Tolerance and Efficacy Nicotinamide (vitamin B3) in Dominant Optic Atrophy OPA1

NCT ID: NCT06007391

Last Updated: 2025-02-24

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 25 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-01-23
• Study Completion Date: 2026-09-30

Brief Summary

Dominant Optic Atrophy (hereafter known as DOA) is a neurodegenerative pathology of the optic nerve inducing progressive loss of central visual field and visual acuity. There is currently no proven treatment for this disease. The metabolomics work of Pascal Reynier's team revealed a specific metabolomic signature of DOA in the plasma of patients. This metabolomic signature revealed a relative deficiency in nicotinamide compared to a control population, a vitamin compound (vitamin B3) known to be neuroprotective for the optic nerve and mitochondria. Note that the investigator have also identified this nicotinamide deficiency in primary open-angle glaucoma and Leber's hereditary optic neuropathy, the other most common cause of hereditary optic neuropathy, these three optic nerve conditions sharing a common pathophysiological mechanism of mitochondrial deficit. In addition, an American team demonstrated the high neuroprotective power on the optic nerve of nicotinamide in a mouse model of glaucoma. These arguments converge towards the potential therapeutic interest of this vitamin in degenerative pathologies of the optic nerve. This is encouraged by the fact that two randomized clinical trials have confirmed a benefit of nicotinamide in glaucoma. The objective of this pilot study is to test the tolerance and efficacy of nicotinamide in DOA and DOA+ patients.

Conditions

Nicotinamide Adverse Reaction

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

nicotinamide

Group Type: EXPERIMENTAL

Nicotinamide

Intervention Type: DRUG

nicotinamide 3g per day

Interventions

Nicotinamide

nicotinamide 3g per day

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Adult patients
• Patients with DOA or DOA+ due to a heterozygous pathogenic variant in the OPA1 gene
• Naïve patients (> 3 months) in terms of taking nicotinamide
• Patients able to take oral medication and comply with specific study procedures
• Patients affiliated or beneficiaries of a social security scheme
• Signature of voluntary, free and informed consent to participate in the study

Exclusion Criteria

• Asymptomatic patients (= healthy carriers of an OPA1 mutation but not having developed optic neuropathy)
• Patients with another associated severe ophthalmological pathology (advanced glaucoma, retinal pathology, etc.)
• Patients treated with Idebenone
• Patients with a level of transaminase(s) (ASAT and/or ALAT) twice higher than the high normal value.
• Pregnant, breastfeeding or parturient women
• Patients with a contraindication to nicotinamide
• Persons deprived of liberty by administrative or judicial decision
• Patients subject to a legal protection measure
• Persons undergoing psychiatric treatment under duress
• Persons unable to express their consent

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University Hospital, Angers

OTHER_GOV

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Angers University Hospital

Angers, , France

Site Status: RECRUITING

Countries

France

Central Contacts

Pascal Reynier

Role: CONTACT

PaReynier@chu-angers.fr

0241355542

Facility Contacts

Pascal REYNIER

Role: primary

PaReynier@chu-angers.fr

02 41 35 36 37

Other Identifiers

49RC23_0195

Identifier Type: -

Identifier Source: org_study_id