Phase 3 N=29 Randomized Quadruple-blind Treatment

Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)

Friedreich's Ataxia

Bottom Line

View on ClinicalTrials.gov: NCT01303406 ↗

Enrolled (actual)

Serious AEs

3.5%

Results posted

Mar 2016

Primary outcome: Primary: Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone — 6; 8 participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Idebenone (Drug); Placebo (Drug)
Age: Pediatric, Adult, Older Adult · 10+ yrs
Sex: All
Sponsor: Santhera Pharmaceuticals
Primary completion: Jul 2012

Outcome Measures

Outcome	Result	p-value
PRIMARY Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone	6; 8	—
SECONDARY Comparison of the Percentage of Participants Randomised to Idebenone and Placebo Who Withdrew Early Due to Recurrence or Worsening of FRDA Symptoms	0; 0	—

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 received during the randomised phase of the trial, and identify any potential changes on symptoms or activities.

Eligibility Criteria

Inclusion Criteria

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

Exclusion Criteria

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01303406). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.