Nicotinamide Adverse ReactionPhase 2/3NICOPA1-TOLNCT06007391
Tolerance and Efficacy Nicotinamide (Vitamin B3) in Dominant Optic Atrophy OPA1
A Phase 2/3 study testing Nicotinamide in people with Nicotinamide Adverse Reaction, run by University Hospital, Angers at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Nicotinamide Adverse Reaction
- What is being tested?
- Nicotinamide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteFrance
- Study dates
- 23 Jan 2024to Sep 2026
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 10Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Pilot Study of Tolerance and Efficacy Nicotinamide (Vitamin B3) in Dominant Optic Atrophy OPA1
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
- Being tested
- Nicotinamide
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NicotinamideDrug
nicotinamide 3g per day
| Study group | Role | What they receive |
|---|---|---|
| nicotinamide | Experimental | Not described Receives: Drug: Nicotinamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patient with neurologic or ophtalmological adverse event
photopic negative response PhNR, optical coherence tomography
Measured: at 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2023
- Study start23 Jan 2024
- Primary completionSep 2026 (estimated)
- Study completionSep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Nicotinamide Adverse ReactionStudies of the same condition, open and under way.
- More trials run by University Hospital, AngersThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Angers
- Sponsor type
- Other government
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06007391
- Sponsor's protocol ID
- 49RC23_0195
- Acronym
- NICOPA1-TOL
- Status last verified
- Mar 2026
- First submitted
- 6 Jul 2023
- Last updated
- 1 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Tolerance and Efficacy Nicotinamide (Vitamin B3) in Dominant Optic Atrophy OPA1
- ClinicalTrials.gov ID
- NCT06007391
- Acronym / sponsor ID
- NICOPA1-TOL / 49RC23_0195
- Conditions
- Nicotinamide Adverse Reaction
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- University Hospital, AngersOther government
- Study start
- 23 Jan 2024 (actual)
- Primary completion
- Sep 2026 (estimated)
- Study completion
- Sep 2026 (estimated)
- First posted
- 23 Aug 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| nicotinamide | Experimental | Drug: Nicotinamide |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nicotinamidenicotinamide 3g per day | Drug | nicotinamide |
Primary outcome
Number of patient with neurologic or ophtalmological adverse event
Time frame: at 6 months
photopic negative response PhNR, optical coherence tomography
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Apr 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Nicotinamide in people with Nicotinamide Adverse Reaction, run by University Hospital, Angers at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 1 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of patient with neurologic or ophtalmological adverse event (time frame: at 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Apr 2026.