<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/NCT03815916</url>
  </required_header>
  <id_info>
    <org_study_id>CNMAu8.202</org_study_id>
    <nct_id>NCT03815916</nct_id>
  </id_info>
  <brief_title>31P-MRS Imaging to Assess the Effects of CNM-Au8 on Impaired Neuronal Redox State in Parkinson's Disease</brief_title>
  <acronym>REPAIR-PD</acronym>
  <official_title>A Phase 2, Pilot Open Label, Sequential Group, Investigator Blinded Study of Magnetic Resonance Spectroscopy (31P-MRS) to Assess the Effects of CNM-Au8 for the Bioenergetic Improvement of Impaired Neuronal Redox State in Parkinson's Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Clene Nanomedicine</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>University of Texas Southwestern Medical Center</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Clene Nanomedicine</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      REPAIR-PD is a single-center open label pilot, sequential group, investigator and patient&#xD;
      blinded study to assess the CNS metabolic effects, safety, pharmacokinetics, and&#xD;
      pharmacodynamics of CNM-Au8 in patients who have been diagnosed with Parkinson's Disease (PD)&#xD;
      within three (3) years of Screening. The primary endpoint is the ratio of the oxidized to&#xD;
      reduced form of nicotinamide adenine dinucleotide (NAD+:NADH) measured non-invasively by&#xD;
      31phosphorous magnetic resonance spectroscopy (31P-MRS).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a single-center open label pilot, sequential group, investigator blinded study of the&#xD;
      CNS metabolic effects, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 in patients&#xD;
      who have been diagnosed with Parkinson's Disease within three years of Screening. The Sponsor&#xD;
      will select a starting treatment dose of CNM-Au8 for the initial treatment. Investigators and&#xD;
      patients will be blinded to each cohort's study dose. Upon completion of the first treatment&#xD;
      cohort, the Sponsor will select a single dose or two different doses for the subsequent&#xD;
      second cohort from a pre-specified dosing selection plan based on the evaluation of the&#xD;
      31P-Magnetic Resonance Spectroscopy (31P-MRS) changes versus baseline in the first cohort. Up&#xD;
      to a total of two treatment cohorts may be studied (n=15 patients/cohort, total n=30&#xD;
      patients). All patients will receive daily oral treatment over twelve consecutive weeks&#xD;
      during each cohort's Treatment Period.&#xD;
&#xD;
      There will be three study periods per treatment cohort:&#xD;
&#xD;
        1. A six-week screening period (Screening Period);&#xD;
&#xD;
        2. A twelve-week treatment period (Treatment Period);&#xD;
&#xD;
        3. A four-week follow-up period (End-of-Study Assessment).&#xD;
&#xD;
      The primary study outcome, CNS metabolic changes, will be assessed based upon each patient's&#xD;
      Week 12 study visit versus the pre-treatment baseline. The primary endpoint is the brain&#xD;
      metabolic effects of treatment with CNM-Au8 as assessed by an improvement of 31P-MRS&#xD;
      assessment of Brain Tissue Cellular Redox Potential defined by the measured tissue ratio of&#xD;
      NAD+:NADH concentrations following 12 weeks of once daily treatment.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">December 19, 2019</start_date>
  <completion_date type="Actual">June 7, 2021</completion_date>
  <primary_completion_date type="Actual">June 7, 2021</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Sequential Assignment</intervention_model>
    <intervention_model_description>Open Label, Investigator Blinded, Sequential Cohort (max of 2 cohorts amongst the possible 4 interventions)</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
    <masking_description>Research participants and site personnel are not masked to study drug, but will be blinded to study dose for each cohort (single-blinded).</masking_description>
  </study_design_info>
  <primary_outcome>
    <measure>Change in 31P-MRS Redox Ratio (NAD+/NADH)</measure>
    <time_frame>At 12 Weeks</time_frame>
    <description>Mean change in average NAD+/NADH measured brain Redox Ratio by treatment group</description>
  </primary_outcome>
