Autosomal dominant optic atrophy (ADOA) MedDRA version: 20.0 Level: PT Classification code 10019895 Term: Hereditary optic atrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Raxone 150 mg film-coated tablets
Product Name: Raxone 150 mg film-coated tablets
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Idebenone
CAS Number: 58186-27-9
Other descri
Sponsors
Medizinische Universität Graz, Univ.-Augenklinik
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: ADOA patients with confirmed OPA1 mutation Age of 12 years or more Willingness and ability to comply with study related procedures Informed consent Are the trial subjects under 18? yes Number of subjects for this age range: 3 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 13 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: - patients in the state of pregnancy or lactation at the time of the examination or a planned pregnancy within the next 12 months - patients with allergies or hypersensitivity to the active substance or to any of the excipients contained in Raxone® - patients with hereditary diseases like galactose intolerance, the Lapp lactase deficiency or glucose-galactose malabsorption - patients with a high-grade hepatic or renal impairment - previous treatment with idebenone - patients with other diseases which would limit the compliance required for trial participation Before the prescription of the trial medication, the clinical investigator will conduct a detailed medication anamnesis. If there are possible drug interactions, the patient can be excluded from the trial - patients who have participated in other pharmaceutical product or medicine related trials in the last 3 months - Patients with a glaucoma or with any optic neuropathy other than ADOA - Patients with a baseline best- corrected- visual acuity less than counting fingers
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Evaluation of the therapeutic effect of 900mg Raxone® per day regarding visual acuity in ADOA patients with OPA1 mutation within a 12 month period. ;Primary end point(s): Best recovery/ least deterioration of visual acuity from baseline to 12 months measured with ETDRS charts (converted to logMAR). ;Timepoint(s) of evaluation of this end point: 12 months;Secondary Objective: Evaluation of the therapeutic effect of 900mg Raxone® per day in ADOA patients with OPA1 gene mutation regarding visual field, colour vision, contrast vision, change of the retinal nerve fibre layer (RNFL) and change of the visual performance- related quality of life (NEI-VFQ) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - change of visual acuity on the better eye at the time of baseline examination measured with ETDRS charts (in logMAR) - change of visual acuity of both eyes measured with ETDRS charts (in logMAR) - change of visual field measured with Goldmann and/or Octopus perimetry - change of colour vision measured with Ishihara plates - change of contrast vision measured with Pelli Robson charts - change of retinal nerve fibre layer (OCT) - change of visual performance- related quality of life measured with National Eye Institute Visual Function Questionnaire (NEI-VFQ) - change of all primary and secondary outcome measures ;Timepoint(s) of evaluation of this end point: 12 months | — |
Countries
Austria
Contacts
Public ContactClinical trials information
Medizinische Universität Graz, Univ.-Augenklinik
Outcome results
None listed