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

Sponsored by:	Santhera Pharmaceuticals
Information provided by:	Santhera Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT00697073

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 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, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety Study
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

Further study details as provided by Santhera Pharmaceuticals:

Primary Outcome Measures:

Hematology [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
Nature and frequency of AEs [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
Blood and urine chemistry [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
Physical/neurological examination and vital signs [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
Electrocardiograms (ECGs) [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

FARS [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
ICARS [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
FACT [ Time Frame: 12 Months ] [ Designated as safety issue: No ]

Estimated Enrollment:	51
Study Start Date:	July 2008

Arms	Assigned Interventions
1: Experimental 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 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 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

No publications provided

Responsible Party:	Santhera Pharmaceuticals (Switzerland) Ltd. ( Thomas Meier, PhD / Chief Scientific Officer )
Study ID Numbers:	SNT-III-002-E
Study First Received:	June 11, 2008
Last Updated:	October 17, 2008
ClinicalTrials.gov Identifier:	NCT00697073 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 07, 2009