Safety Study of Carbamylated Erythropoietin to Treat Patients With the Neurodegenerative Disorder Friedreich's Ataxia

• Status: Completed
• Trial end date: 2011-04-01
• Target enrollment: 0
• Participant gender: All

Summary

The primary purpose of the study is to determine whether carbamylated erythropoietin is a safe treatment for patients who suffer from Friedreich's Ataxia.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: H. Lundbeck A/S
• Treatments: Epoetin Alfa

Criteria

Inclusion Criteria:

• The patient has been diagnosed with FRDA and has had a genetic test demonstrating >400 GAA nucleotide triplet repeats on the shorter of the two frataxin alleles

• The patient has a SARA (Stance) sub-score of <=6

• The patient has a SARA (Gait) sub-score of <=6

• Man or woman, aged 18 years or over

• If female then woman should agree not to try to become pregnant during the study, and use adequate protection/abstinence or not be of child bearing potential

Exclusion Criteria:

• Clinically significant unstable illnesses such as liver, kidney, heart, stomach problems unrelated to their disease of FRDA

• Disallowed medications

• Serious underlying disease

• Clinically significant abnormal vital signs unrelated to the underlying disease of FRDA

• Abnormal laboratory blood results considered by the doctor as clinically significant, e.g.anaemia

• Treatment with idebenone within 6 weeks prior to screening

• Treatment with erythropoietin within 16 weeks prior to screening

• Clinically significant abnormal ECG

• Received or donated blood within previous 3 months

• Participation within another clinical trial within past 30 days

• Pregnancy or breast feeding

• History of drug allergies or hypersensitivities

• Current (or within past 6 months) disorder related to drug or alcohol abuse (as defined DSM-IV-TR)