drugset / Trial / NCT00631202
Efficacy of Epoetin Alfa in Patients With Friedreich's Ataxia
Non-randomizedSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2008-02
- Primary completion
- 2008-12
- Completion
- 2009-06
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | epoetin alfa | Protein / enzyme biologic | 600 iu/kg | Subcutaneous |
| Subject | epoetin alfa | Protein / enzyme biologic | 1200 iu/kg | Subcutaneous |