NCT00537680

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
70

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Dec 2007

Shorter than P25 for phase_3

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 7, 2007

Completed
24 days until next milestone

First Posted

Study publicly available on registry

October 1, 2007

Completed
2 months until next milestone

Study Start

First participant enrolled

December 1, 2007

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2009

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2009

Completed
2.7 years until next milestone

Results Posted

Study results publicly available

December 29, 2011

Completed
Last Updated

July 11, 2023

Status Verified

June 1, 2023

Enrollment Period

1.3 years

First QC Date

September 7, 2007

Results QC Date

June 7, 2011

Last Update Submit

June 22, 2023

Conditions

Friedreich's Ataxia

Keywords

Friedreich's AtaxiaIdebenoneICARS

Outcome Measures

Primary Outcomes (1)

  • Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24

    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. Minimum score: 0, maximum score 100, higher score indicates greater impairment Calculation details: ICARS score at week 24 minus ICARS score baseline

    baseline and 6 months

Secondary Outcomes (11)

  • Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24

    baseline and 6 months

  • Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24

    baseline and 6 months

  • Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12

    baseline and 12 weeks

  • Absolute Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle

    baseline and 6 months

  • Percent Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle

    baseline and 6 months

  • +6 more secondary outcomes

Study Arms (3)

Arm 1 - 900 mg/day Idebenone

EXPERIMENTAL

mid dose Idebenone

Drug: Idebenone

Arm 2 - up to 2250 mg/day Idebenone

EXPERIMENTAL

high dose Idebenone

Drug: Idebenone

Arm 3 - Placebo

PLACEBO COMPARATOR

Placebo

Drug: Placebo

Interventions

IdebenoneDRUG

Patients ≤ 45 kg/99 lbs: idebenone 450 mg/day; Patients \> 45 kg/99 lbs: idebenone 900 mg/day

Arm 1 - 900 mg/day Idebenone
PlaceboDRUG

Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.

Arm 3 - Placebo

Eligibility Criteria

Age8 Years - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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)

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

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 (3)

  • 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: 21779958RESULT

  • Lagedrost SJ, Sutton MS, Cohen MS, Satou GM, Kaufman BD, Perlman SL, Rummey C, Meier T, Lynch DR. Idebenone in Friedreich ataxia cardiomyopathy-results from a 6-month phase III study (IONIA). Am Heart J. 2011 Mar;161(3):639-645.e1. doi: 10.1016/j.ahj.2010.10.038. Epub 2011 Jan 31.

    PMID: 21392622DERIVED

  • Lynch DR, Perlman SL, Meier T. A phase 3, double-blind, placebo-controlled trial of idebenone in friedreich ataxia. Arch Neurol. 2010 Aug;67(8):941-7. doi: 10.1001/archneurol.2010.168.

    PMID: 20697044DERIVED

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

idebenone

Condition Hierarchy (Ancestors)

Spinocerebellar DegenerationsCerebellar DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesSpinal Cord DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMitochondrial DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Results Point of Contact

Title
Dr Thomas Meier
Organization
Santhera Pharmaceuticals
thomas.meier@santhera.com
+41619068964

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
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 7, 2007

First Posted

October 1, 2007

Study Start

December 1, 2007

Primary Completion

April 1, 2009

Study Completion

April 1, 2009

Last Updated

July 11, 2023

Results First Posted

December 29, 2011

Record last verified: 2023-06

Locations

US(2)