Idebenone versus placebo in dominant optic atrophy

Phase 2

• Conditions: Dominant Optic Atrophy; Mitochondrial Disease; Neurological - Other neurological disorders; Eye - Diseases / disorders of the eye; Human Genetics and Inherited Disorders - Other human genetics and inherited disorders

• Registration Number: ACTRN12621000826842
• Lead Sponsor: orthern Sydney Local Health District

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-06-28
• Last Update Posted: 5 July 2021

Recruitment & Eligibility

• Status: ot yet recruiting
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

(i)Dominant Optic Atrophy confirmed by pathogenic OPA1 mutation
(ii)Best-corrected visual acuity of logMAR 0.1 or worse in at least one eye
(iii)Primary cause of reduced visual acuity must be DOA/DOA plus
(iv)Aged 14 years to 85 years

Exclusion Criteria

(i)Pregnant women
(ii)Women planning to become pregnant during the course of the study
(iii)Women who are currently breastfeeding
(iv)eGFR < 30
(v)Elevations greater than 3 times the upper limit of normal of AST or ALT and/or cirrhosis
(vi)Lactose intolerance
(vii)Cognitive impairment that in the investigator’s opinion would render the participant unable to participate in the assessments
(viii)Weekly alcohol intake greater than 35 units for males or 24 units for females
(ix)Current drug abuse
(x)Participation in another clinical trial of any investigational drug within 3 months prior to baseline
(xi)Primary cause of reduced visual acuity is not DOA
(xii)History of amblyopia
(xiii)Unable to reliably perform HVF or OCT tests
(xiv)Other condition that in the investigator’s opinion is likely to interfere with the evaluation of the patient’s safety and of the study outcome.

Study & Design

• Study Type: Interventional
• Study Design: Not specified