other Study ID Numbers  ICMJE FA_EPO_3
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Prof. Alessandro Filla, Dipartimento di Scienze Neurologiche
Study Sponsor  ICMJE Federico II University
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Alessandro Filla, MD Dipartimento di Scienze Neurologice, University "Federico II" Naples
PRS Account Federico II University
Verification Date May 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP