31P-MRS Imaging to Assess the Effects of CNM-Au8 on Impaired Neuronal Redox State in Parkinson's Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 29 November 2021
• Main ID: NCT03815916
• Date of registration: 18/01/2019
• Prospective Registration: Yes
• Primary sponsor: Clene Nanomedicine
• Public title: 31P-MRS Imaging to Assess the Effects of CNM-Au8 on Impaired Neuronal Redox State in Parkinson's Disease REPAIR-PD
• Scientific 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
• Date of first enrolment: December 19, 2019
• Target sample size: 13
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03815916
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Richard Dewey, MD
• Affiliation: UT Southwestern

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Able to understand and give written informed consent and follow study procedures.

2. Male or female, aged 30 - 80 years or age (inclusive) at the time of PD diagnosis.

3. PD subjects will be recruited in accordance with the MDS Clinical Diagnostic Criteria
for

PD:

1. Parkinsonism present (bradykinesia + either rest tremor or rigidity)

2. 2 of the following 4 supportive criteria:

i. Clear and dramatic beneficial response to dopaminergic medication

ii. Presence of levodopa-induced dyskinesias

iii. Rest tremor of a limb

iv. Olfactory loss or cardiac sympathetic denervation seen on prior MIBG SPECT

4. Duration of PD since diagnosis is
5. Modified Hoehn and Yahr stage
6. Treatment with dopaminergic therapy for at least 12-weeks and with no change in
current medications within the prior 6-weeks

Exclusion Criteria:

1. Atypical parkinsonism, including that due to drugs, metabolic disorders, encephalitis,
cerebrovascular disease, normal pressure hydrocephalus, or other neurodegenerative
disease.

2. The presence of any of the following:

1. Unequivocal cerebellar abnormalities

2. Downward vertical gaze limitation or slowing of downward saccades

3. Diagnosis of behavioral variant frontotemporal dementia or primary progressive
aphasia

4. Parkinsonian features restricted to the lower limbs for > 3 years

5. Treatment with dopamine blockers or depleters in a time course consistent with
drug-induced parkinsonism

6. Absence of an observable response to high dose levodopa despite moderate disease
severity

7. Expert considers a diagnosis of alternative syndrome more likely than PD

8. Rapid progression of gait impairment requiring wheelchair within 5 years of onset

9. Complete absence of progression of motor symptoms over 3 years unless due to
treatment

10. Early bulbar dysfunction within the first 5 years since diagnosis

11. Inspiratory respiratory dysfunction (stridor or frequent sighs)

12. Severe autonomic failure in the first 5 years

13. Recurrent falls (>1 per year) because of impaired balance in the first 3 years

14. Disproportionate dystonic anterocollis or hand contractures of hands or feet
within 10 years

15. Absence of any of the common non-motor features of PD despite 5 years of disease

16. Otherwise unexplained pyramidal tract signs (weakness, hyperreflexia, or extensor
toe signs)

17. Bilateral symmetric parkinsonism

3. Mini-Mental State Exammination (MMSE) score of less than 19.

4. Patient with a history of any clinically significant or unstable medical condition
based on the Investigator's judgment.

5. History of human immunodeficiency virus (HIV), hepatitis C (HepC) virus antibody, or
hepatitis B (HepB) virus antibody.

6. Based on the investigator's judgment, patients who may have difficulty complying with
the protocol and/or study procedures.

7. Patient with clinically significant abnormalities in hematology, blood chemistry, ECG,
or physical examination not resolved by the Baseline visit which according to
Investigator may interfere with study participation.

8. Patients with clinically significant hepatic or renal dysfunction or clinical
laboratory findings that would limit the interpretability of change in liver or kidney
function, or those with low platelet counts (<150 x 109 per liter) or eosinophilia
(absolute eosinophil count of =500 eosinophils per microliter) at Screening.

9. Patient participating in any other investigational drug trial or using investigational
drug (within 12 weeks prior to screening and thereafter)

10. Positive screen for drugs of abuse or known history of alcohol abuse.

11. Women of child-bearing potential, or men, who are unwilling or unable to use accepted
methods of birth control for up to 6 months following study participation.

12. Women with a positive pregnancy test, are lactating, or are planning to become
pregnant during the study or within 6 months of the end of this trial.

13. Patients with implanted metal objects in their body that may be affected by an MRI
procedure.

14. Patients who are claustrophobic or otherwise unlikely to be able to complete the MRI
scanning procedures.

15. History of allergy to gold in any form.

16. Patient is considered a suicide risk in the opinion of the Investigator, has
previously made a suicide attempt, or is currently demonstrating active suicidal
ideation. Subjects with intermittent passive suicidal ideation are not necessarily
excluded based on the assessment of the Investigator.



Age minimum: 30 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Parkinson's Disease

Intervention(s)

Drug: Gold Nanocrystals

Primary Outcome(s)

Change in 31P-MRS Redox Ratio (NAD+/NADH) [Time Frame: At 12 Weeks]

Secondary ID(s)

CNMAu8.202

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

University of Texas Southwestern Medical Center