Tolerance and Efficacy Nicotinamide (Vitamin B3) in Dominant Optic Atrophy OPA1 (NICOPA1-TOL)
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| ClinicalTrials.gov Identifier: NCT06007391 |
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Recruitment Status :
Not yet recruiting
First Posted : August 23, 2023
Last Update Posted : August 30, 2023
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Sponsor:
University Hospital, Angers
Information provided by (Responsible Party):
University Hospital, Angers
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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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Nicotinamide Adverse Reaction | Drug: Nicotinamide | Phase 2 Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 25 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | Pilot Study of Tolerance and Efficacy Nicotinamide (Vitamin B3) in Dominant Optic Atrophy OPA1 |
| Estimated Study Start Date : | September 2023 |
| Estimated Primary Completion Date : | March 2025 |
| Estimated Study Completion Date : | September 2025 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Optic atrophy type 1
| Arm | Intervention/treatment |
|---|---|
| Experimental: nicotinamide |
Drug: Nicotinamide
nicotinamide 3g per day |
Primary Outcome Measures :
- Number of patient with neurologic or ophtalmological adverse event [ Time Frame: at 6 months ]photopic negative response PhNR, optical coherence tomography
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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
No Contacts or Locations Provided
| Responsible Party: | University Hospital, Angers |
| ClinicalTrials.gov Identifier: | NCT06007391 |
| Other Study ID Numbers: |
49RC23_0195 |
| First Posted: | August 23, 2023 Key Record Dates |
| Last Update Posted: | August 30, 2023 |
| Last Verified: | June 2023 |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
| Product Manufactured in and Exported from the U.S.: | No |
Additional relevant MeSH terms:
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Optic Atrophy Optic Atrophy, Autosomal Dominant Atrophy Pathological Conditions, Anatomical Optic Nerve Diseases Cranial Nerve Diseases Nervous System Diseases Eye Diseases Optic Atrophies, Hereditary Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Mitochondrial Diseases |
Metabolic Diseases Niacinamide Niacin Nicotinic Acids Vitamin B Complex Vitamins Micronutrients Physiological Effects of Drugs Hypolipidemic Agents Antimetabolites Molecular Mechanisms of Pharmacological Action Lipid Regulating Agents Vasodilator Agents |