Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

NCT00697073 | Completed Phase 3 Results DMC

• 1 other identifier: SNT-III-002-E
• Study Type: interventional
• Target: 68
• Locations: 1 country
• Sites: 2

Brief Summary

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

Conditions

Friedreich's Ataxia

Keywords

Friedreich's Ataxia; Idebenone; ICARS

Outcome Measures

Primary Outcomes (1)

• Change in ICARS

  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. A total score of 0 points represents the best possible score, and a value of 100 points is the worst possible score. Therefore, a negative change in ICARS score represents an improvement. A higher score also indicates more disability.

  baseline and 12 months

Secondary Outcomes (3)

• FARS (Friedreich's Ataxia Rating Scale)

  baseline and 12 Months

• Nature of Adverse Events

  12 Months

• Frequency/Number of Mild, Moderate, and Severe Adverse Events

  12 months

Study Arms (1)

1

EXPERIMENTAL

high dose Idebenone

Drug: Idebenone

Interventions

Idebenone DRUG

Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients \> 45 kg/99 lbs: idebenone 2250 mg/day

1

Eligibility Criteria

• Age: 8 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Santhera Pharmaceuticals: lead

Study Sites (2)

David Geffen School of Medicine, UCLA

Los Angeles, California, 90095-1769, United States

Location

The Children's Hopsital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (1)

• 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. doi: 10.1007/s00415-011-6174-y. Epub 2011 Jul 22.

  PMID: 21779958 RESULT

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

idebenone

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Dr Thomas Meier
• Organization: Santhera

thomas.meier@santhera.com

+41 61 906 8964

Study Officials

• Susan Perlman, MD

  University of California, Los Angeles

  PRINCIPAL INVESTIGATOR

• David Lynch, MD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 11, 2008
• First Posted: June 13, 2008
• Study Start: July 1, 2008
• Primary Completion: May 1, 2010
• Study Completion: May 1, 2010
• Last Updated: July 11, 2023
• Results First Posted: August 4, 2011

Record last verified: 2023-06

Locations

US (2)