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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called CNM-Au8, which involves gold nanocrystals, to determine its potential benefits for people with Multiple Sclerosis (MS). Researchers aim to assess how this treatment affects brain metabolism and evaluate its safety and effectiveness. The trial includes different groups that will receive various doses of the treatment. Individuals diagnosed with MS within the last 15 years and on stable medication may be suitable candidates for this study. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, providing participants an opportunity to contribute to significant advancements in MS treatment.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that CNM-Au8, a treatment using tiny gold particles, appears safe for people. In earlier studies, researchers tested CNM-Au8 on both healthy individuals and those with conditions like Multiple Sclerosis (MS) and Amyotrophic Lateral Sclerosis (ALS), focusing on treatment tolerance.

One study found that people with MS generally tolerated CNM-Au8 well, with no serious side effects directly linked to it. Another study on individuals with ALS showed that CNM-Au8 improved energy use in the body without causing major side effects.

These results suggest that CNM-Au8 is safe at the doses tested. However, as this trial remains in an early stage, researchers continue to learn about its full safety profile. Participants will be closely monitored for any side effects. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatment for MS?

Researchers are excited about CNM-Au8 for multiple sclerosis because it uses gold nanocrystals to potentially repair damaged brain cells. Unlike traditional treatments that mainly focus on managing symptoms or slowing disease progression, CNM-Au8 targets cellular energy production, aiming to improve nerve function directly. The treatment's unique formulation involves suspending the gold nanocrystals in a sodium bicarbonate solution, which could enhance their bioavailability and effectiveness. This novel approach could offer a new way to tackle multiple sclerosis, giving hope for improved outcomes for patients.

What evidence suggests that this trial's treatments could be effective for Multiple Sclerosis?

Research has shown that CNM-Au8, a treatment using tiny gold particles, may benefit people with multiple sclerosis (MS). Studies have found that it can improve nerve function in MS patients, even addressing issues that other treatments have not resolved. CNM-Au8 increases brain energy and protects nerve cells. Earlier findings suggest it can help repair damaged nerve coverings, a major issue in MS. Overall, early results are promising regarding its potential to aid those with MS. ^{1 2 4 6 7}

Who Is on the Research Team?

Peter Sguigna, MD

Principal Investigator

University of Texas Southwestern Medical Center

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03536559

Study Details | NCT03536559 | Nanocrystalline Gold to ... The objective of this trial is to assess the efficacy and safety of CNM-Au8 as a remyelinating treatment for vision-impairing MS lesions.

2. neurology.org neurology.org/doi/10.1212/WNL.0000000000203858

VISIONARY-MS Top-line Results: A Phase 2, Randomized ... These data provide evidence for improved neurological function in stable RMS patients treated with CNM-Au8 adjunctively to DMTs.

3. medcentral.com medcentral.com/neurology/ms/multiple-sclerosis-study-shows-cnm-au8-effective-in-remyelination

Multiple Sclerosis Study Shows CNM-Au8 Effective in ... Results show measurable improvements in previously fixed deficits “that we really haven't seen any therapy in MS do.” An AAN meeting highlight.

4. thelancet.com thelancet.com/journals/eclinm/article/PIIS2589-5370(23)00213-4/fulltext

Efficacy and safety of CNM-Au8 in amyotrophic lateral ... CNM-Au8 is a catalytically-active gold nanocrystal neuroprotective agent that enhances intracellular energy metabolism and reduces oxidative stress.

5. firstwordpharma.com firstwordpharma.com/story/6109118

Clene Presents New Clinical Data at ECTRIMS 2025 ... Clene Presents New Clinical Data at ECTRIMS 2025 Meeting Demonstrating CNM-Au8® Improves Brain Energy Metabolism in Multiple Sclerosis Patients.

6. medrxiv.org medrxiv.org/content/10.1101/2023.05.11.23289871v1.full

A first-in-human study in healthy subjects of the safety and ... CNM-Au8 is a suspension of catalytically-active gold nanocrystals, developed to treat neurodegenerative disease. A first-in-human single- and ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT03993171

31P-MRS Imaging to Assess the Effects of CNM-Au8 on ... CNM-Au8 is a dark red/purple-colored liquid formulation consisting of a stable suspension of faceted clean surfaced elemental gold nanocrystals in buffered ...