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Tracking Information | |||||
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First Received Date ICMJE | February 28, 2008 | ||||
Last Updated Date | May 26, 2010 | ||||
Start Date ICMJE | February 2008 | ||||
Primary Completion Date | December 2008 (final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
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 ] [ Designated as safety issue: No ] | ||||
Original Primary Outcome Measures ICMJE |
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 ] [ Designated as safety issue: No ] | ||||
Change History | Complete list of historical versions of study NCT00631202 on ClinicalTrials.gov Archive Site | ||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Outcome Measures ICMJE | |||||
Original Other Outcome Measures ICMJE | |||||
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. |
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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. |
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Study Type ICMJE | Interventional | ||||
Study Phase | Phase 2 | ||||
Study Design ICMJE | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
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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 |
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Study Arms | Experimental: I
Treatment arm
Intervention: Drug: Epoetin alfa |
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Publications * | |||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Completed | ||||
Estimated Enrollment ICMJE | 10 | ||||
Completion Date | June 2009 | ||||
Primary Completion Date | December 2008 (final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Gender | Both | ||||
Ages | 18 Years to 50 Years | ||||
Accepts Healthy Volunteers | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Location Countries ICMJE | Italy | ||||
Administrative Information | |||||
NCT Number ICMJE | NCT00631202 | ||||
Other Study ID Numbers ICMJE | FA_EPO_3 | ||||
Has Data Monitoring Committee | No | ||||
Responsible Party | Prof. Alessandro Filla, Dipartimento di Scienze Neurologiche | ||||
Study Sponsor ICMJE | Federico II University | ||||
Collaborators ICMJE | |||||
Investigators ICMJE |
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Information Provided By | 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 |