31P-MRS Imaging to Assess the Effects of CNM-Au8 on Impaired Neuronal Redox State in Amyotrophic … (NCT03843710) | Clinical Trial Compass
WithdrawnPhase 2
31P-MRS Imaging to Assess the Effects of CNM-Au8 on Impaired Neuronal Redox State in Amyotrophic Lateral Sclerosis (REPAIR-ALS)
Stopped: Study execution discontinued at this time.
United States0Started 2020-03
Plain-language 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. 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).
Who can participate
Age range
35 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial is listed as 'withdrawn' — can you help me understand what that means for my situation, and whether the research into CNM-Au8 for ALS is continuing in any other active studies I might be eligible for?
2Since this was a Phase 2 trial, what does it tell us about how much was actually known about CNM-Au8's safety and effectiveness in ALS patients at the time it was withdrawn?
3The trial was measuring something called the NAD+/NADH redox ratio using a brain imaging technique called 31P-MRS — can you explain what that means in plain terms, and why targeting neuronal redox state might matter for ALS treatment?
4Given that this trial was withdrawn before completing, are there other approaches — including standard care or other clinical trials — that are currently addressing the same underlying biological pathway in ALS?
5Is there anything about my specific ALS diagnosis, stage, or overall health that would make me a good or poor candidate if a similar trial to this one were to open in the future?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. Patients who are ambulatory (e.g., normal ambulation, early ambulation difficulties, or walks with assistance) on the ALSFRS-R scale.
Exclusion criteria
. 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.
. 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.