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

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ClinicalTrials.gov Identifier: NCT01016366
Recruitment Status : Completed
First Posted : November 19, 2009
Last Update Posted : November 8, 2016
Sponsor:
Information provided by (Responsible Party):
H. Lundbeck A/S

Tracking Information
First Submitted Date  ICMJE November 18, 2009
First Posted Date  ICMJE November 19, 2009
Last Update Posted Date November 8, 2016
Study Start Date  ICMJE October 2009
Actual Primary Completion Date March 2011   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 18, 2009)
To evaluate the safety and tolerability of 2 weeks treatment with Lu AA24493 in patients with Friedreich's Ataxia [ Time Frame: 2 week treatment phase + 4 week follow up period ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01016366 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: November 18, 2009)
  • To explore biomarkers of efficacy, including frataxin, 8-OHdG & peroxides [ Time Frame: 2 week treatment phase + 4 week follow up period ]
  • To explore efficacy by neurological assessment (Scale for the Assessment and Rating of Ataxia (SARA), Friedreich's Ataxia Rating Scale (FARS)) [ Time Frame: 2 week treatment phase + 4 week follow up period ]
  • To explore efficacy by the Clinical Global Impression scales (CGI-I/S) [ Time Frame: 2 week treatment phase + 4 week follow up period ]
  • To explore population pharmacokinetic parameters of Lu AA24493 [ Time Frame: 2 week treatment phase + 4 week follow up period ]
  • To evaluate the immunogenicity of Lu AA24493 [ Time Frame: 2 week treatment phase + 4 week follow up period ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety Study of Carbamylated Erythropoietin to Treat Patients With the Neurodegenerative Disorder Friedreich's Ataxia
Official Title  ICMJE Randomised, Double Blind, Placebo Controlled Study of Lu AA24493 in Patients With Friedreich's Ataxia to Evaluate Safety and Tolerability and to Explore Efficacy
Brief 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.
Detailed Description

Friedreich's Ataxia (FRDA) is a hereditary, progressive neurodegenerative disorder caused by mutations in the gene encoding frataxin. The mutation results in a severe reduction in levels of the mitochondrial protein, frataxin. A decline in frataxin levels and its associated consequences is believed to be the primary cause of symptoms in FRDA patients. The clinical symptoms of FRDA include progressive gait and limb ataxia, dysarthria, diabetes mellitus and hypertrophic cardiomyopathy. First symptoms usually appear between the age of 5 and 15 years. As the disease progresses the patient becomes confined to a wheel chair and at later stages the patients become increasingly incapacitated. There is currently no effective treatment for FRDA.

The naturally occurring hormone, erythropoietin (EPO), is able to protect various neuronal tissues from ischemic injury. Recombinant human erythropoietin (EPO) increases frataxin expression in lymphocytes from patients with FRDA. Also, EPO treatment of FRDA patients resulted in a favourable outcome compared to baseline as assessed by the levels of frataxin and biomarkers of oxidative stress. In a pilot study with EPO in FRDA patients, the treatment was well tolerated apart from the expected haematological (haematopoietic) side effects. Lu AA24493 (CEPO) is a modified (carbamylated) version of EPO, which is neuroprotective but without the haematopoietic side effects. Lu AA24493 is being developed for treatment of patients with FRDA.

Although the target for the non-haematological effects of Lu AA24493 (and EPO) is currently unknown, Lu AA24493 (CEPO) can protect cells and tissue from various types of injuries. Furthermore, in vitro Lu AA24493 (CEPO) increases the frataxin levels in lymphocytes from FRDA patients as well as from control patients. This study aims to evaluate the safety of 2 weeks treatment (6 doses, 3 doses per week) of CEPO in patients with FRDA and to explore efficacy by using neurological rating scales and by exploring levels of frataxin and biomarkers of oxidative stress.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Friedreich's Ataxia
Intervention  ICMJE
  • Drug: Lu AA24493
    Vials with solution for i.v. injection. 325mcg Lu AA24493 dosed 3 times per week for two weeks. Vials will be supplied in concentrations ready for injection.
    Other Name: CEPO
  • Drug: Placebo
    Vials with solution for i.v. injection. Placebo dosed 3 times per week for two weeks.
Study Arms  ICMJE
  • Experimental: Lu AA24493
    Intervention: Drug: Lu AA24493
  • Placebo Comparator: Placebo
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: November 18, 2009)
36
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE April 2011
Actual Primary Completion Date March 2011   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Austria,   Germany,   Italy
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01016366
Other Study ID Numbers  ICMJE 12631A
2008-003662-25 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party H. Lundbeck A/S
Study Sponsor  ICMJE H. Lundbeck A/S
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Email contact via H. Lundbeck A/S LundbeckClinicalTrials@lundbeck.com
PRS Account H. Lundbeck A/S
Verification Date November 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP