Friedreich's AtaxiaPhase 3IONIANCT00537680

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

A Phase 3 study testing Idebenone in people with Friedreich's Ataxia, run by Santhera Pharmaceuticals at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 8 to 17 years
Conditions the study is for: Friedreich's Ataxia
What is being tested?: Idebenone
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 70 participants
Where: 2 sitesUnited States
Study dates: Dec 2007to Apr 2009
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 8 to 17 years
All sexes
No healthy volunteers
Child

Who may be able to join

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)

Who cannot join

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

Trial locations and contacts

2 sites in 1 country.

United States2 sites

David Geffen School of Medicine, UCLA
Los Angeles, California, 90095-1769
The Children's Hopsital of Philadelphia
Philadelphia, Pennsylvania, 19104

Other trials in United States

About this 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

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.

Read the detailed description

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

Conditions: Friedreich's Ataxia
Being tested: Idebenone, Placebo
Keywords: Friedreich's Ataxia, Idebenone, ICARS
Medical subject headings: Friedreich Ataxia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

IdebenoneDrug
Patients ≤ 45 kg/99 lbs: idebenone 450 mg/day; Patients > 45 kg/99 lbs: idebenone 900 mg/day
IdebenoneDrug
Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day
PlaceboDrug
Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.

Study groups in NCT00537680
Study group	Role	What they receive
Arm 1 - 900 mg/day Idebenone	Experimental	mid dose Idebenone Receives: Drug: Idebenone
Arm 2 - up to 2250 mg/day Idebenone	Experimental	high dose Idebenone Receives: Drug: Idebenone
Arm 3 - Placebo	Placebo comparator	Placebo Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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
Measured: baseline and 6 months

Secondary outcomes (11)

Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24
To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on neurological function as assessed by the Friedreich's Ataxia Rating Scale (FARS)
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 exam scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability.
Calculation details FARS score at week 24 minus FARS score baseline
Measured: 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
To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on Activities of Daily Living (ADL) assessed by the ADL scale of the Friedreich's Ataxia Rating Scale (FARS)
score 0 to 36, higher score indicates greater impairment
Measured: baseline and 6 months
Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12
To compare the efficacy of 3 months' treatment with 2 different doses of idebenone with that of placebo on neurological impairment as assessed by the International Cooperative Ataxia Rating Scale (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.
Minimum score: 0, maximum score 100, higher score indicates greater impairment
Calculation details: ICARS score at week 12 minus ICARS score baseline
Measured: baseline and 12 weeks
Absolute Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Measured: baseline and 6 months
Percent Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Measured: baseline and 6 months
Absolute Change From Baseline to Week 24 in Left Ventricular Mass Index (LVMI)
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Measured: baseline and 6 months
Percent Change From Baseline to Week 24 in Left Ventricular Mass Index
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Measured: baseline and 6 months
Absolute Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Measured: baseline and 6 months
Percent Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Measured: baseline and 6 months
Absolute Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS)
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Measured: baseline and 6 months
Percent Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS)
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Measured: baseline and 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Dec 2007Apr 2009

First posted1 Oct 2007 (estimated)
Study startDec 2007
Primary completionApr 2009
Study completionApr 2009

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (3)Papers the sponsor has linked to this study.
Other trials for Friedreich's AtaxiaStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 70 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00537680
Sponsor's protocol ID: SNT-III-002
Acronym: IONIA
Status last verified: Jun 2023
First submitted: 7 Sep 2007
Last updated: 11 Jul 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

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.
PubMed 21779958
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.
PubMed 21392622
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.
PubMed 20697044

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
Friedreich Ataxia
Recruiting
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
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Active, not recruitingObservational study
Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia
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RecruitingObservational study
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All Friedreich's Ataxia trials

Study at a glance

From the registry record, last updated by the sponsor on 11 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT00537680
Acronym / sponsor ID: IONIA / SNT-III-002
Conditions: Friedreich's Ataxia
Keywords: Friedreich's Ataxia, Idebenone, ICARS
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 70 participants (actual)
Lead sponsor: Santhera PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: Dec 2007
Primary completion: Apr 2009 (actual)
Study completion: Apr 2009 (actual)
First posted: 1 Oct 2007 (estimated)
Results: Posted 29 Dec 2011

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Detailed description

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

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 3

Arm	Type	Receives
Arm 1 - 900 mg/day Idebenone	Experimental	Drug: Idebenonemid dose Idebenone
Arm 2 - up to 2250 mg/day Idebenone	Experimental	Drug: Idebenonehigh dose Idebenone
Arm 3 - Placebo	Placebo comparator	Drug: PlaceboPlacebo

Who may qualify

Ages: 8 to 17 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (6 listed)

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

Key exclusion criteria (7 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
IdebenonePatients ≤ 45 kg/99 lbs: idebenone 450 mg/day; Patients > 45 kg/99 lbs: idebenone 900 mg/day	Drug	Arm 1 - 900 mg/day Idebenone
IdebenonePatients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day	Drug	Arm 2 - up to 2250 mg/day Idebenone
PlaceboPlacebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.	Drug	Arm 3 - Placebo

Primary outcome

Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24
Time frame: baseline and 6 months
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

11 secondary outcomes

Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24
Time frame: 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
Time frame: baseline and 6 months
Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12
Time frame: baseline and 12 weeks
Absolute Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle
Time frame: baseline and 6 months
Percent Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle
Time frame: baseline and 6 months
Absolute Change From Baseline to Week 24 in Left Ventricular Mass Index (LVMI)
Time frame: baseline and 6 months
Percent Change From Baseline to Week 24 in Left Ventricular Mass Index
Time frame: baseline and 6 months
Absolute Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle
Time frame: baseline and 6 months
Percent Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle
Time frame: baseline and 6 months
Absolute Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS)
Time frame: baseline and 6 months
Percent Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS)
Time frame: baseline and 24 weeks

Results and safety

Results posted to the registry on 29 Dec 2011, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Mid Dose Idebenone	22	22
High Dose Idebenone	24	22
Placebo	24	24

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

Time frame: baseline and 6 months. Mean (Standard Deviation). Unit: ICARS points

Measure	Mid Dose Idebenone n = 22	High Dose Idebenone n = 24	Placebo n = 24
Value	-2.5 (6.3)	-2.4 (4.8)	-1.3 (4.4)

11 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 1% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Mid Dose Idebenone	0 of 22 (0%)	18 of 22 (81.8%)	Not reported
High Dose Idebenone	2 of 24 (8.3%)	23 of 24 (95.8%)	Not reported
Placebo	0 of 24 (0%)	21 of 24 (87.5%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 11 Jul 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 21779958Meier 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.
Derived from this study: PubMed 21392622Lagedrost 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.
Derived from this study: PubMed 20697044Lynch 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.

Related trials for Friedreich's Ataxia

RecruitingPhase 1
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
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Active, not recruiting
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
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Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Susan Perlman, MD principal investigator

University of California, Los Angeles

David Lynch, MD principal investigator

Children's Hospital of Philadelphia

2 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Idebenone in people with Friedreich's Ataxia, run by Santhera Pharmaceuticals at 2 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 11 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 29 Dec 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24 (time frame: baseline and 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00537680, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jul 2023.