Multiple SclerosisPhase 2REPAIR-MSNCT03993171
31P-MRS Imaging to Assess the Effects of CNM-Au8 on Impaired Neuronal Redox State in Multiple Sclerosis.
A Phase 2 study testing gold nanocrystals in people with Multiple Sclerosis, run by Clene Nanomedicine at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Multiple Sclerosis
- What is being tested?
- gold nanocrystals
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Dec 2019to 31 Jul 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- No healthy volunteers
- Adult
Patients enrolled in Cohort 1 must meet the following Inclusion Criteria:
- 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.
Patients enrolled in Cohort 2 must meet the following Inclusion Criteria:
- 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.
Patients enrolled in Cohort 1 must meet the following Exclusion Criteria:
- 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.
- 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.
- Any active ophthalmological cause for retinal damage other than MS (e.g. cataracts, uveitis, macular degeneration, macular exudate, macular edema, glaucoma, severe astigmatism, ocular trauma, neuromyelitis optica, ischemic optic neuropathy, congenital nystagmus, retinal detachment, amblyopia, optic disk drusen).
- Severe refractive defects: refractive errors (-5 dioptres to +5 dioptres or more in either eye or axial eye length >26 mm), hypermetropia (> 5 dioptres; cylinder > 3 dioptres); or based on the Investigator's judgment any other ophthalmic diseases that might confound the study results or optical coherence tomography assessment.
- PRN use of stimulant medications including: amphetamine, dextroamphetamine, lisdexamfetamine, methylphenidate, or modafinil; however, stimulant medications, taken on a consistent daily dose for at least 12-weeks are allowed. No changes in the dose of any stimulant medications are allowed during the study.
Patients enrolled in Cohort 2 must meet the following Exclusion Criteria:
- History of MS relapses or gadolinium-enhancing lesions seen on brain MRI scans within the five (5) years prior to the Screening visit.
- History of AQP4, MOG Ab(+) status, or documented ≥ 3 contiguous segment lesion in the spinal cord.
- Any diagnosis other than PPMS or SPMS that could explain a participant's signs and symptoms.
- Participants taking any MS disease-modifying therapy other than Bcell depleting therapy (e.g., ocrelizumab, rituximab) or S1P modulator therapy (e.g., siponimod), or not taking disease modifying therapy.
- 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.
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas Southwestern
Dallas, Texas, 75390
About this study
Official title: A Phase 2, 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 Multiple Sclerosis.
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.
Read the detailed description
This is a single-center open-label, sequential group, investigator blinded study of the CNS metabolic effects, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 in patients who have been diagnosed with relapsing multiple sclerosis (RMS) within fifteen (15) years of Screening. Patients will be screened over up to a 6-week period.Patients who meet inclusion criteria and none of the exclusion criteria may be enrolled into the clinical study. The initial cohort of patients (Cohort 1) will begin treatment at a dose of 15mg or 30mg CNM-Au8. Upon completion of the first Treatment Period, dose(s) will be selected for the subsequent cohort (Cohort 2), based on the results of the 31P-MRS change versus baseline from the first cohort. A total of two treatment cohorts may be studied. Investigators and patients will remain blinded to study dose until the study database is formally locked. All patients will receive daily oral treatment over twelve (12) consecutive weeks during each cohort's Treatment Period.
There will be three study periods per treatment cohort:
- A (6) six week screening period (Screening Period);
- A (12) twelve-week treatment period (Treatment Period);
- A (6) six-week follow-up period (End-of-Study Assessment).
Patients will be contacted by phone to assess safety and tolerability at Week 2. At Weeks 4, 8, and 12 patients will return to the clinic to complete PK, PD, visual acuity testing, and safety assessments. At the Baseline and Week 12 visits patients will complete 31P-MRS, MRI, and OCT imaging assessments, the 9-Hole Peg Test, Timed 25-Foot Walk Test, Visual Acuity (high and low contrast letter/visual acuity) and the Symbol Digit Modalities Test (SDMT). Following treatment discontinuation at Week 12, patients will complete an end-of-study (EOS) visit at Week 18. All patients who are prematurely discontinued from treatment will complete the end-of-study visit 4-weeks after discontinuation.
