xml: <clinical_study>
    <!-- This xml conforms to an XML Schema at:
      https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
    <required_header>
      <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
      <link_text>Link to the current ClinicalTrials.gov record.</link_text>
      <url>https://clinicaltrials.gov/ct2/show/NCT06007391</url>
    </required_header>
    <id_info>
      <org_study_id>49RC23_0195</org_study_id>
      <nct_id>NCT06007391</nct_id>
    </id_info>
    <brief_title>Tolerance and Efficacy Nicotinamide (Vitamin B3) in Dominant Optic Atrophy OPA1</brief_title>
    <acronym>NICOPA1-TOL</acronym>
    <official_title>Pilot Study of Tolerance and Efficacy Nicotinamide (Vitamin B3) in Dominant Optic Atrophy OPA1</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>University Hospital, Angers</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>University Hospital, Angers</source>
    <oversight_info>
      <is_fda_regulated_drug>No</is_fda_regulated_drug>
      <is_fda_regulated_device>No</is_fda_regulated_device>
      <is_us_export>No</is_us_export>
    </oversight_info>
    <brief_summary>
      <textblock>
        Dominant Optic Atrophy (hereafter known as DOA) is a neurodegenerative pathology of the optic&#xD;
        nerve inducing progressive loss of central visual field and visual acuity. There is currently&#xD;
        no proven treatment for this disease. The metabolomics work of Pascal Reynier's team revealed&#xD;
        a specific metabolomic signature of DOA in the plasma of patients. This metabolomic signature&#xD;
        revealed a relative deficiency in nicotinamide compared to a control population, a vitamin&#xD;
        compound (vitamin B3) known to be neuroprotective for the optic nerve and mitochondria. Note&#xD;
        that the investigator have also identified this nicotinamide deficiency in primary open-angle&#xD;
        glaucoma and Leber's hereditary optic neuropathy, the other most common cause of hereditary&#xD;
        optic neuropathy, these three optic nerve conditions sharing a common pathophysiological&#xD;
        mechanism of mitochondrial deficit. In addition, an American team demonstrated the high&#xD;
        neuroprotective power on the optic nerve of nicotinamide in a mouse model of glaucoma. These&#xD;
        arguments converge towards the potential therapeutic interest of this vitamin in degenerative&#xD;
        pathologies of the optic nerve. This is encouraged by the fact that two randomized clinical&#xD;
        trials have confirmed a benefit of nicotinamide in glaucoma. The objective of this pilot&#xD;
        study is to test the tolerance and efficacy of nicotinamide in DOA and DOA+ patients.&#xD;
      </textblock>
    </brief_summary>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">September 2023</start_date>
    <completion_date type="Anticipated">September 2025</completion_date>
    <primary_completion_date type="Anticipated">March 2025</primary_completion_date>
    <phase>Phase 2/Phase 3</phase>
    <study_type>Interventional</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <allocation>N/A</allocation>
      <intervention_model>Single Group Assignment</intervention_model>
      <primary_purpose>Treatment</primary_purpose>
      <masking>None (Open Label)</masking>
    </study_design_info>
    <primary_outcome>
      <measure>Number of patient with neurologic or ophtalmological adverse event</measure>
      <time_frame>at 6 months</time_frame>
      <description>photopic negative response PhNR, optical coherence tomography</description>
    </primary_outcome>
    <number_of_arms>1</number_of_arms>
    <enrollment type="Anticipated">25</enrollment>
    <condition>Nicotinamide Adverse Reaction</condition>
    <arm_group>
      <arm_group_label>nicotinamide</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
    </arm_group>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Nicotinamide</intervention_name>
      <description>nicotinamide 3g per day</description>
      <arm_group_label>nicotinamide</arm_group_label>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  Adult patients&#xD;
  &#xD;
            -  Patients with DOA or DOA+ due to a heterozygous pathogenic variant in the OPA1 gene&#xD;
  &#xD;
            -  Naïve patients (&gt; 3 months) in terms of taking nicotinamide&#xD;
  &#xD;
            -  Patients able to take oral medication and comply with specific study procedures&#xD;
  &#xD;
            -  Patients affiliated or beneficiaries of a social security scheme&#xD;
  &#xD;
            -  Signature of voluntary, free and informed consent to participate in the study&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Asymptomatic patients (= healthy carriers of an OPA1 mutation but not having developed&#xD;
               optic neuropathy)&#xD;
  &#xD;
            -  Patients with another associated severe ophthalmological pathology (advanced glaucoma,&#xD;
               retinal pathology, etc.)&#xD;
  &#xD;
            -  Patients treated with Idebenone&#xD;
  &#xD;
            -  Patients with a level of transaminase(s) (ASAT and/or ALAT) twice higher than the high&#xD;
               normal value.&#xD;
  &#xD;
            -  Pregnant, breastfeeding or parturient women&#xD;
  &#xD;
            -  Patients with a contraindication to nicotinamide&#xD;
  &#xD;
            -  Persons deprived of liberty by administrative or judicial decision&#xD;
  &#xD;
            -  Patients subject to a legal protection measure&#xD;
  &#xD;
            -  Persons undergoing psychiatric treatment under duress&#xD;
  &#xD;
            -  Persons unable to express their consent&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>18 Years</minimum_age>
      <maximum_age>N/A</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <verification_date>June 2023</verification_date>
    <study_first_submitted>July 6, 2023</study_first_submitted>
    <study_first_submitted_qc>August 17, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 23, 2023</study_first_posted>
    <last_update_submitted>August 29, 2023</last_update_submitted>
    <last_update_submitted_qc>August 29, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 30, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Optic Atrophy</mesh_term>
      <mesh_term>Optic Atrophy, Autosomal Dominant</mesh_term>
      <mesh_term>Atrophy</mesh_term>
    </condition_browse>
    <intervention_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Niacinamide</mesh_term>
      <mesh_term>Niacin</mesh_term>
      <mesh_term>Nicotinic Acids</mesh_term>
    </intervention_browse>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: download date: ClinicalTrials.gov processed this data on September 20, 2023
  link text: Link to the current ClinicalTrials.gov record.
  url: https://clinicaltrials.gov/ct2/show/NCT06007391
  org study id: 49RC23_0195
  nct id: NCT06007391
  lead sponsor: 
  is fda regulated drug: No
  is fda regulated device: No
  is us export: No
  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.
  allocation: N/A
  intervention model: Single Group Assignment
  primary purpose: Treatment
  masking: None (Open Label)
  measure: Number of patient with neurologic or ophtalmological adverse event
  time frame: at 6 months
  description: photopic negative response PhNR, optical coherence tomography
  arm group label: nicotinamide
  arm group type: Experimental
  intervention type: Drug
  intervention name: Nicotinamide
  description: nicotinamide 3g per day
  arm group label: nicotinamide
  criteria: 
  gender: All
  minimum age: 18 Years
  maximum age: N/A
  healthy volunteers: No
  responsible party type: Sponsor
  mesh term: Optic Atrophy
  mesh term: Optic Atrophy, Autosomal Dominant
  mesh term: Atrophy
  mesh term: Niacinamide
  mesh term: Niacin
  mesh term: Nicotinic Acids