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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00697073
First received: June 11, 2008
Last updated: May 24, 2013
Last verified: May 2013
  Purpose

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


Condition Intervention Phase
Friedreich's Ataxia
Drug: Idebenone
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase III Open-Label, Single Group Extension Study of the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

Resource links provided by NLM:


Further study details as provided by Santhera Pharmaceuticals:

Primary Outcome Measures:
  • Change in ICARS [ Time Frame: baseline and 12 months ] [ Designated as safety issue: No ]

    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.

    Scores for each subscale quantify the extent of ataxia in each clinically important area. Subscale scores are summed to give a total score ranging from 0 (best) to 100 (worst).



Secondary Outcome Measures:
  • FARS (Friedreich's Ataxia Rating Scale) [ Time Frame: baseline and 12 Months ] [ Designated as safety issue: No ]
  • Nature and Frequency of Adverse Events [ Time Frame: 12 Months ] [ Designated as safety issue: Yes ]

Enrollment: 68
Study Start Date: July 2008
Study Completion Date: May 2010
Primary Completion Date: May 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

Detailed Description:

The study involves 6 clinic visits.

  Eligibility

Ages Eligible for Study:   8 Years to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00697073

Locations
United States, California
David Geffen School of Medicine, UCLA
Los Angeles, California, United States, 90095-1769
United States, Pennsylvania
The Children's Hopsital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Santhera Pharmaceuticals
Investigators
Principal Investigator: Susan Perlman, MD University of California, Los Angeles
Principal Investigator: David Lynch, MD Children's Hospital of Philadelphia
  More Information

Publications:
Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00697073     History of Changes
Other Study ID Numbers: SNT-III-002-E
Study First Received: June 11, 2008
Results First Received: June 7, 2011
Last Updated: May 24, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Santhera Pharmaceuticals:
Friedreich's Ataxia
Idebenone
ICARS

Additional relevant MeSH terms:
Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinocerebellar Degenerations
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Idebenone
Antioxidants
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Protective Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on July 20, 2014