- Conditions
- Multiple Sclerosis
- Being tested
- gold nanocrystals
- Keywords
- neurodegeneration, gold, nanoparticle, NAD+, redox, 31P-MRS, multiple sclerosis, magnetic resonance spectroscopy, nanocrystal, NADH, primary progressive multiple sclerosis, relapsing multiple sclerosis
- Medical subject headings
- Multiple Sclerosis, Nerve Degeneration, Multiple Sclerosis, Chronic Progressive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
gold nanocrystalsDrug
Also called 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.
| Study group | Role | What they receive |
|---|---|---|
| 7.5mg CNM-Au8 | Experimental | 7.5mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water Receives: Drug: gold nanocrystals |
| 15mg CNM-Au8 | Experimental | 15mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water Receives: Drug: gold nanocrystals |
| 30mg CNM-Au8 | Experimental | 30mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water Receives: Drug: gold nanocrystals |
| 60mg CNM-Au8 | Experimental | 60mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water Receives: Drug: gold nanocrystals |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: At 12 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jun 2019
- Study start19 Dec 2019
- Primary completion27 May 2025
- Study completion31 Jul 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Open Label, Investigator Blinded, Sequential Cohort (max of 2 cohorts amongst the possible 4 interventions)
- Masking
- None (open label)
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- Clene Nanomedicine
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- University of Texas Southwestern Medical Center
- Responsible party
- Sponsor
- Investigators
- Peter Sguigna, MD, Principal investigator, University of Texas Southwestern Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03993171
- Sponsor's protocol ID
- CNMAu8.207
- Acronym
- REPAIR-MS
- Status last verified
- Dec 2025
- First submitted
- 31 May 2019
- Last updated
- 19 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, 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 Multiple Sclerosis.
- ClinicalTrials.gov ID
- NCT03993171
- Acronym / sponsor ID
- REPAIR-MS / CNMAu8.207
- Conditions
- Multiple Sclerosis
- Keywords
- neurodegeneration, gold, nanoparticle, NAD+, redox, 31P-MRS, multiple sclerosis, magnetic resonance spectroscopy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- Clene NanomedicineIndustry (pharma, biotech, devices)
- Collaborators
- University of Texas Southwestern Medical Center
- Study start
- 19 Dec 2019 (actual)
- Primary completion
- 27 May 2025 (actual)
- Study completion
- 31 Jul 2025 (actual)
- First posted
- 20 Jun 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a single-center open-label, sequential group, investigator blinded study of the CNS metabolic effects, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 in patients who have been diagnosed with relapsing multiple sclerosis (RMS) within fifteen (15) years of Screening. Patients will be screened over up to a 6-week period.Patients who meet inclusion criteria and none of the exclusion criteria may be enrolled into the clinical study. The initial cohort of patients (Cohort 1) will begin treatment at a dose of 15mg or 30mg CNM-Au8. Upon completion of the first Treatment Period, dose(s) will be selected for the subsequent cohort (Cohort 2), based on the results of the 31P-MRS change versus baseline from the first cohort. A total of two treatment cohorts may be studied. Investigators and patients will remain blinded to study dose until the study database is formally locked. All patients will receive daily oral treatment over twelve (12) consecutive weeks during each cohort's Treatment Period.
There will be three study periods per treatment cohort:
- A (6) six week screening period (Screening Period);
- A (12) twelve-week treatment period (Treatment Period);
- A (6) six-week follow-up period (End-of-Study Assessment).
Patients will be contacted by phone to assess safety and tolerability at Week 2. At Weeks 4, 8, and 12 patients will return to the clinic to complete PK, PD, visual acuity testing, and safety assessments. At the Baseline and Week 12 visits patients will complete 31P-MRS, MRI, and OCT imaging assessments, the 9-Hole Peg Test, Timed 25-Foot Walk Test, Visual Acuity (high and low contrast letter/visual acuity) and the Symbol Digit Modalities Test (SDMT). Following treatment discontinuation at Week 12, patients will complete an end-of-study (EOS) visit at Week 18. All patients who are prematurely discontinued from treatment will complete the end-of-study visit 4-weeks after discontinuation.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- Open Label, Investigator Blinded, Sequential Cohort (max of 2 cohorts amongst the possible 4 interventions)
| Arm | Type | Receives |
|---|---|---|
| 7.5mg CNM-Au8 | Experimental | Drug: gold nanocrystals7.5mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water |
| 15mg CNM-Au8 | Experimental | Drug: gold nanocrystals15mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water |
| 30mg CNM-Au8 | Experimental | Drug: gold nanocrystals30mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water |
| 60mg CNM-Au8 | Experimental | Drug: gold nanocrystals60mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| gold nanocrystalsAlso: CNM-Au8CNM-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. | Drug | 15mg CNM-Au8, 30mg CNM-Au8, 60mg CNM-Au8, 7.5mg CNM-Au8 |
Primary outcome
The change from baseline to week 12 in CNS metabolic changes, based on 31P-MRS Redox Ratio.
Time frame: At 12 Weeks
Mean change in average NAD+/NADH measured brain Redox Ratio by treatment cohort from Baseline to Week 12.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Dec 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Peter Sguigna, MD principal investigator
University of Texas Southwestern Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing gold nanocrystals in people with Multiple Sclerosis, run by Clene Nanomedicine at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The change from baseline to week 12 in CNS metabolic changes, based on 31P-MRS Redox Ratio. (time frame: At 12 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Dec 2025; AzTrials last refreshed its copy on 8 Nov 2026.