Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients (IONIA-E)

June 22, 2023 updated by: Santhera Pharmaceuticals

A Phase III Open-Label, Single Group Extension Study of the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

This study is meant to assess the safety and tolerability of idebenone in patients with Friedreich's Ataxia over a 12 months period.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

The study involves 6 clinic visits.

Study Type

Interventional

Enrollment (Actual)

68

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

8 years to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Inclusion criteria:

  • Friedreich's ataxia patients completing core study SNT-III-002 (NCT00537680) and presenting at Week 24 (Visit 5) of that study
  • Body weight ≥ 25kg/55 lbs
  • Negative urine pregnancy test
  • Patients who in the opinion of the investigator are able to comply with the requirements of this study

Exclusion criteria:

  • Adverse events during the course of SNT-III-002(NCT00537680)which in the opinion of the investigator are attributable to idebenone and preclude further treatment with idebenone
  • Clinically significant abnormalities of clinical hematology or biochemistry including, but not limited to, elevations greater than 2 times the upper limit of normal of AST, ALT or creatinine
  • Treatment with coenzyme Q10, vitamin E (if taken at a dose 5 times above the daily requirement) or other sources of idebenone within the past month
  • Parallel participation in another clinical drug trial
  • Past or present history of abuse of drugs or alcohol
  • Pregnancy or breast-feeding

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 1
high dose Idebenone
Drug: Idebenone
Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in ICARS
Time Frame: baseline and 12 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.

A total score of 0 points represents the best possible score, and a value of 100 points is the worst possible score. Therefore, a negative change in ICARS score represents an improvement. A higher score also indicates more disability.
baseline and 12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
FARS (Friedreich's Ataxia Rating Scale)
Time Frame: baseline and 12 Months
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 consists of three subscales, comprising a general score for ataxia, a score for activities of daily living (ADL) and a neurological examination. The scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability.
baseline and 12 Months
Nature of Adverse Events
Time Frame: 12 Months
12 Months
Frequency/Number of Mild, Moderate, and Severe Adverse Events
Time Frame: 12 months
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Santhera Pharmaceuticals

Investigators

  • Principal Investigator: David Lynch, MD, Children's Hospital of Philadelphia
  • Principal Investigator: Susan Perlman, MD, University of California, Los Angeles

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

July 1, 2008

Primary Completion (Actual)

May 1, 2010

Study Completion (Actual)

May 1, 2010

Study Registration Dates

First Submitted

June 11, 2008

First Submitted That Met QC Criteria

June 12, 2008

First Posted (Estimated)

June 13, 2008

Study Record Updates

Last Update Posted (Actual)

July 11, 2023

Last Update Submitted That Met QC Criteria

June 22, 2023

Last Verified

June 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SNT-III-002-E

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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