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

• Sponsor: Santhera Pharmaceuticals (Industry)
• Overall Status: Completed
• CT.gov ID: NCT00697073
• Collaborator: (none)
• Enrollment: 68
• Locations: 2
• Arm: 1
• Duration (Months): 22
• Patients Per Site: 34
• Patients Per Site Per Month: 1.5

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Friedreich's Ataxia	Drug: Idebenone	Phase 3

Detailed Description

The study involves 6 clinic visits.

Study Design

• Study Type: Interventional
• Actual Enrollment: 68 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (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
• Study Start Date: Jul 1, 2008
• Actual Primary Completion Date: May 1, 2010
• Actual Study Completion Date: May 1, 2010

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Change in ICARS [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. 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

1. FARS (Friedreich's Ataxia Rating Scale) [baseline and 12 Months]

2. Nature and Frequency of Adverse Events [12 Months]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 8 Years to 18 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Locations

	Site	City	State	Country	Postal Code
1	David Geffen School of Medicine, UCLA	Los Angeles	California	United States	90095-1769
2	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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Santhera Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT00697073
• Other Study ID Numbers: SNT-III-002-E
• First Posted: Jun 13, 2008
• Last Update Posted: Oct 18, 2021
• Last Verified: Sep 1, 2021

• Keywords provided by Santhera Pharmaceuticals: Friedreich's Ataxia; Idebenone; ICARS
• Additional relevant MeSH terms: Ataxia; Cerebellar Ataxia; Friedreich Ataxia; Idebenone

Study Results

Participant Flow

Arm/Group Title	High Dose Idebenone
Arm/Group Description	Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day (3 x 150 mg tablets, t.i.d.) Patients > 45 kg/99 lbs: idebenone 2250 mg/day (5 x 150 mg tablets, t.i.d.)
Period Title: Overall Study
STARTED	68
COMPLETED	59
NOT COMPLETED	9

Baseline Characteristics

Arm/Group Title	High Dose Idebenone
Arm/Group Description	Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day (3 x 150 mg tablets, t.i.d.) Patients > 45 kg/99 lbs: idebenone 2250 mg/day (5 x 150 mg tablets, t.i.d.)
Overall Participants	68
Age (Count of Participants)
<=18 years	68 100%
Between 18 and 65 years	0 0%
>=65 years	0 0%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	14.0 (2.72)
Sex: Female, Male (Count of Participants)
Female	36 52.9%
Male	32 47.1%
Region of Enrollment (participants) [Number]
United States	68 100%

Outcome Measures

1. Primary Outcome

Title	Change in ICARS
Description	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).
Time Frame	baseline and 12 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	High Dose Idebenone
Arm/Group Description	Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day (3 x 150 mg tablets, t.i.d.) Patients > 45 kg/99 lbs: idebenone 2250 mg/day (5 x 150 mg tablets, t.i.d.)
Measure Participants	68
Mean (Standard Deviation) [ICARS points]	1.1 (5.64)

2. Secondary Outcome

Title	FARS (Friedreich's Ataxia Rating Scale)
Time Frame	baseline and 12 Months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title

Arm/Group Description

3. Secondary Outcome

Title	Nature and Frequency of Adverse Events
Time Frame	12 Months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title

Arm/Group Description

Adverse Events

Arm/Group Title	High Dose Idebenone
Arm/Group Description	Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day (3 x 150 mg tablets, t.i.d.) Patients > 45 kg/99 lbs: idebenone 2250 mg/day (5 x 150 mg tablets, t.i.d.)
All Cause Mortality
	High Dose Idebenone
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	High Dose Idebenone
	Affected / at Risk (%)	# Events
Total	4/68 (5.9%)
Cardiac disorders
cardiac failure	1/68 (1.5%)	1
tachycardia	1/68 (1.5%)	1
Endocrine disorders
Hyperthyroidism	1/68 (1.5%)	1
General disorders
Hypotension	1/68 (1.5%)	1
Deep vein thrombosis	1/68 (1.5%)	1
Dehydratation	1/68 (1.5%)	1
Infections and infestations
Pharyngitis streptococcal	1/68 (1.5%)	1
Pneumonia	1/68 (1.5%)	1
Pregnancy, puerperium and perinatal conditions
Pregnancy	1/68 (1.5%)	1
intra uterine death	1/68 (1.5%)	1
abortion spontaneous	1/68 (1.5%)	1
Other (Not Including Serious) Adverse Events
	High Dose Idebenone
	Affected / at Risk (%)	# Events
Total	66/68 (97.1%)
Gastrointestinal disorders
nausea	15/68 (22.1%)
General disorders
headache	21/68 (30.9%)
pyrexia	14/68 (20.6%)
Infections and infestations
upper respiratory tract infection	18/68 (26.5%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.