31P-MRS Imaging to Assess the Effects of CNM-Au8 on Impaired Neuronal Redox State in Multiple Sclerosis.

NCT03993171 | Completed Phase 2 FDA Drug

• 1 other identifier: CNMAu8.207
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Multiple Sclerosis

Keywords

neurodegeneration; gold; nanoparticle; NAD+; redox; 31P-MRS; multiple sclerosis; magnetic resonance spectroscopy; nanocrystal; NADH; primary progressive multiple sclerosis; relapsing multiple sclerosis

Outcome Measures

Primary Outcomes (1)

• The change from baseline to week 12 in CNS metabolic changes, based on 31P-MRS Redox Ratio.

  Mean change in average NAD+/NADH measured brain Redox Ratio by treatment cohort from Baseline to Week 12.

  At 12 Weeks

Other Outcomes (29)

• Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of ATP (a, B, y) by subject per dosing group.

  At 12 Weeks

• Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of NAD+/NADH pool by subject per dosing group.

  At 12 Weeks

• Mean Change in 31P-MRS Bioenergetic Metabolite CNS Tissue Concentration of Phosphocreatine (PCr) by subject per dosing group.

  At 12 Weeks

Study Arms (4)

7.5mg CNM-Au8

EXPERIMENTAL

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

Drug: gold nanocrystals

15mg CNM-Au8

EXPERIMENTAL

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

Drug: gold nanocrystals

30mg CNM-Au8

EXPERIMENTAL

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

Drug: gold nanocrystals

60mg CNM-Au8

EXPERIMENTAL

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

Drug: gold nanocrystals

Interventions

gold nanocrystals DRUG

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.

Also known as: CNM-Au8

15mg CNM-Au8; 30mg CNM-Au8; 60mg CNM-Au8; 7.5mg CNM-Au8

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• At least 18 years of age and up to 55 years (inclusive) of age at Screening.
• Clinical diagnosis of Relapsing Multiple Sclerosis (RMS) (meeting McDonald criteria, 2017).
• Diagnosis of MS no longer than 15 years prior to Screening.
• Stable treatment with natalizumab, defined as a stable dose maintained at the standard infusion interval of 28-days (±5 days) for at least the prior six (6) months.
• Stable disease activity based on the Investigator's judgment over the prior three (3) months.
• Any hematological parameters and/or biochemical parameters that fall outside the Within Normal Limits range at Screening must be assessed as Not Clinically Significant (NCS) and deemed stable or transient in nature.
• Able to understand and give written informed consent.
• Male or female, aged 18 - 70 years or age (inclusive);
• Diagnosis of primary progressive multiple sclerosis (PPMS) or nonactive secondary progressive multiple sclerosis (SPMS), according to revised 2017 McDonald criteria at the Screening visit;
• EDSS score at the Screening visit of less than or equal to 6.5 (inclusive);
• Participants must be taking either B-cell depleting therapy (e.g., ocrelizumab, rituximab) or S1P modulator therapy (e.g., siponimod) with consistent stable dosing for at least 48 weeks prior to the Screening visit;
• Any hematological parameters and/or biochemical parameters that fall outside the Within Normal Limits range at Screening must be assessed as Not Clinically Significant (NCS) and deemed stable or transient in nature; and
• Able to understand and give written informed consent.

You may not qualify if:

• Patients with a clinical relapse requiring systemic steroid treatment within the prior three (3) months.
• Patients treated with any other MS therapy other than natalizumab; or treated with clemastine fumarate.
• Based on the Investigator's judgment, patients with a history of significant other major medical condition that may interfere with the conduct of the study or interpretation of the study results.
• Based on the Investigator's judgment, patients who may have difficulty complying with the protocol and/or study procedures.
• History of any clinically significant abnormality in hematology, blood chemistry, ECG, or physical examination not resolved by the Baseline visit which according to Investigator can interfere with study participation.
• 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.
• Patients with a prior history of, or positive serological assay for the presence of HIV infection, or laboratory evidence of active or chronic infection with hepatitis C (HCV) or hepatitis B (HBV). Note, participants who have been vaccinated for HBV and have detectable HB antibodies are not excluded unless positive for hepatitis surface antigen (HBsAg).
• Patients participating in any other investigational drug trial or using an investigational drug (within 12 weeks prior to screening and thereafter).
• Positive screen for drugs of abuse or known alcohol abuse.
• Females who are pregnant, have a positive pregnancy test, are nursing, or who plan to get pregnant during the course of this clinical trial or within 6 months of the end of this trial.
• Women of child-bearing potential, or men, who are unwilling or unable to use accepted methods of birth control during the study and for 6 months following completion of study participation.
• Patients with implanted metal objects in their body that may be affected by an MRI procedure.
• Patients who are claustrophobic or otherwise unlikely to be able to complete the MRI scanning procedures.
• Patients with a history of gold allergy.

Sponsors & Collaborators

• Clene Nanomedicine: lead
• University of Texas Southwestern Medical Center: collaborator

Study Sites (1)

University of Texas Southwestern

Dallas, Texas, 75390, United States

Location

MeSH Terms

Conditions

Multiple Sclerosis; Nerve Degeneration; Multiple Sclerosis, Chronic Progressive

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Chronic Disease; Disease Attributes

Study Officials

• Peter Sguigna, MD

  University of Texas Southwestern Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Masking Details: Research participants and site personnel are not masked to study drug, but will be blinded to study dose for each cohort (single-blinded).
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open Label, Investigator Blinded, Sequential Cohort (max of 2 cohorts amongst the possible 4 interventions)

Study Record Dates

• First Submitted: May 31, 2019
• First Posted: June 20, 2019
• Study Start: December 19, 2019
• Primary Completion: May 27, 2025
• Study Completion: July 31, 2025
• Last Updated: December 19, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)