Study to Assess the Safety and Tolerability of Idebenone in the - Full Text View

Purpose

This study is meant to assess the safety and tolerability of idebenone in patients with Friedreich's Ataxia over a 12 months period.

Condition	Intervention	Phase
Friedreich's Ataxia	Drug: Idebenone	Phase 3

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	A Phase III Open-Label, Single Group Extension Study of the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

Resource links provided by NLM:

Genetics Home Reference related topics: ataxia neuropathy spectrum childhood myocerebrohepatopathy spectrum deoxyguanosine kinase deficiency Friedreich ataxia infantile-onset spinocerebellar ataxia Marinesco-Sjögren syndrome mitochondrial neurogastrointestinal encephalopathy disease myoclonic epilepsy myopathy sensory ataxia spinocerebellar ataxia type 1 spinocerebellar ataxia type 2 spinocerebellar ataxia type 3 spinocerebellar ataxia type 6

MedlinePlus related topics: Friedreich's Ataxia

U.S. FDA Resources

Further study details as provided by Santhera Pharmaceuticals:

Primary Outcome Measures:

Change in ICARS [ Time Frame: baseline and 12 months ] [ Designated as safety issue: No ]
International Cooperative Ataxia Rating Scale (ICARS):

ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability.

Scores for each subscale quantify the extent of ataxia in each clinically important area. Subscale scores are summed to give a total score ranging from 0 (best) to 100 (worst).

Secondary Outcome Measures:

FARS (Friedreich's Ataxia Rating Scale) [ Time Frame: baseline and 12 Months ] [ Designated as safety issue: No ]
Nature and Frequency of Adverse Events [ Time Frame: 12 Months ] [ Designated as safety issue: Yes ]

Enrollment:	68
Study Start Date:	July 2008
Study Completion Date:	May 2010
Primary Completion Date:	May 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: 1 high dose Idebenone	Drug: Idebenone Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day

Detailed Description:

The study involves 6 clinic visits.

Eligibility

Ages Eligible for Study:	8 Years to 18 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion criteria:

Friedreich's ataxia patients completing core study SNT-III-002 (NCT00537680) and presenting at Week 24 (Visit 5) of that study
Body weight ≥ 25kg/55 lbs
Negative urine pregnancy test
Patients who in the opinion of the investigator are able to comply with the requirements of this study

Exclusion criteria:

Adverse events during the course of SNT-III-002(NCT00537680)which in the opinion of the investigator are attributable to idebenone and preclude further treatment with idebenone
Clinically significant abnormalities of clinical hematology or biochemistry including, but not limited to, elevations greater than 2 times the upper limit of normal of AST, ALT or creatinine
Treatment with coenzyme Q10, vitamin E (if taken at a dose 5 times above the daily requirement) or other sources of idebenone within the past month
Parallel participation in another clinical drug trial
Past or present history of abuse of drugs or alcohol
Pregnancy or breast-feeding

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00697073

Locations

United States, California
David Geffen School of Medicine, UCLA
Los Angeles, California, United States, 90095-1769
United States, Pennsylvania
The Children's Hopsital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104

Sponsors and Collaborators

Santhera Pharmaceuticals

Investigators

Principal Investigator:	Susan Perlman, MD	University of California, Los Angeles
Principal Investigator:	David Lynch, MD	Children's Hospital of Philadelphia

More Information

Publications:

Meier T, Perlman SL, Rummey C, Coppard NJ, Lynch DR. Assessment of neurological efficacy of idebenone in pediatric patients with Friedreich's ataxia: data from a 6-month controlled study followed by a 12-month open-label extension study. J Neurol. 2012 Feb;259(2):284-91. Epub 2011 Jul 22.

Responsible Party:	Thomas Meier, PhD / Chief Scientific Officer, Santhera Pharmaceuticals (Switzerland) Ltd.
ClinicalTrials.gov Identifier:	NCT00697073 History of Changes
Other Study ID Numbers:	SNT-III-002-E
Study First Received:	June 11, 2008
Results First Received:	June 7, 2011
Last Updated:	February 2, 2012
Health Authority:	United States: Food and Drug Administration

Keywords provided by Santhera Pharmaceuticals:

Friedreich's Ataxia
Idebenone
ICARS

Additional relevant MeSH terms:

Ataxia
Friedreich Ataxia
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
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Idebenone
Antioxidants
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Protective Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on May 21, 2013

Study to Assess the Safety and Tolerability of Idebenone in the (IONIA-E)