Status:
COMPLETED
Efficacy Study of Epoetin Alfa in Friedreich Ataxia
Lead Sponsor:
Federico II University
Collaborating Sponsors:
Friedreich's Ataxia Research Alliance
Associazione Italiana per la lotta alle Sindromi Atassiche (AISA)
Conditions:
Friedreich Ataxia
Eligibility:
All Genders
12+ years
Phase:
PHASE2
Brief Summary
Friedreich's ataxia (FRDA) is a rare genetic disorder characterised by severe neurological disability and cardiomyopathy. Friedreich's ataxia is the consequence of frataxin deficiency. Although severa...
Detailed Description
Friedreich's ataxia (FA) is an autosomal recessive ataxia caused by a trinucleotide GAA expansion in the first intron of the FXN gene. The gene encodes for a 210aa mitochondrial protein called frataxi...
Eligibility Criteria
Inclusion
- Molecular diagnosis of Friedreich Ataxia
- Age ≥12 years
- Body weight ≥30, ≤90 Kg
- SARA score ≤30
- Patient able to read and sign the informed consent
- Patients able to perform a cardiopulmonary test
Exclusion
- Treatment with Erythropoietin in the previous 12 months
- Treatment with Idebenone
- Contraindications to CPET: cardiac valve disease, ischemic cardiomyopathy, atrial fibrillation, asthma, chronic obstructive pulmonary disease, other arrhythmias judged as not compatible with exercise.
- Any Cardiac and/or Hepatic and/or Renal disease judged as clinically relevant by the investigator
- Any clinically relevant ECG abnormalities that may interfere with the study
- Any abnormal and clinically relevant laboratory exams at screening visit that may interfere with the trial
- Anemia with Hemoglobin \<10 g/dL
- Positive history for venous and/or arterial thrombosis
- Drug-resistant arterial hypertension
- Positive history for drug-resistant epilepsy
- Patients in treatment with not allowed study drugs (starting from 3 months prior to screening)
- Any acute/chronic disease that might interfere with the clinical trial, as judged by the investigator
- Hypersensitivity to Epoetin alfa or any other component of the study drug
- Patients not able to comply to the study
- For female patients (Sexually not active, hysterectomized, sterilized, menopause patients are excluded from the following criteria): pregnancy and/or breastfeeding and/or inadequate contraception.
Key Trial Info
Start Date :
January 1 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 1 2015
Estimated Enrollment :
56 Patients enrolled
Trial Details
Trial ID
NCT01493973
Start Date
January 1 2013
End Date
June 1 2015
Last Update
August 11 2015
Active Locations (3)
Enter a location and click search to find clinical trials sorted by distance.
1
Università di Bari
Bari, BA, Italy, 70124
2
Università la Sapienza, Neurologia C
Rome, RM, Italy, 00186
3
Dipartimento di Scienze Neurologiche
Napoli, Italy, 80131