  <other_outcome>
    <measure>Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of NAD+</measure>
    <time_frame>At 12 Week</time_frame>
    <description>Mean change in average CNS concentration of NAD+ [mmol/kg] by treatment group</description>
  </other_outcome>
  <other_outcome>
    <measure>Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of NADH</measure>
    <time_frame>At 12 Week</time_frame>
    <description>Mean change in average CNS concentration of NADH [mmol/kg] by treatment group</description>
  </other_outcome>
  <other_outcome>
    <measure>Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of Pooled NAD+/NADH</measure>
    <time_frame>At 12 Weeks</time_frame>
    <description>Mean change in average CNS concentration of pooled NAD+/NADH [mmol/kg] by treatment group</description>
  </other_outcome>
  <other_outcome>
    <measure>Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of ATP</measure>
    <time_frame>At 12 Week</time_frame>
    <description>Mean change in average CNS concentration of ATP [mmol/kg] (as internal reference) by treatment group</description>
  </other_outcome>
  <other_outcome>
    <measure>Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of Phosphocreatine (PCr)</measure>
    <time_frame>At 12 Week</time_frame>
    <description>Mean change in average CNS concentration of PCr [mmol/kg] by treatment group</description>
  </other_outcome>
  <other_outcome>
    <measure>Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of Intracellular Inorganic Phosphate (Pi(in))</measure>
    <time_frame>At 12 Week</time_frame>
    <description>Mean change in average CNS concentration of Pi(in) [mmol/kg] by treatment group</description>
  </other_outcome>
  <other_outcome>
    <measure>Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of Extracellular Inorganic Phosphate (Pi(ex))</measure>
    <time_frame>At 12 Week</time_frame>
    <description>Mean change in average CNS concentration of Pi(ex) [mmol/kg] by treatment group</description>
  </other_outcome>
  <other_outcome>
    <measure>Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of Uridine Diphosphate Glucose (UDPG)</measure>
    <time_frame>At 12 Week</time_frame>
    <description>Mean change in average CNS concentration of UDPG [mmol/kg] by treatment group</description>
  </other_outcome>
  <other_outcome>
    <measure>Mean Change in 31P-MRS Membrane Component Tissue Concentration of Phosphoethanolamine (PE)</measure>
    <time_frame>At 12 Weeks</time_frame>
    <description>Mean change in average CNS concentration of PE [mmol/kg] by treatment group</description>
  </other_outcome>
  <other_outcome>
    <measure>Mean Change in 31P-MRS Membrane Component CNS Tissue Concentration of Phosphocholine (PC)</measure>
    <time_frame>At 12 Weeks</time_frame>
    <description>Mean change in average CNS concentration of PC [mmol/kg] by treatment group</description>
  </other_outcome>
  <other_outcome>
    <measure>Mean Change in 31P-MRS Membrane Component CNS Tissue Concentration of Glycerolphophoethanolamine (GPE)</measure>
    <time_frame>At 12 Weeks</time_frame>
    <description>Mean change in average CNS concentration of GPE [mmol/kg] by treatment group</description>
  </other_outcome>
  <other_outcome>
    <measure>Mean Change in 31P-MRS Membrane Component CNS Tissue Concentration of Glycerophosphocholine (GPC)</measure>
    <time_frame>At 12 Weeks</time_frame>
    <description>Mean change in average CNS concentration of GPC [mmol/kg] by treatment group</description>
  </other_outcome>
  <other_outcome>
    <measure>Measures of Gait</measure>
    <time_frame>at 12 weeks</time_frame>
    <description>Measured by APDM instrumented Timed up and go test.</description>
  </other_outcome>
  <other_outcome>
    <measure>Measures of Balance</measure>
    <time_frame>at 12 weeks</time_frame>
    <description>Measured by APDM Instrumented Postural Sway Test</description>
  </other_outcome>
  <other_outcome>
    <measure>Measure of Mobility</measure>
    <time_frame>at 12 weeks</time_frame>
    <description>Measured by APDM instrumented Walk Test</description>
  </other_outcome>
  <other_outcome>
    <measure>Measurements of Global Impression of Disease Improvement</measure>
    <time_frame>at 12 weeks</time_frame>
