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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03536559




Registration number
NCT03536559
Ethics application status
Date submitted
2/05/2018
Date registered
24/05/2018
Date last updated
18/01/2019

Titles & IDs
Public title
Nanocrystalline Gold to Treat Remyelination Failure in Chronic Optic Neuropathy In Multiple Sclerosis
Scientific title
A Phase 2, Randomized, DB-PC, Parallel Group Study for the Treatment of Visual Pathway Deficits In Chronic Optic Neuropathy to Assess the Efficacy, Safety, Tolerability and Pharmacokinetics of CNM-Au8 For Remyelination In Multiple Sclerosis
Secondary ID [1] 0 0
CNMAu8.201
Universal Trial Number (UTN)
Trial acronym
VISIONARY-MS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Relapsing Remitting Multiple Sclerosis 0 0
Optic Neuropathy 0 0
Optic; Neuritis, With Demyelination 0 0
Condition category
Condition code
Eye 0 0 0 0
Diseases / disorders of the eye
Neurological 0 0 0 0
Other neurological disorders
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - CNM-Au8
Treatment: Drugs - Placebo

Experimental: 15mg CNM-Au8 - 15mg suspension of clean-surfaced, faceted, gold nanocrystals in 60ml of sodium bicarbonate buffered water

Experimental: 30mg CNM-Au8 - 30mg suspension of clean-surfaced, faceted, gold nanocrystals in 60ml of sodium bicarbonate buffered water

Placebo Comparator: Placebo - The matched placebo to be used in this study will consist of water, sodium bicarbonate, and food coloring to match volume and color of the experimental treatments.


Treatment: Drugs: CNM-Au8
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.

Treatment: Drugs: Placebo
Placebo is liquid with identical color and taste

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Multifocal Visual Evoked Potential (mfVEP) Latency - mfVEP latency is an electrophysiologic measure of remyelination that assesses the speed of conduction of electrical signals in neurons of the visual system.
Timepoint [1] 0 0
At six month (24-weeks)
Secondary outcome [1] 0 0
Low Contrast Letter Acuity (LCLA) - LCLA is a functional measure of contrast vision
Timepoint [1] 0 0
At six months and then every 12 weeks thereafter, up to 48-weeks

Eligibility
Key inclusion criteria
1. At least 18 years of age and up to 50 years of age (inclusive).

2. Clinical diagnosis of MS (meeting McDonald criteria, 2010) who have had RRMS no longer
than 10 years from diagnosis.

3. Presence of chronic optic neuropathy defined by clinical and/or multifocal visual
evoked potential (mf-VEP) criteria.

4. Retinal Nerve Fiber Layer (RNFL) thickness > 70 µm.

5. Stable disease activity based on the Investigator's judgment over the prior 12 months.

6. Able to understand and give written informed consent.
Minimum age
18 Years
Maximum age
50 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. An acute optic neuritis episode or systemic steroid treatment within the prior 6
months.

2. MS-related clinical relapse requiring systemic steroid treatment within the prior 12
months.

3. Unstable treatment with a disease modifying therapy (DMT) defined as a treatment
change within prior 3-months unless due to intolerability.

4. Any immunosuppression therapy other than those approved for the treatment of MS.

5. Any drug known or suspected of producing retinal or optic nerve toxicity including
hydroxychloroquine, chloroquine, clofazimine, vigabatrin, or ethambutol.

6. Any active ophthalmological causes for retinal damage other than MS.

7. Diabetes with retinopathy or a previous diagnosis of Diabetes Mellitus or history of
prior impaired fasting glucose =126 mg/dL (or = 200 mg/dL after oral glucose tolerance
test).

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

9. History of gold allergy.

10. Patients taking stimulant medications (including: amphetamine, dextroamphetamine,
lisdexamfetamine, methylphenidate, or modafinil).

11. Patients taking clemastine fumarate or 4-aminopyridine (fampridine).

12. Females who have a positive pregnancy test result, or who are pregnant, breastfeeding,
or planning to conceive during the study.

13. History or evidence of substance abuse or alcohol abuse within 5 years prior to
Screening, including alcoholism; or severe tobacco use (>1 pack/day).

14. Clinical history of toxic neuropathy (e.g., secondary to treatment with ethambutol,
isoniazid, linezolid, gentamycin, chloramphenicol, vincristine, or penicillamine).

15. Current enrollment in any other drug or device treatment study within 3 months prior
to Baseline. Participation in an observational non-interventional study (i.e., no drug
or device therapy) is not an exclusion criterion.

16. Inability to undergo any planned study procedures such as VEP or MRI, including
reduced renal clearance (screening: GFR < 45 mL/min), history of severe
hypersensitivity to gadolinium-DTPA, claustrophobia; or inability to comply with study
requirements based on Investigator judgment.

17. Based on the investigator's judgment, concurrent chronic or acute illness or unstable
medical condition that may deteriorate that could confound the results of safety
assessments.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Sydney Brain Mind Centre - Sydney
Recruitment postcode(s) [1] 0 0
- Sydney

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Clene Nanomedicine
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Clene Australia Pty Ltd
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
The objective of this trial is to assess the efficacy and safety of CNM-Au8 as a
remyelinating treatment for vision-impairing MS lesions in participants who have chronic
vision impairment as a result of Relapsing Remitting Multiple Sclerosis. The primary endpoint
is the improvement in remyelination as measured by visual evoked potentials after 24 weeks of
treatment. The secondary endpoint is the improvement in low contrast vision as measured by
low contrast letter acuity after 24 weeks of treatment.
Trial website
https://clinicaltrials.gov/show/NCT03536559
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Heidi Beadnall, MD
Address 0 0
University of Sydney
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Glen Frick, MD, PhD
Address 0 0
Country 0 0
Phone 0 0
801-676-9695
Fax 0 0
Email 0 0
info@clene.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03536559