Pilot study of safety and efficacy of nicotinamide (vitamin B3) in OPA1 Dominant Optic Atrophy - NICOPA1-TOL

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-506214-52-00

Enrollment

Registered

2023-09-13

Start date

2023-12-27

Completion date

Unknown

Last updated

2024-12-19

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

Conditions

Dominant Optic Atrophy

Brief summary

The primary endpoint will be assessed by the number of patients with reported ophthalmological and/or neurological adverse events.

Detailed description

Number of patients with improved visual function at follow-up visits compared with baseline at inclusion., Increase in plasma and whole blood nicotinamide levels in all patients, comparing baseline at inclusion with two follow-up periods., Differences on the NEI-VFQ-25 scale between M0 and M6, Clinical neurological improvement at follow-up visits compared with baseline at inclusion.

Interventions

DRUGNICOBION 500 mg

DRUGcomprimé pelliculé

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Secondary

Measure	Time frame
Number of patients with improved visual function at follow-up visits compared with baseline at inclusion., Increase in plasma and whole blood nicotinamide levels in all patients, comparing baseline at inclusion with two follow-up periods., Differences on the NEI-VFQ-25 scale between M0 and M6, Clinical neurological improvement at follow-up visits compared with baseline at inclusion.	—

Primary

Measure	Time frame
The primary endpoint will be assessed by the number of patients with reported ophthalmological and/or neurological adverse events.	—

Countries

France

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026