Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia - Full Text View

Sponsored by:	Santhera Pharmaceuticals
Information provided by:	Santhera Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT00537680

Purpose

This study is meant to assess the effectiveness of idebenone on neurological outcome measures in patients with Friedreich's Ataxia over a 6 months period.

Condition	Intervention	Phase
Friedreich's Ataxia	Drug: Idebenone Drug: Placebo	Phase III

Genetics Home Reference related topics: Friedreich ataxia Marinesco-Sjögren syndrome mitochondrial neurogastrointestinal encephalopathy disease

MedlinePlus related topics: Friedreich's Ataxia

Drug Information available for: CV 2619

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Parallel Assignment, Safety/Efficacy Study
Official Title:	A Phase III Double-Blind, Randomized, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

Further study details as provided by Santhera Pharmaceuticals:

Primary Outcome Measures:

ICARS [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

FARS [ Time Frame: 6 Months ] [ Designated as safety issue: No ]
ADL of FARS [ Time Frame: 6 Months ] [ Designated as safety issue: No ]
FACT [ Time Frame: 6 Months ] [ Designated as safety issue: No ]

Estimated Enrollment:	51
Study Start Date:	December 2007
Estimated Study Completion Date:	May 2009
Estimated Primary Completion Date:	May 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Experimental mid dose Idebenone	Drug: Idebenone Patients ≤ 45 kg/99 lbs: idebenone 450 mg/day; Patients > 45 kg/99 lbs: idebenone 900 mg/day
2: Experimental high dose Idebenone	Drug: Idebenone Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day
3: Placebo Comparator	Drug: Placebo

Detailed Description:

The study involves 6 clinic visits and upon completion the possibility to join a 12 months extension study were all patients will receive high dose Idebenone.

Eligibility

Ages Eligible for Study:	8 Years to 17 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion criteria:

Documented diagnosis of Friedreich's Ataxia (FRDA) with confirmed FRDA mutations
Patients 8 - 17 years of age at baseline
Patients with a body weight greater than 25 kg/55 lbs at baseline
Patients able to walk at least 10 meters without accompanying person (ICARS Walking Capacities score ≤6) at screening and baseline
Patients who in the opinion of the investigator are able to comply with the requirements of the study, including swallowing the study medication
Negative urine pregnancy test at screening and baseline (female patients of childbearing potential)

Exclusion criteria:

Treatment with idebenone, Coenzyme Q10 or vitamin E (if taken at a dose 5 times above the daily requirement) within the past month
Patients with International Cooperative Ataxia Rating Scale (ICARS) score of greater than 54 or less than 10 at screening
Pregnancy and/or breast-feeding
Clinically significant abnormalities of clinical hematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of normal of Aspartate Aminotransferase (AST; also known as GOT), Alanine Aminotransferase (ALT; also known as GPT) or creatinine
History of abuse of drugs or alcohol
Participation in the previous (Phase II) study of idebenone at the National Institutes of Health (NIH)
Participation in a trial of another investigational drug within the last 3 months

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00537680

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

No publications provided

Responsible Party:	Santhera Pharmaceuticals (Switzerland) Ltd. ( Thomas Meier, PhD / Chief Scientific Officer )
Study ID Numbers:	SNT-III-002
Study First Received:	September 7, 2007
Last Updated:	April 15, 2009
ClinicalTrials.gov Identifier:	NCT00537680 History of Changes
Health Authority:	United States: Food and Drug Administration

Keywords provided by Santhera Pharmaceuticals:

Friedreich's Ataxia
Idebenone
ICARS

Study placed in the following topic categories:

Antioxidants
Metabolic Diseases
Spinal Cord Diseases
Central Nervous System Diseases
Idebenone
Mitochondrial Diseases
Brain Diseases
Neurodegenerative Diseases
Dyskinesias

Signs and Symptoms
Heredodegenerative Disorders, Nervous System
Friedreich Ataxia
Genetic Diseases, Inborn
Ataxia
Neurologic Manifestations
Cerebellar Diseases
Metabolic Disorder
Spinocerebellar Degenerations

Additional relevant MeSH terms:

Antioxidants
Metabolic Diseases
Molecular Mechanisms of Pharmacological Action
Spinal Cord Diseases
Physiological Effects of Drugs
Nervous System Diseases
Central Nervous System Diseases
Idebenone
Mitochondrial Diseases
Brain Diseases
Neurodegenerative Diseases

Protective Agents
Dyskinesias
Pharmacologic Actions
Signs and Symptoms
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn
Friedreich Ataxia
Ataxia
Neurologic Manifestations
Cerebellar Diseases
Spinocerebellar Degenerations

ClinicalTrials.gov processed this record on May 06, 2009