Idebenone versus placebo in dominant optic atrophy

Phase II randomised, double blinded, placebo-controlled, proof-of-concept study of the efficacy of idebenone in dominant optic atrophy

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

ANZCTR

Registry ID

ACTRN12621000826842

Enrollment

Registered

2021-06-28

Start date

2021-07-13

Completion date

2021-07-14

Last updated

2021-07-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

Brief summary

Patients with an OPA1 mutation inherit a condition called dominant optic atrophy (DOA) which causes vision loss over time. A medication called idebenone has been shown to decrease the vision loss in patients with a similar condition, called Leber's Hereditary Optic Neuropathy, and thus this trial will investigate whether similar results can be seen in patients with dominant optic atrophy. The study will be a blinded randomised placebo-controlled study. One arm of the study will include participants with DOA who will receive idebenone, and the second arm will include participants with DOA who will receive a placebo. At the end of 12 months, the groups will be investigated to see if there is a between group difference in the rate of visual decline.

Interventions

Idebenone in Dominant Optic Atrophy will be a placebo controlled trial, the intervention will include 20 participants with genetically confirmed OPA1 mutation who will receive idebenone 300mg by mouth Three times per day (900mg daily) for 12 months. Adherence to intervention will be monitored by the clinical trials pharmacy by checking the number of capsules left at the visits that happen every three months during the intervention.

Study design

Allocation

Randomised controlled trial

Intervention model

Parallel

Primary purpose

Treatment

Masking

Blinded (masking used) (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

All

Age

14 Years to 85 Years

Healthy volunteers

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.

Outcome results