    <description>Using Clinician Global Impression Scale. Scale is rated from 1-7, with 1 representing Very much improved and 7 representing very much worse.</description>
  </other_outcome>
  <other_outcome>
    <measure>Measurements of Global Impression of Disease Severity</measure>
    <time_frame>at 12 weeks</time_frame>
    <description>Using Patient Global Impression Scale. Scale is rated from 1-7, with 1 representing very mush improved, and 7 representing very much worse.</description>
  </other_outcome>
  <other_outcome>
    <measure>Measurements of disease progression</measure>
    <time_frame>at 12 weeks</time_frame>
    <description>Using Unified Parkinson's Disease Rating Scale. The scale is based off of participants symptoms, with a lower value representing a being closer to no impairments.</description>
  </other_outcome>
  <number_of_arms>4</number_of_arms>
  <enrollment type="Actual">13</enrollment>
  <condition>Parkinson's Disease</condition>
  <arm_group>
    <arm_group_label>7.5mg CNM-Au8</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>7.5mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water</description>
  </arm_group>
  <arm_group>
    <arm_group_label>15mg CNM-Au8</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>15mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water</description>
  </arm_group>
  <arm_group>
    <arm_group_label>30mg CNM-Au8</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>30mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water</description>
  </arm_group>
  <arm_group>
    <arm_group_label>60mg CNM-Au8</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>60mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Gold Nanocrystals</intervention_name>
    <description>CNM-Au8 is a dark red/purple-colored liquid formulation consisting of a stable suspension of faceted clean surfaced elemental gold nanocrystals in buffered deionized water with a concentration of up to 0.5 mg/mL of gold. The formulation is buffered by sodium bicarbonate present at a concentration of 0.546 mg/mL. There are no other excipients. The drug product is formulated to be taken orally and will be provided in single dose HDPE containers. The study doses vary by the concentration of gold nanocrystals per milliliter in a volume of 60 mL.</description>
    <arm_group_label>15mg CNM-Au8</arm_group_label>
    <arm_group_label>30mg CNM-Au8</arm_group_label>
    <arm_group_label>60mg CNM-Au8</arm_group_label>
    <arm_group_label>7.5mg CNM-Au8</arm_group_label>
    <other_name>CNM-Au8</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Able to understand and give written informed consent and follow study procedures.&#xD;
&#xD;
          2. Male or female, aged 30 - 80 years or age (inclusive) at the time of PD diagnosis.&#xD;
&#xD;
          3. PD subjects will be recruited in accordance with the MDS Clinical Diagnostic Criteria&#xD;
             for&#xD;
&#xD;
             PD:&#xD;
&#xD;
               1. Parkinsonism present (bradykinesia + either rest tremor or rigidity)&#xD;
&#xD;
               2. 2 of the following 4 supportive criteria:&#xD;
&#xD;
             i. Clear and dramatic beneficial response to dopaminergic medication&#xD;
&#xD;
             ii. Presence of levodopa-induced dyskinesias&#xD;
&#xD;
             iii. Rest tremor of a limb&#xD;
&#xD;
             iv. Olfactory loss or cardiac sympathetic denervation seen on prior MIBG SPECT&#xD;
&#xD;
          4. Duration of PD since diagnosis is &lt;/= 3 years (inclusive)&#xD;
&#xD;
          5. Modified Hoehn and Yahr stage &lt;/= 3&#xD;
&#xD;
          6. Treatment with dopaminergic therapy for at least 12-weeks and with no change in&#xD;
             current medications within the prior 6-weeks&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Atypical parkinsonism, including that due to drugs, metabolic disorders, encephalitis,&#xD;
             cerebrovascular disease, normal pressure hydrocephalus, or other neurodegenerative&#xD;
             disease.&#xD;
&#xD;
          2. The presence of any of the following:&#xD;
&#xD;
               1. Unequivocal cerebellar abnormalities&#xD;
&#xD;
               2. Downward vertical gaze limitation or slowing of downward saccades&#xD;
&#xD;
               3. Diagnosis of behavioral variant frontotemporal dementia or primary progressive&#xD;
