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

Friedreich's Ataxia Clinical Trial

— IONIA-E  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00697073
• Other study ID #: SNT-III-002-E
• Status: Completed
• Phase: Phase 3
• Start date: July 2008
• Est. completion date: May 2010

Study information

• Verified date: June 2023
• Source: Santhera Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Description:

The study involves 6 clinic visits.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 68
• Est. completion date: May 2010
• Est. primary completion date: May 2010
• Accepts healthy volunteers: No
• Gender: All
• Age group: 8 Years to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Friedreich Ataxia
• Friedreich's Ataxia

Intervention

Drug:
• Idebenone
Patients = 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day

Locations

Country	Name	City	State
United States	David Geffen School of Medicine, UCLA	Los Angeles	California
United States	The Children's Hopsital of Philadelphia	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Santhera Pharmaceuticals

Country where clinical trial is conducted

United States, 

References & 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 Fe — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	FARS (Friedreich's Ataxia Rating Scale)	FARS consists of a 25 manoeuvre exam along with 4 quantitative performance measures. The neurological exam covers bulbar function, upper limb coordination, lower limb coordination, peripheral nervous system function, deep tendon reflexes, stability and gait. The use of FARS has been recently validated as a sensitive scale for this population. The FARS consists of three subscales, comprising a general score for ataxia, a score for activities of daily living (ADL) and a neurological examination. The scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability.	baseline and 12 Months
Secondary	Nature of Adverse Events		12 Months
Secondary	Frequency/Number of Mild, Moderate, and Severe Adverse Events		12 months