Tolerance and Efficacy Nicotinamide (Vitamin B3) in Dominant Optic Atrophy OPA1 (NCT06007391) | Clinical Trial Compass
Active — Not RecruitingPhase 2/3
Tolerance and Efficacy Nicotinamide (Vitamin B3) in Dominant Optic Atrophy OPA1
France25 participantsStarted 2024-01-23
Plain-language 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of patient with neurologic or ophtalmological adverse event