                  aphasia&#xD;
&#xD;
               4. Parkinsonian features restricted to the lower limbs for &gt; 3 years&#xD;
&#xD;
               5. Treatment with dopamine blockers or depleters in a time course consistent with&#xD;
                  drug-induced parkinsonism&#xD;
&#xD;
               6. Absence of an observable response to high dose levodopa despite moderate disease&#xD;
                  severity&#xD;
&#xD;
               7. Expert considers a diagnosis of alternative syndrome more likely than PD&#xD;
&#xD;
               8. Rapid progression of gait impairment requiring wheelchair within 5 years of onset&#xD;
&#xD;
               9. Complete absence of progression of motor symptoms over 3 years unless due to&#xD;
                  treatment&#xD;
&#xD;
              10. Early bulbar dysfunction within the first 5 years since diagnosis&#xD;
&#xD;
              11. Inspiratory respiratory dysfunction (stridor or frequent sighs)&#xD;
&#xD;
              12. Severe autonomic failure in the first 5 years&#xD;
&#xD;
              13. Recurrent falls (&gt;1 per year) because of impaired balance in the first 3 years&#xD;
&#xD;
              14. Disproportionate dystonic anterocollis or hand contractures of hands or feet&#xD;
                  within 10 years&#xD;
&#xD;
              15. Absence of any of the common non-motor features of PD despite 5 years of disease&#xD;
&#xD;
              16. Otherwise unexplained pyramidal tract signs (weakness, hyperreflexia, or extensor&#xD;
                  toe signs)&#xD;
&#xD;
              17. Bilateral symmetric parkinsonism&#xD;
&#xD;
          3. Mini-Mental State Exammination (MMSE) score of less than 19.&#xD;
&#xD;
          4. Patient with a history of any clinically significant or unstable medical condition&#xD;
             based on the Investigator's judgment.&#xD;
&#xD;
          5. History of human immunodeficiency virus (HIV), hepatitis C (HepC) virus antibody, or&#xD;
             hepatitis B (HepB) virus antibody.&#xD;
&#xD;
          6. Based on the investigator's judgment, patients who may have difficulty complying with&#xD;
             the protocol and/or study procedures.&#xD;
&#xD;
          7. Patient with clinically significant abnormalities in hematology, blood chemistry, ECG,&#xD;
             or physical examination not resolved by the Baseline visit which according to&#xD;
             Investigator may interfere with study participation.&#xD;
&#xD;
          8. Patients with clinically significant hepatic or renal dysfunction or clinical&#xD;
             laboratory findings that would limit the interpretability of change in liver or kidney&#xD;
             function, or those with low platelet counts (&lt;150 x 109 per liter) or eosinophilia&#xD;
             (absolute eosinophil count of ≥500 eosinophils per microliter) at Screening.&#xD;
&#xD;
          9. Patient participating in any other investigational drug trial or using investigational&#xD;
             drug (within 12 weeks prior to screening and thereafter)&#xD;
&#xD;
         10. Positive screen for drugs of abuse or known history of alcohol abuse.&#xD;
&#xD;
         11. Women of child-bearing potential, or men, who are unwilling or unable to use accepted&#xD;
             methods of birth control for up to 6 months following study participation.&#xD;
&#xD;
         12. Women with a positive pregnancy test, are lactating, or are planning to become&#xD;
             pregnant during the study or within 6 months of the end of this trial.&#xD;
&#xD;
         13. Patients with implanted metal objects in their body that may be affected by an MRI&#xD;
             procedure.&#xD;
&#xD;
         14. Patients who are claustrophobic or otherwise unlikely to be able to complete the MRI&#xD;
             scanning procedures.&#xD;
&#xD;
         15. History of allergy to gold in any form.&#xD;
&#xD;
         16. Patient is considered a suicide risk in the opinion of the Investigator, has&#xD;
             previously made a suicide attempt, or is currently demonstrating active suicidal&#xD;
             ideation. Subjects with intermittent passive suicidal ideation are not necessarily&#xD;
             excluded based on the assessment of the Investigator.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>30 Years</minimum_age>
