Raxone® treatment for patients with autosomal dominant optic atrophy (inherited optic nerve disorder)

Phase 1

• Conditions: Autosomal dominant optic atrophy (ADOA); MedDRA version: 20.0Level: PTClassification code 10019895Term: Hereditary optic atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Eye Diseases [C11]

• Registration Number: EUCTR2019-001493-28-AT
• Lead Sponsor: Medizinische Universität Graz, Univ.-Augenklinik

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-09-09
• Last Update Posted: 21 August 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 16

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

- 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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified