Amyotrophic Lateral SclerosisPhase 2REPAIR-ALSNCT03843710
31P-MRS Imaging to Assess the Effects of CNM-Au8 on Impaired Neuronal Redox State in Amyotrophic Lateral Sclerosis (REPAIR-ALS)
A Phase 2 study testing Gold Nanocrystals in people with Amyotrophic Lateral Sclerosis, run by Clene Nanomedicine at 1 site in United States.
Trial summary
REPAIR-ALS is a single-center open label pilot, 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 Amyotrophic Lateral Sclerosis (ALS) within twelve (12) months of Screening. Read more about this study
- Who is it for?
- Ages 35 to 75 years
- Conditions the study is for
- Amyotrophic Lateral 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
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2020to Mar 2022
- 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 35 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Able to understand and give written informed consent.
- Male or female patients aged 35 years or greater (inclusive) and less than 75 years of age at the time of ALS diagnosis.
- Patients with a confirmed ALS diagnosis: "definite ALS" or "probable ALS" or "possible" diagnostic criteria per the revised El Escorial Criteria as determined by a neurologist subspecializing in ALS (e.g., the Principal Investigator by study site).
- Stable background therapy (e.g., stable dosing of riluzole within the prior 6-weeks) per Investigator discretion.
- At the time of Screening disease duration less than or equal to 24-months from symptom onset OR within 12-moths of a confirmed ALS diagnosis.
- Forced vital capacity (FVC) >/= 60% of predicted value as adjusted for gender, height, and age at the Screening Visit.
- Patients who are ambulatory (e.g., normal ambulation, early ambulation difficulties, or walks with assistance) on the ALSFRS-R scale.
Who cannot join
- At Screening patients who utilize, or in the Investigator's judgment will be imminently dependent upon during the course of this study:
- Non-invasive ventilation
- Gastrostomy (e.g., use of percutaneous endoscopic gastrostomy tube)
- Use of wheel chair
- Patient who have previously undergone tracheostomy.
- Patient with a history of significant other major medical condition based on the Investigator's judgment.
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- Based on the investigator's judgment, patients who may have difficulty complying with the protocol and/or study procedures.
- Patient with clinically significant abnormalities in hematology, blood chemistry, ECG, or physical examination not resolved by the Baseline visit which according to Investigator can interfere with study participation.
- Patient participating in any other investigational drug trial or using investigational drug (within 12 weeks prior to screening and thereafter)
- Females who are pregnant or nursing or who plan to get pregnant during the course of this clinical trial or within 6 months of the end of this trial.
- Positive screen for drugs of abuse or known alcohol abuse.
- Women of child-bearing potential, or men, who are unwilling or unable to use accepted methods of birth control during the study or for 6 months following completion of study participation.
- Women with a positive pregnancy test, are lactating, or are planning to become pregnant during the study.
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 2, Pilot 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 Amyotrophic Lateral Sclerosis
REPAIR-ALS is a single-center open label pilot, 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 Amyotrophic Lateral Sclerosis (ALS) within twelve (12) months of Screening. The primary endpoint is the ratio of the oxidized to reduced form of nicotinamide adenine dinucleotide (NAD+:NADH) measured non-invasively by 31phosphorous magnetic resonance spectroscopy (31P-MRS).
Read the detailed description
This is a single-center open label pilot, sequential group, investigator blinded study of the CNS metabolic effects, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 in patients who have been diagnosed with Amyotrophic Lateral Sclerosis within twelve months of Screening. The Sponsor will select a starting treatment dose of CNM-Au8 for the initial treatment. Investigators and patients will be blinded to each cohort's study dose. Upon completion of the first treatment cohort, the Sponsor will select a single dose or two different doses for the subsequent second cohort from a pre-specified dosing selection plan based on the evaluation of the 31P-Magnetic Resonance Spectroscopy (31P-MRS) changes versus baseline in the first cohort. Up to a total of two treatment cohorts may be studied (n=12 patients/cohort, total n=24 patients). All patients will receive daily oral treatment over twelve consecutive weeks during each cohort's Treatment Period.
There will be three study periods per treatment cohort:
A four-week screening period (Screening Period); A twelve-week treatment period (Treatment Period); A four-week follow-up period (End-of-Study Assessment).
The primary study outcome, CNS metabolic changes, will be assessed based upon each patient's Week 12 study visit versus the pre-treatment baseline. The primary endpoint is the brain metabolic effects of treatment with CNM-Au8 as assessed by an improvement of 31P-MRS assessment of Brain Tissue Cellular Redox Potential defined by the measured tissue ratio of NAD+:NADH concentrations following 12 weeks of once daily treatment.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Gold Nanocrystals
- Keywords
- Neurodegeneration, gold, nanocrystal, NAD+, NADH, Redox, Redox Ratio, ALS, Amyotrophic Lateral Sclerosis, 31P-MRS, MRS, magnetic resonance spectroscopy, nanoparticle, nanomedicine
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Nerve Degeneration
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.
