Efficacy of Epoetin Alfa in Patients With Friedreich's Ataxia

Status Completed

Clinical Trial 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.

Clinical Trial 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 Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT00631202
Study type	Interventional
Source	Federico II University
Contact
Status	Completed
Phase	Phase 2
Start date	February 2008
Completion date	June 2009

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT02660112` - (+) Epicatechin to Treat Friedreich's Ataxia	Phase 2
Recruiting	`NCT02497534` - Biomarkers in Friedreich's Ataxia
Completed	`NCT04102501` - A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia	Phase 3
Completed	`NCT01962363` - EPI-743 in Friedreich's Ataxia Point Mutations	Phase 2
Completed	`NCT02179333` - Preliminary Study of the Scale To Assess Ataxia and Neurologic Dysfunction (STAND)
Completed	`NCT01016366` - Safety Study of Carbamylated Erythropoietin to Treat Patients With the Neurodegenerative Disorder Friedreich's Ataxia	Phase 2
Recruiting	`NCT02069509` - Patient Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS)
Terminated	`NCT00803868` - Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia	Phase 2/Phase 3
Completed	`NCT02797080` - Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia	Phase 3
Completed	`NCT02415127` - Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia	Phase 3
Completed	`NCT00897221` - A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia	Phase 2
Completed	`NCT02840669` - A Study to Characterize the Cardiac Phenotype of Individuals With Friedreich's Ataxia (CARFA Study)	N/A
Completed	`NCT00697073` - Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients	Phase 3
Recruiting	`NCT02316314` - Characterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA)
Completed	`NCT01728064` - Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia	Phase 2
Completed	`NCT02593773` - Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study	Phase 3
Completed	`NCT01035671` - Safety and Efficacy Study of A0001 in Subjects With Friedreich's Ataxia	Phase 2
Completed	`NCT00811681` - Effect of Pioglitazone Administered to Patients With Friedreich's Ataxia: Proof of Concept	Phase 3
Completed	`NCT00537680` - Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia	Phase 3
Completed	`NCT02445794` - A First in Human Study of RT001 in Patients With Friedreich's Ataxia	Phase 1/Phase 2

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.