- To gather long-term data on the safety and tolerability of idebenone inFriedreich*s Ataxia- To explore the effect of idebenone after longer term administration onneurological symptoms and signs as assessed by the InternationalCooperative Ataxia…
ID
Source
Brief title
Condition
- Congenital cardiac disorders
- Musculoskeletal and connective tissue disorders congenital
- Congenital and peripartum neurological conditions
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Measures of safety and tolerability
- nature and frequency of AEs
- haematological and biochemical parameters
- physical examinations and vital signs
- ECGs
Absolute change in scores from baseline to Month 12, and to Month 24 assessed
by:
- International Cooperative Ataxia Rating Scale (ICARS)
- Friedreich*s Ataxia Rating Scale (FARS)
Secondary outcome
Not Applicable
Background summary
A phase III study is being conducted to investigate efficacy, safety and
tolerability of idebenone in the treatment of patients with Friedreich*s Ataxia
over a 52 week treatment period. Having completed the treatment period patients
who wish to enter into this open-label extension study SNT-III-001-E may do so
if they are found to be eligible by the investigator. The scientific aim of the
present study is to monitor long-term safety and tolerability of idebenone in
patients with Friedreich*s Ataxia.
Study objective
- To gather long-term data on the safety and tolerability of idebenone in
Friedreich*s Ataxia
- To explore the effect of idebenone after longer term administration on
neurological symptoms and signs as assessed by the International
Cooperative Ataxia Rating Scale (ICARS) and the Friedreich*s Ataxia Rating
Scale (FARS)
Study design
The baseline visit should coincide with Visit 7/Week 52 of Study SNT-III-001.
Safety assessments
will be performed at every visit. Neurological assessments will be performed at
Visit
1/Baseline, Visit 5/Month 12 and Visit 7/Month 24. The study period is planned
up to 24
months or until marketing approval/reimbursement of idebenone is achieved in
the respective
country (after 52 weeks in study SNT-III-001), whichever is shorter.
Intervention
All patients will get idebenone, there is no placebo arm.
Study burden and risks
As with any experimental medication, the medication used in this study may
cause some unexpected side effects, which are not known at the present time.
Blood sampling may cause some discomfort for a short time. The amount of blood
to be taken will not cause any health problems.
The doctor will be measuring the patient*s progress at each visit. If he/she
feels that there is any risk at any time, he/she will remove the patient from
the study.
All patients will get idebenone, there is no placebo arm. We hope that the
treatment will help the patients, but it is not yet known whether idebenone
will help their illness. However, other people with a similar condition may
benefit from the knowledge obtained from this trial.
Hammerstrasse 47
CH-4410 Liestal
CH
Hammerstrasse 47
CH-4410 Liestal
CH
Listed location countries
Age
Inclusion criteria
- Completion of 52 weeks in study SNT-III-001
- Body weight >= 25kg
- Negative urine pregnancy test
- Eligibility to participate in the present extension study as confirmed by the
investigator
Exclusion criteria
- Safety or tolerability issues arising during the course of SNT-III-001 which in
the opinion of the investigator preclude further treatment with idebenone
- Clinically significant abnormalities of haematology or biochemistry including,
but not limited to, elevations greater than 1.5 times the upper limit of SGOT,
SGPT or creatinine
- Parallel participation in another clinical drug trial
- Pregnancy or breast-feeding
- Abuse of drugs or alcohol
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2007-001646-40-NL |
| CCMO | NL23974.042.08 |