Change in 31P-MRS Redox Ratio (NAD+/NADH)
Mean change in average NAD+/NADH measured brain Redox Ratio by treatment group
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 posted18 Feb 2019
- Study startMar 2020 (estimated)
- Primary completionFeb 2022 (estimated)
- Study completionMar 2022 (estimated)
Why it stopped: Study execution discontinued at this time.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral 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
- 0 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
- Jeffery Elliott, MD, Principal investigator, UT Southwestern
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03843710
- Sponsor's protocol ID
- CNMAu8.203
- Acronym
- REPAIR-ALS
- Status last verified
- Mar 2023
- First submitted
- 14 Feb 2019
- Last updated
- 3 Apr 2023
- 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 3 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Pilot 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 Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT03843710
- Acronym / sponsor ID
- REPAIR-ALS / CNMAu8.203
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- Neurodegeneration, gold, nanocrystal, NAD+, NADH, Redox, Redox Ratio, ALS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study execution discontinued at this time.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Clene NanomedicineIndustry (pharma, biotech, devices)
- Collaborators
- University of Texas Southwestern Medical Center
- Study start
- Mar 2020 (estimated)
- Primary completion
- Feb 2022 (estimated)
- Study completion
- Mar 2022 (estimated)
- First posted
- 18 Feb 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-ALS is a single-center open label pilot, 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 Amyotrophic Lateral Sclerosis (ALS) within twelve (12) months of Screening. The primary endpoint is the ratio of the oxidized to reduced form of nicotinamide adenine dinucleotide (NAD+:NADH) measured non-invasively by 31phosphorous magnetic resonance spectroscopy (31P-MRS).
Detailed description
This is a single-center open label pilot, sequential group, investigator blinded study of the CNS metabolic effects, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 in patients who have been diagnosed with Amyotrophic Lateral Sclerosis within twelve months of Screening. The Sponsor will select a starting treatment dose of CNM-Au8 for the initial treatment. Investigators and patients will be blinded to each cohort's study dose. Upon completion of the first treatment cohort, the Sponsor will select a single dose or two different doses for the subsequent second cohort from a pre-specified dosing selection plan based on the evaluation of the 31P-Magnetic Resonance Spectroscopy (31P-MRS) changes versus baseline in the first cohort. Up to a total of two treatment cohorts may be studied (n=12 patients/cohort, total n=24 patients). All patients will receive daily oral treatment over twelve consecutive weeks during each cohort's Treatment Period.
There will be three study periods per treatment cohort:
A four-week screening period (Screening Period); A twelve-week treatment period (Treatment Period); A four-week follow-up period (End-of-Study Assessment).
The primary study outcome, CNS metabolic changes, will be assessed based upon each patient's Week 12 study visit versus the pre-treatment baseline. The primary endpoint is the brain metabolic effects of treatment with CNM-Au8 as assessed by an improvement of 31P-MRS assessment of Brain Tissue Cellular Redox Potential defined by the measured tissue ratio of NAD+:NADH concentrations following 12 weeks of once daily treatment.
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
- 35 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Able to understand and give written informed consent.
- Male or female patients aged 35 years or greater (inclusive) and less than 75 years of age at the time of ALS diagnosis.
- Patients with a confirmed ALS diagnosis: "definite ALS" or "probable ALS" or "possible" diagnostic criteria per the revised El Escorial Criteria as determined by a neurologist subspecializing in ALS (e.g., the Principal Investigator by study site).
- Stable background therapy (e.g., stable dosing of riluzole within the prior 6-weeks) per Investigator discretion.
- At the time of Screening disease duration less than or equal to 24-months from symptom onset OR within 12-moths of a confirmed ALS diagnosis.
Key exclusion criteria (16 listed)
- At Screening patients who utilize, or in the Investigator's judgment will be imminently dependent upon during the course of this study:
- Patient who have previously undergone tracheostomy.
- Patient with a history of significant other major medical condition based on the Investigator's judgment.
- Based on the investigator's judgment, patients who may have difficulty complying with the protocol and/or study procedures.
- Patient with clinically significant abnormalities in hematology, blood chemistry, ECG, or physical examination not resolved by the Baseline visit which according to Investigator can interfere with study participation.
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
Change in 31P-MRS Redox Ratio (NAD+/NADH)
Time frame: At 12 Weeks
Mean change in average NAD+/NADH measured brain Redox Ratio by treatment group
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2023.
- 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.
Jeffery Elliott, MD principal investigator
UT Southwestern
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 Amyotrophic Lateral 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?
The sponsor lists 7 inclusion and 16 exclusion criteria. 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: withdrawn, as of the sponsor's last update on 3 Apr 2023. 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 was withdrawn before enrolling participants, so there are no results.
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?
Change in 31P-MRS Redox Ratio (NAD+/NADH) (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 3 Apr 2023; AzTrials last refreshed its copy on 8 Nov 2026.