    <maximum_age>80 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Richard Dewey, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>UT Southwestern</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>UT Southwestern</name>
      <address>
        <city>Dallas</city>
        <state>Texas</state>
        <zip>75390</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>November 2021</verification_date>
  <study_first_submitted>January 18, 2019</study_first_submitted>
  <study_first_submitted_qc>January 23, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">January 24, 2019</study_first_posted>
  <last_update_submitted>November 18, 2021</last_update_submitted>
  <last_update_submitted_qc>November 18, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">November 22, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>neurodegeneration</keyword>
  <keyword>gold</keyword>
  <keyword>nanocrystal</keyword>
  <keyword>NAD+</keyword>
  <keyword>redox</keyword>
  <keyword>31P-MRS</keyword>
  <keyword>Parkinson's Disease</keyword>
  <keyword>magnetic resonance spectroscopy</keyword>
  <keyword>nanoparticle</keyword>
  <keyword>nanomedicine</keyword>
  <keyword>NADH</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Parkinson Disease</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <pending_results>
    <submitted>September 5, 2023</submitted>
  </pending_results>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


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/NCT03815916
org study id: CNMAu8.202
nct id: NCT03815916
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
REPAIR-PD is a single-center open label pilot, sequential group, investigator and patient
      blinded study to assess the CNS metabolic effects, safety, pharmacokinetics, and
      pharmacodynamics of CNM-Au8 in patients who have been diagnosed with Parkinson's Disease (PD)
      within three (3) years of Screening. The primary endpoint is the ratio of the oxidized to
      reduced form of nicotinamide adenine dinucleotide (NAD+:NADH) measured non-invasively by
      31phosphorous magnetic resonance spectroscopy (31P-MRS).
This is a single-center open label pilot, sequential group, investigator blinded study of the
      CNS metabolic effects, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 in patients
      who have been diagnosed with Parkinson's Disease within three years of Screening. The Sponsor
      will select a starting treatment dose of CNM-Au8 for the initial treatment. Investigators and
      patients will be blinded to each cohort's study dose. Upon completion of the first treatment
      cohort, the Sponsor will select a single dose or two different doses for the subsequent
      second cohort from a pre-specified dosing selection plan based on the evaluation of the
      31P-Magnetic Resonance Spectroscopy (31P-MRS) changes versus baseline in the first cohort. Up
      to a total of two treatment cohorts may be studied (n=15 patients/cohort, total n=30
      patients). All patients will receive daily oral treatment over twelve consecutive weeks
      during each cohort's Treatment Period.

      There will be three study periods per treatment cohort:

        1. A six-week screening period (Screening Period);

        2. A twelve-week treatment period (Treatment Period);

        3. A four-week follow-up period (End-of-Study Assessment).

      The primary study outcome, CNS metabolic changes, will be assessed based upon each patient's
      Week 12 study visit versus the pre-treatment baseline. The primary endpoint is the brain
      metabolic effects of treatment with CNM-Au8 as assessed by an improvement of 31P-MRS
      assessment of Brain Tissue Cellular Redox Potential defined by the measured tissue ratio of
      NAD+:NADH concentrations following 12 weeks of once daily treatment.
allocation: Non-Randomized
intervention model: Sequential Assignment
intervention model description: Open Label, Investigator Blinded, Sequential Cohort (max of 2 cohorts amongst the possible 4 interventions)
primary purpose: Treatment
masking: None (Open Label)
masking description: Research participants and site personnel are not masked to study drug, but will be blinded to study dose for each cohort (single-blinded).
