Using Nicotinamide to Treat Dominant Optic Atrophy
Pilot Study of Tolerance and Efficacy Nicotinamide (vitamin B3) in Dominant Optic Atrophy OPA1
This study is testing if a vitamin B3 supplement called nicotinamide can help improve vision in adults with Dominant Optic Atrophy who have a specific genetic mutation.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Enrollment | 25 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | University Hospital, Angers Government |
| Locations | 1 site (Angers) |
| Trial ID | NCT06007391 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the efficacy and tolerance of nicotinamide, a form of vitamin B3, in patients with Dominant Optic Atrophy (DOA), a condition that leads to progressive vision loss. The study is based on findings that suggest a deficiency of nicotinamide in DOA patients, which may contribute to the neurodegenerative process affecting the optic nerve. Participants will be adult patients with a confirmed genetic mutation in the OPA1 gene who have not previously taken nicotinamide. The trial aims to assess the potential neuroprotective effects of nicotinamide on visual function.
Who should consider this trial
Good fit: Ideal candidates are adults diagnosed with Dominant Optic Atrophy due to a pathogenic variant in the OPA1 gene who have not previously taken nicotinamide.
Not a fit: Patients who are asymptomatic carriers of the OPA1 mutation or those with other severe ophthalmological conditions may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from Dominant Optic Atrophy.
How similar studies have performed: While this approach is based on promising preliminary findings, the specific use of nicotinamide for DOA is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Angers
- Angers University Hospital — Angers, France (Recruiting)
Study contacts
- Study coordinator: Pascal Reynier
- Email: PaReynier@chu-angers.fr
- Phone: 0241355542
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.