Friedreich's AtaxiaPhase 3PROTINCT01303406
Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)
A Phase 3 study testing Idebenone in people with Friedreich's Ataxia, run by Santhera Pharmaceuticals at 6 sites in 4 countries.
Trial summary
This is a Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study. The aim is to further investigate the effects of idebenone in patients with Friedreich's ataxia. The objective of the PROTI study is to establish whether patients can correctly determine which treatment assignment (placebo or idebenone) they… Read more about this study
- Who is it for?
- Ages 10 years and older
- 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
- 29 participants
- Where
- 6 sitesin 4 countries
- Study dates
- Apr 2011to Jul 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 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Completion of V5 (Month 12), V6 (Month 18), or V7 (Month 24) in the MICONOS extension study
- Patients who in the opinion of the investigator are able to comply with the requirements of the study
- Body weight ≥ 25kg
- Negative urine pregnancy test
Who cannot join
- AE during the course of the MICONOS extension study which in the opinion of the investigator is attributable to idebenone and precludes further treatment with idebenone
- Clinically significant abnormalities of clinical haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of normal SGOT, SGPT or creatinine
- Parallel participation in another clinical drug trial
- Pregnancy or breast-feeding
- Abuse of drugs or alcohol
- Any change of concomitant medication within the last 30 days that in the opinion of the investigator the intake could negatively impact the study
Trial locations and contacts
6 sites in 4 countries.
6 sites in 4 countries
United Kingdom1 site
The National Hospital, University College London
London, WC 1N 3BG
About this study
Official title: A Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study of Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone
This is a Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study. The aim is to further investigate the effects of idebenone in patients with Friedreich's ataxia.
The objective of the PROTI study is to establish whether patients can correctly determine which treatment assignment (placebo or idebenone) they received during the randomised phase of the trial, and identify any potential changes on symptoms or activities.
- Conditions
- Friedreich's Ataxia
- Being tested
- Idebenone, Placebo
- Keywords
- randomized withdrawal, idebenone, friedreich's ataxia, Miconos, Patient reported outcome, 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
Also called Catena (approved name in Canada)
All PROTI patients randomised to idebenone treatment will receive high dose idebenone. This is defined according to body weight. In patients weighing 45 kg or less, it is 1350 mg/day (3 x 150 mg tablets three times per day with meals). In patients weighing more than 45 kg, it is 2250 mg/day (5 x 150 mg tablets three times per day with meals).
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Following the body weight, patients will be allocated to one of the following regimen:
Placebo Patients < 45 kg - 3 tablets 3 times a day with meals
Placebo Patients > 45 kg - 5 tablets 3 times a day with meals Receives: Drug: Placebo |
| idebenone | Experimental | Following the body weight, patients will be allocated to one of the following regimen:
Idebenone Patients < 45 kg - 3 tablets 3 times a day with meals
Idebenone Patients > 45 kg - 5 tablets 3 times a day with meals Receives: Drug: Idebenone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone
The primary efficacy endpoint was the comparison of the number of patients randomized to idebenone and placebo, who assessed that they received idebenone treatment.
Measured: At 2 months after study start
Secondary outcomes (1)
Comparison of the Percentage of Participants Randomised to Idebenone and Placebo Who Withdrew Early Due to Recurrence or Worsening of FRDA Symptoms
There was no Withdrawal due to recurrence or worsening of FRDA symptoms
Measured: Within 2 months (i.e. Early withdrawal visit)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2011 (estimated)
- Study startApr 2011
- Primary completionJul 2012
- Study completionJul 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 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
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- Santhera Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Paola Giunti, M.D, Principal investigator, Institute of Neurology, The National Hospital, University College London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01303406
- Sponsor's protocol ID
- SNT-III-004
- Acronym
- PROTI
- Status last verified
- Feb 2016
- First submitted
- 22 Feb 2011
- Last updated
- 9 Mar 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study of Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone
- ClinicalTrials.gov ID
- NCT01303406
- Acronym / sponsor ID
- PROTI / SNT-III-004
- Conditions
- Friedreich's Ataxia
- Keywords
- randomized withdrawal, idebenone, friedreich's ataxia, Miconos, Patient reported outcome, ICARS
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 29 participants (actual)
- Lead sponsor
- Santhera PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Apr 2011
- Primary completion
- Jul 2012 (actual)
- Study completion
- Jul 2012 (actual)
- First posted
- 24 Feb 2011 (estimated)
- Results
- Posted 9 Mar 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study. The aim is to further investigate the effects of idebenone in patients with Friedreich's ataxia.
The objective of the PROTI study is to establish whether patients can correctly determine which treatment assignment (placebo or idebenone) they received during the randomised phase of the trial, and identify any potential changes on symptoms or activities.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboFollowing the body weight, patients will be allocated to one of the following regimen: Placebo Patients < 45 kg - 3 tablets 3 times a day with meals Placebo Patients > 45 kg - 5 tablets 3 times a day with meals |
| idebenone | Experimental | Drug: IdebenoneFollowing the body weight, patients will be allocated to one of the following regimen: Idebenone Patients < 45 kg - 3 tablets 3 times a day with meals Idebenone Patients > 45 kg - 5 tablets 3 times a day with meals |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Completion of V5 (Month 12), V6 (Month 18), or V7 (Month 24) in the MICONOS extension study
- Patients who in the opinion of the investigator are able to comply with the requirements of the study
- Body weight ≥ 25kg
- Negative urine pregnancy test
Key exclusion criteria (6 listed)
- AE during the course of the MICONOS extension study which in the opinion of the investigator is attributable to idebenone and precludes further treatment with idebenone
- Clinically significant abnormalities of clinical haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of normal 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 |
|---|---|---|
| IdebenoneAlso: Catena (approved name in Canada)All PROTI patients randomised to idebenone treatment will receive high dose idebenone. This is defined according to body weight. In patients weighing 45 kg or less, it is 1350 mg/day (3 x 150 mg tablets three times per day with meals). In patients weighing more than 45 kg, it is 2250 mg/day (5 x 150 mg tablets three times per day with meals). | Drug | idebenone |
| Placebo | Drug | Placebo |
Primary outcome
Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone
Time frame: At 2 months after study start
The primary efficacy endpoint was the comparison of the number of patients randomized to idebenone and placebo, who assessed that they received idebenone treatment.
1 secondary outcome
Comparison of the Percentage of Participants Randomised to Idebenone and Placebo Who Withdrew Early Due to Recurrence or Worsening of FRDA Symptoms
Time frame: Within 2 months (i.e. Early withdrawal visit)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Paola Giunti, M.D principal investigator
Institute of Neurology, The National Hospital, University College London
6 sites in 4 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 Friedreich's Ataxia, run by Santhera Pharmaceuticals at 6 sites in 4 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 6 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 9 Mar 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Germany, Austria, Netherlands, United Kingdom. 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 9 Mar 2016). 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?
Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone (time frame: At 2 months after study start). 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 9 Mar 2016.