Trial record 12 of 33 for:    "friedreich ataxia"

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

This study has been completed.
Sponsor:
Information provided by:
H. Lundbeck A/S
ClinicalTrials.gov Identifier:
NCT01016366
First received: November 18, 2009
Last updated: June 8, 2011
Last verified: June 2011
  Purpose

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


Condition Intervention Phase
Friedreich's Ataxia
Drug: Lu AA24493
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: Randomised, Double Blind, Placebo Controlled Study of Lu AA24493 in Patients With Friedreich's Ataxia to Evaluate Safety and Tolerability and to Explore Efficacy

Resource links provided by NLM:


Further study details as provided by H. Lundbeck A/S:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • To explore biomarkers of efficacy, including frataxin, 8-OHdG & peroxides [ Time Frame: 2 week treatment phase + 4 week follow up period ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • To explore efficacy by the Clinical Global Impression scales (CGI-I/S) [ Time Frame: 2 week treatment phase + 4 week follow up period ] [ Designated as safety issue: No ]
  • To explore population pharmacokinetic parameters of Lu AA24493 [ Time Frame: 2 week treatment phase + 4 week follow up period ] [ Designated as safety issue: No ]
  • To evaluate the immunogenicity of Lu AA24493 [ Time Frame: 2 week treatment phase + 4 week follow up period ] [ Designated as safety issue: No ]

Enrollment: 36
Study Start Date: October 2009
Study Completion Date: April 2011
Primary Completion Date: March 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Lu AA24493 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
Placebo Comparator: Placebo Drug: Placebo
Vials with solution for i.v. injection. Placebo dosed 3 times per week for two weeks.

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01016366

Locations
Austria
AT001
Innsbruck, Austria, 6020
Germany
DE004
Bochum, Germany, 44791
DE002
Bonn, Germany, 53127
DE001
Munich, Germany, 80336
DE003
Tuebingen, Germany, 72026
Italy
IT001
Milano, Italy, 20133
IT002
Naples, Italy, 80131
Sponsors and Collaborators
H. Lundbeck A/S
Investigators
Study Director: Email contact via H. Lundbeck A/S LundbeckClinicalTrials@lundbeck.com
  More Information

No publications provided

Responsible Party: H. Lundbeck A/S
ClinicalTrials.gov Identifier: NCT01016366     History of Changes
Other Study ID Numbers: 12631A, 2008-003662-25
Study First Received: November 18, 2009
Last Updated: June 8, 2011
Health Authority: Austria: Federal Office for Safety in Health Care
Germany: Federal Institute for Drugs and Medical Devices
Italy: Ethics Committee

Keywords provided by H. Lundbeck A/S:
Friedreich's Ataxia
FRDA
Neurodegenerative
Erythropoietin
Carbamylated
Neuroprotection
Frataxin

Additional relevant MeSH terms:
Friedreich Ataxia
Ataxia
Neurodegenerative Diseases
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinocerebellar Degenerations
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Epoetin Alfa
Hematinics
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 20, 2014