Gold Nanocrystals for Multiple Sclerosis

(REPAIR-MS Trial)

Overseen By Jeremy Evan, PA-C

Age: 18 - 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Clene Nanomedicine

Must be taking: Natalizumab, B-cell depleting, S1P modulator

Must not be taking: Clemastine fumarate, other MS therapies

Disqualifiers: HIV, Hepatitis B/C, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 5

Duration: 12 weeks

30 Participants Needed

Trial Summary

What is the purpose of this trial?

REPAIR-MS is a single-center open label, 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 Multiple Sclerosis (MS) within fifteen (15) years of Screening. The primary endpoint for this study changes from baseline to week 12 in CNS metabolic changes, based on 31P-MRSimaging.

Will I have to stop taking my current medications?

Participants in Cohort 1 must continue their stable treatment with natalizumab, and those in Cohort 2 must continue their B-cell depleting therapy or S1P modulator therapy. The trial does not specify stopping other medications, but participants cannot be on other MS therapies or investigational drugs.

What data supports the effectiveness of the treatment CNM-Au8 for multiple sclerosis?

Research shows that gold nanocrystals can help repair damaged nerve coverings in animal models of multiple sclerosis, improving motor functions and promoting nerve cell growth. Additionally, gold nanoparticles have been found to reduce inflammation and slow disease progression in similar conditions. ^{1 2 3 4 5}

Is CNM-Au8 safe for humans?

CNM-Au8, a gold nanocrystal treatment, has been tested in clinical trials for conditions like multiple sclerosis and amyotrophic lateral sclerosis. These studies, including the RESCUE-ALS trial, have evaluated its safety, and it has been generally well-tolerated in humans. ^{2 3 4 6 7}

How is the drug CNM-Au8 different from other treatments for multiple sclerosis?

CNM-Au8 is unique because it uses gold nanocrystals to improve energy metabolism in brain cells, which helps protect nerves and repair damaged myelin (the protective covering of nerve fibers). Unlike other treatments, it is taken orally and works by enhancing the brain's energy processes, potentially offering a novel approach to treating multiple sclerosis. ^{1 2 5 6 7}

Research Team

Peter Sguigna, MD

Principal Investigator

University of Texas Southwestern Medical Center

Eligibility Criteria

Adults aged 18-70 with Multiple Sclerosis (MS) diagnosed within the last 15 years. Participants must have stable MS or be on specific therapies for at least 48 weeks. They should not have had recent relapses, metal implants affected by MRI, significant medical conditions, drug abuse history, pregnancy plans during and post-trial without contraception use, or a gold allergy.

Inclusion Criteria

I have been on a stable dose of natalizumab every 28 days for at least 6 months.

My blood and biochemical tests are stable, even if some results are outside the normal range.

I have been diagnosed with Relapsing Multiple Sclerosis.

Exclusion Criteria

I don't have unresolved health issues that could affect my study participation.

I have no unresolved health issues that could affect my study participation.

Cohort 1: Based on the Investigator's judgment, patients who may have difficulty complying with the protocol and/or study procedures

Timeline

Screening

Participants are screened for eligibility to participate in the trial

6 weeks

Treatment

Participants receive daily oral treatment with CNM-Au8 for CNS metabolic effects, safety, pharmacokinetics, and pharmacodynamics assessment

12 weeks

4 visits (in-person) at Weeks 4, 8, and 12; phone contact at Week 2

Follow-up

Participants are monitored for safety and effectiveness after treatment discontinuation

6 weeks

1 visit (in-person) at Week 18

Treatment Details

Interventions

• CNM-Au8

Trial Overview The REPAIR-MS study is testing CNM-Au8 gold nanocrystals to see if they can improve brain metabolism in MS patients. This involves taking the treatment and having special brain scans called '31P-MRS imaging' over a period of 12 weeks to measure any changes.

Participant Groups

Group I: 7.5mg CNM-Au8 Experimental Treatment 1 Intervention

7.5mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water

Group II: 60mg CNM-Au8 Experimental Treatment 1 Intervention

60mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water

Group III: 30mg CNM-Au8 Experimental Treatment 1 Intervention

30mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water

Group IV: 15mg CNM-Au8 Experimental Treatment 1 Intervention

15mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water

Who Is Running the Clinical Trial?

Clene Nanomedicine

Lead Sponsor

Trials

13

Recruited

800+

University of Texas Southwestern Medical Center

Collaborator

Trials

1,102

Recruited

1,077,000+

References

1. United States pubmed.ncbi.nlm.nih.gov

Clinical translation of gold nanoparticles. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Nanocatalytic activity of clean-surfaced, faceted nanocrystalline gold enhances remyelination in animal models of multiple sclerosis. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

Neuroinflammatory Regulation of Gold Nanoparticles Conjugated to Ethylene Dicysteine Diethyl Ester in Experimental Autoimmune Encephalomyelitis. [2021]

4. Switzerland pubmed.ncbi.nlm.nih.gov

Gold Nanoparticles: Multifaceted Roles in the Management of Autoimmune Disorders. [2022]

5. Germany pubmed.ncbi.nlm.nih.gov

Metallic gold slows disease progression, reduces cell death and induces astrogliosis while simultaneously increasing stem cell responses in an EAE rat model of multiple sclerosis. [2021]

6. England pubmed.ncbi.nlm.nih.gov

Evidence of brain target engagement in Parkinson's disease and multiple sclerosis by the investigational nanomedicine, CNM-Au8, in the REPAIR phase 2 clinical trials. [2023]

7. England pubmed.ncbi.nlm.nih.gov

Efficacy and safety of CNM-Au8 in amyotrophic lateral sclerosis (RESCUE-ALS study): a phase 2, randomised, double-blind, placebo-controlled trial and open label extension. [2023]