Freidreich's AtaxiaPhase 3NCT00993967
Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension)
A Phase 3 study testing idebenone in people with Freidreich's Ataxia, run by Santhera Pharmaceuticals at 11 sites in 5 countries.
Trial summary
This is an Extension study of the MICONOS main randomised placebo-controlled trial (NCT00905268), and open to those patients completing the main study. The scientific aim of this extension study is to monitor safety and tolerability of idebenone over two years in patients with Friedreich's Ataxia. Read more about this study
- Who is it for?
- Ages 9 years and older
- Conditions the study is for
- Freidreich's Ataxia
- What is being tested?
- idebenone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 200 participants
- Where
- 11 sitesin 5 countries
- Study dates
- Jun 2007to Jun 2012
- 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 9 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Completion of 52 weeks in study SNT-III-001
- Body weight ≥ 25 kg
- Negative urine pregnancy test
- Eligibility to participate in the present extension study as confirmed by investigator
Who cannot join
- Safety or tolerability issues arising during the course of SNT-III-001 which in the opinion of the investigator preclude further treatment with idebenone
- Clinically significant abnormalities of haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of SGOT, SGPT or creatinine
- Parallel participation in another clinical drug trial
- Pregnancy or breast-feeding
- Abuse of drugs or alcohol
Trial locations and contacts
11 sites in 5 countries.
11 sites in 5 countries
Germany7 sites
HELIOS Klinikum Berlin
Berlin, 13125
Neurologische Universitätsklinik und Ploklinik - Universitätsklimikum Bonn
Bonn, 53105
Klinik II, Neuropädiatrie und Muskelerkrankungen - Universitätsklinik Freiburg
Freiburg im Breisgau, 79106
Zentrum für Neurologische Medizin
Göttingen, 37073
UKE Hamburg Neuropädiatrie - Zentrum für Frauen, Kinder und Jugendmedizin
Hamburg, 20246
Neurologische Klinik - Klinikum Grosshadern
München, 81377
Neurologische Universitätsklinik und Poliklinik
Tübingen, 72076
France1 site
Hôpital de la Salpétrière - INSERM U679, Neurologie et Thérapeutique expérimentale
Paris, 75651
About this study
Official title: A Phase III Open-Label, Single-Group, Extension Study to Obtain Long-Term Safety and Tolerability Data of Idebenone in the Treatment of Friedreich's Ataxia Patients.
This is an Extension study of the MICONOS main randomised placebo-controlled trial (NCT00905268), and open to those patients completing the main study. The scientific aim of this extension study is to monitor safety and tolerability of idebenone over two years in patients with Friedreich's Ataxia.
- Conditions
- Freidreich's Ataxia
- Being tested
- idebenone
- Keywords
- FRDA, idebenone, FRDA disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
idebenoneDrug
Idebenone 1350 mg/d, patients < or equal 45 kg Idebenone 2250 mg/d, patients > 45 kg
| Study group | Role | What they receive |
|---|---|---|
| Idebenone | Experimental | 1350 mg/day or 2250 mg/day for patients weighing ≤45 kg or >45 kg, respectively.In case of poor tolerability, dose reduction to 450 mg/day or 900 mg/day, respectively, were allowed. Receives: Drug: idebenone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)
Global Overvbiew of accurance of AEs-Safety population. The Safety population included all subjects who received at least one dose of the study medication.
Measured: overall study, up to 24 months
Absolute Change in The International Cooperative Ataxia Rating Scale (ICARS)
The International Cooperative Ataxia Rating Scale (ICARS) is a commonly used evaluation and is composed of four clinical sub-scores involving the following: posture and gait, limb coordination, speech and oculomotor function.The ICARS score is the total sum of the sub scores and ranges from 0 to 100, with 100 indicative of the most severely affected outcome.
Measured: Baseline, Month 12 and month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2009 (estimated)
- Study startJun 2007
- Primary completionJun 2012
- Study completionJun 2012
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.
- Other trials for Freidreich'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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 200 participants (actual)
Sponsor and investigators
- Lead sponsor
- Santhera Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Nick Wood, Professor, Principal investigator, Dept of Molecular Neuroscience, Institute of Neurology. The National Hospital, University College London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00993967
- Sponsor's protocol ID
- SNT-III-001-E
- Status last verified
- Aug 2017
- First submitted
- 13 Oct 2009
- Last updated
- 5 Mar 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III Open-Label, Single-Group, Extension Study to Obtain Long-Term Safety and Tolerability Data of Idebenone in the Treatment of Friedreich's Ataxia Patients.
- ClinicalTrials.gov ID
- NCT00993967
- Acronym / sponsor ID
- SNT-III-001-E
- Conditions
- Freidreich's Ataxia
- Keywords
- FRDA, idebenone, FRDA disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 200 participants (actual)
- Lead sponsor
- Santhera PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Jun 2007
- Primary completion
- Jun 2012 (actual)
- Study completion
- Jun 2012 (actual)
- First posted
- 14 Oct 2009 (estimated)
- Results
- Posted 5 Mar 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an Extension study of the MICONOS main randomised placebo-controlled trial (NCT00905268), and open to those patients completing the main study. The scientific aim of this extension study is to monitor safety and tolerability of idebenone over two years in patients with Friedreich's Ataxia.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Idebenone | Experimental | Drug: idebenone1350 mg/day or 2250 mg/day for patients weighing ≤45 kg or >45 kg, respectively.In case of poor tolerability, dose reduction to 450 mg/day or 900 mg/day, respectively, were allowed. |
Who may qualify
- Ages
- 9 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Completion of 52 weeks in study SNT-III-001
- Body weight ≥ 25 kg
- Negative urine pregnancy test
- Eligibility to participate in the present extension study as confirmed by investigator
Key exclusion criteria (5 listed)
- Safety or tolerability issues arising during the course of SNT-III-001 which in the opinion of the investigator preclude further treatment with idebenone
- Clinically significant abnormalities of haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of SGOT, SGPT or creatinine
- Parallel participation in another clinical drug trial
- Pregnancy or breast-feeding
- 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 |
|---|---|---|
| idebenoneIdebenone 1350 mg/d, patients < or equal 45 kg Idebenone 2250 mg/d, patients > 45 kg | Drug | Idebenone |
Primary outcome
Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)
Time frame: overall study, up to 24 months
Global Overvbiew of accurance of AEs-Safety population. The Safety population included all subjects who received at least one dose of the study medication.
Absolute Change in The International Cooperative Ataxia Rating Scale (ICARS)
Time frame: Baseline, Month 12 and month 24
The International Cooperative Ataxia Rating Scale (ICARS) is a commonly used evaluation and is composed of four clinical sub-scores involving the following: posture and gait, limb coordination, speech and oculomotor function.The ICARS score is the total sum of the sub scores and ranges from 0 to 100, with 100 indicative of the most severely affected outcome.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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.
Nick Wood, Professor principal investigator
Dept of Molecular Neuroscience, Institute of Neurology. The National Hospital, University College London
11 sites in 5 countries: 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 Freidreich's Ataxia, run by Santhera Pharmaceuticals at 11 sites in 5 countries.
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 4 inclusion and 5 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 5 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in Germany, Austria, Belgium, France, Netherlands. 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. 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 5 Mar 2018). 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?
Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs) (time frame: overall study, up to 24 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 5 Mar 2018.