measure: Change in 31P-MRS Redox Ratio (NAD+/NADH)
time frame: At 12 Weeks
description: Mean change in average NAD+/NADH measured brain Redox Ratio by treatment group
measure: Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of NAD+
time frame: At 12 Week
description: Mean change in average CNS concentration of NAD+ [mmol/kg] by treatment group
measure: Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of NADH
time frame: At 12 Week
description: Mean change in average CNS concentration of NADH [mmol/kg] by treatment group
measure: Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of Pooled NAD+/NADH
time frame: At 12 Weeks
description: Mean change in average CNS concentration of pooled NAD+/NADH [mmol/kg] by treatment group
measure: Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of ATP
time frame: At 12 Week
description: Mean change in average CNS concentration of ATP [mmol/kg] (as internal reference) by treatment group
measure: Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of Phosphocreatine (PCr)
time frame: At 12 Week
description: Mean change in average CNS concentration of PCr [mmol/kg] by treatment group
measure: Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of Intracellular Inorganic Phosphate (Pi(in))
time frame: At 12 Week
description: Mean change in average CNS concentration of Pi(in) [mmol/kg] by treatment group
measure: Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of Extracellular Inorganic Phosphate (Pi(ex))
time frame: At 12 Week
description: Mean change in average CNS concentration of Pi(ex) [mmol/kg] by treatment group
measure: Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of Uridine Diphosphate Glucose (UDPG)
time frame: At 12 Week
description: Mean change in average CNS concentration of UDPG [mmol/kg] by treatment group
measure: Mean Change in 31P-MRS Membrane Component Tissue Concentration of Phosphoethanolamine (PE)
time frame: At 12 Weeks
description: Mean change in average CNS concentration of PE [mmol/kg] by treatment group
measure: Mean Change in 31P-MRS Membrane Component CNS Tissue Concentration of Phosphocholine (PC)
time frame: At 12 Weeks
description: Mean change in average CNS concentration of PC [mmol/kg] by treatment group
measure: Mean Change in 31P-MRS Membrane Component CNS Tissue Concentration of Glycerolphophoethanolamine (GPE)
time frame: At 12 Weeks
description: Mean change in average CNS concentration of GPE [mmol/kg] by treatment group
measure: Mean Change in 31P-MRS Membrane Component CNS Tissue Concentration of Glycerophosphocholine (GPC)
time frame: At 12 Weeks
description: Mean change in average CNS concentration of GPC [mmol/kg] by treatment group
measure: Measures of Gait
time frame: at 12 weeks
description: Measured by APDM instrumented Timed up and go test.
measure: Measures of Balance
time frame: at 12 weeks
description: Measured by APDM Instrumented Postural Sway Test
measure: Measure of Mobility
time frame: at 12 weeks
description: Measured by APDM instrumented Walk Test
measure: Measurements of Global Impression of Disease Improvement
time frame: at 12 weeks
description: Using Clinician Global Impression Scale. Scale is rated from 1-7, with 1 representing Very much improved and 7 representing very much worse.
measure: Measurements of Global Impression of Disease Severity
time frame: at 12 weeks
description: Using Patient Global Impression Scale. Scale is rated from 1-7, with 1 representing very mush improved, and 7 representing very much worse.
measure: Measurements of disease progression
time frame: at 12 weeks
description: Using Unified Parkinson's Disease Rating Scale. The scale is based off of participants symptoms, with a lower value representing a being closer to no impairments.
arm group label: 7.5mg CNM-Au8
arm group type: Experimental
description: 7.5mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water
arm group label: 15mg CNM-Au8
arm group type: Experimental
description: 15mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water
arm group label: 30mg CNM-Au8
arm group type: Experimental
description: 30mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water
arm group label: 60mg CNM-Au8
arm group type: Experimental
description: 60mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water
intervention type: Drug
intervention name: Gold Nanocrystals
description: CNM-Au8 is a dark red/purple-colored liquid formulation consisting of a stable suspension of faceted clean surfaced elemental gold nanocrystals in buffered deionized water with a concentration of up to 0.5 mg/mL of gold. The formulation is buffered by sodium bicarbonate present at a concentration of 0.546 mg/mL. There are no other excipients. The drug product is formulated to be taken orally and will be provided in single dose HDPE containers. The study doses vary by the concentration of gold nanocrystals per milliliter in a volume of 60 mL.
arm group label: 15mg CNM-Au8
arm group label: 30mg CNM-Au8
arm group label: 60mg CNM-Au8
arm group label: 7.5mg CNM-Au8
other name: CNM-Au8
criteria: 
gender: All
minimum age: 30 Years
maximum age: 80 Years
healthy volunteers: No
last name: Richard Dewey, MD
role: Principal Investigator
affiliation: UT Southwestern
facility: 
country: United States
responsible party type: Sponsor
mesh term: Parkinson Disease
sharing ipd: No
submitted: September 5, 2023