NCT01016366

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
36

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Oct 2009

Shorter than P25 for phase_2

Geographic Reach
3 countries

7 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2009

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

November 18, 2009

Completed
1 day until next milestone

First Posted

Study publicly available on registry

November 19, 2009

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2011

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2011

Completed
Last Updated

November 8, 2016

Status Verified

November 1, 2016

Enrollment Period

1.4 years

First QC Date

November 18, 2009

Last Update Submit

November 7, 2016

Conditions

Friedreich's Ataxia

Keywords

Friedreich's AtaxiaFRDANeurodegenerativeErythropoietinCarbamylatedNeuroprotectionFrataxin

Outcome Measures

Primary Outcomes (1)

  • To evaluate the safety and tolerability of 2 weeks treatment with Lu AA24493 in patients with Friedreich's Ataxia

    2 week treatment phase + 4 week follow up period

Secondary Outcomes (5)

  • To explore biomarkers of efficacy, including frataxin, 8-OHdG & peroxides

    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))

    2 week treatment phase + 4 week follow up period

  • To explore efficacy by the Clinical Global Impression scales (CGI-I/S)

    2 week treatment phase + 4 week follow up period

  • To explore population pharmacokinetic parameters of Lu AA24493

    2 week treatment phase + 4 week follow up period

  • To evaluate the immunogenicity of Lu AA24493

    2 week treatment phase + 4 week follow up period

Study Arms (2)

Lu AA24493

EXPERIMENTAL
Drug: Lu AA24493

Placebo

PLACEBO COMPARATOR
Drug: Placebo

Interventions

Lu AA24493DRUG

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.

Also known as: CEPO
Lu AA24493
PlaceboDRUG

Vials with solution for i.v. injection. Placebo dosed 3 times per week for two weeks.

Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

AT001

Innsbruck, 6020, Austria

Location

DE004

Bochum, 44791, Germany

Location

DE002

Bonn, 53127, Germany

Location

DE001

Munich, 80336, Germany

Location

DE003

Tübingen, 72026, Germany

Location

IT001

Milan, 20133, Italy

Location

IT002

Naples, 80131, Italy

Location

MeSH Terms

Conditions

Friedreich Ataxia

Condition Hierarchy (Ancestors)

Spinocerebellar DegenerationsCerebellar DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesSpinal Cord DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMitochondrial DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Email contact via H. Lundbeck A/S

    LundbeckClinicalTrials@lundbeck.com

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 18, 2009

First Posted

November 19, 2009

Study Start

October 1, 2009

Primary Completion

March 1, 2011

Study Completion

April 1, 2011

Last Updated

November 8, 2016

Record last verified: 2016-11

Locations

AU(1)DE(4)IT(2)