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
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.
|Condition or disease||Intervention/treatment||Phase|
|Friedreich's Ataxia||Drug: Epoetin alfa||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Single-Center, Open-Label, Sequential Trial to Test the Efficacy, Safety and Tolerability of Epoetin Alfa in Patients With Friedreich's Ataxia|
|Study Start Date :||February 2008|
|Actual Primary Completion Date :||December 2008|
|Actual Study Completion Date :||June 2009|
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
- 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 ]
- 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 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00631202
|Dipartimento di Scienze Neurologiche|
|Naples, Italy, 80131|
|Study Director:||Alessandro Filla, MD||Dipartimento di Scienze Neurologice, University "Federico II" Naples|