Two Intermediate Expanded Access Protocols (EAP) CNMAu8.EAP01 and CNMAu8.EAP02 for ALS
1 other identifier
expanded_access
N/A
1 country
16
Brief Summary
The primary objective of the intermediate expanded access protocol is to provide access to the investigational product, CNM-Au8, to up to 300 people living with ALS (pALS). No formal clinical hypotheses are being evaluated with concurrent controls. Secondary objectives include assessment of the safety of CNM-Au8 treatment in pALS. Safety will be assessed through the frequency of serious adverse events (SAEs), treatment-emergent adverse events (TEAEs) assessed as 'severe', discontinuations due to TEAEs, and laboratory abnormalities assessed as clinically significant during routine clinical monitoring (as applicable).
Trial Health
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16 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 7, 2022
CompletedFirst Posted
Study publicly available on registry
March 16, 2022
CompletedMarch 27, 2026
March 1, 2026
March 7, 2022
March 24, 2026
Conditions
Keywords
Interventions
CNM-Au8 is an aqueous suspension of clean surfaced faceted nanocrystals consisting of gold atoms self-organized into crystals of various geometrical shapes (hexagonal bi-pyramid, pentagonal bipyramid, tetrahedron, decahedron, planar spheroids). Highly pure elemental Au nanocrystals are suspended in USP purified deionized water buffered with 0.546 mg/mL (6.5 mM) sodium bicarbonate (NaHCO3) nominally concentrated to up to 0.5 mg/mL (500 ppm).
Eligibility Criteria
You may qualify if:
- Able to understand and give written informed consent.
- Male or female participants aged 18 years or greater (inclusive) at the time of ALS diagnosis.
- Participants whose conditions are defined as "definite ALS" or "probable ALS" or "possible ALS" diagnostic criteria by the revised El Escorial Criteria as determined by a neurologist specializing in ALS (e.g., the Principal Investigator or sub-investigator at the site).
- Participant is able to daily consume 60 mL of the investigational drug suspension without substantial dysphagia, OR can intake the drug through a gastrostomy tube.
- In the judgement of the Investigator, the participant's expected survival is greater than six-months
- Participants who have established care with a neurologist at the specialized ALS center involved in the study and will maintain this clinical care throughout the duration of the EAP.
- Participant must have completed standard clinical safety labs within the prior 90 days from the Baseline visit including a chemistry panel (e.g., CMP) and a hematology panel (e.g., CBC).
- Participants will be excluded from the expanded access protocol if they meet any of the following criteria:
- Participant is eligible for a clinical study with CNM-Au8 for the treatment of ALS.
- Participant with a history of any clinically significant or unstable medical condition based on the Investigator's judgment that may interfere with assessment of the study objectives.
- Based on the investigator's judgment, participants who may have difficulty complying with the protocol and/or any study procedures.
- Within the prior 90 days the participant has had clinically significant abnormalities in hematology, blood chemistry, ECG, or physical examination not resolved by the Baseline visit which according to Investigator can interfere with EAP participation.
- Participant is currently involved in another placebo-controlled clinical trial (note: concomitant therapy with other investigational products is permitted with certain restrictions.
- Females who are pregnant or nursing or who plan to get pregnant during the course of this EAP or within 6 months of the end of the EAP.
- Females of child-bearing potential, or men, who are unwilling or unable to use accepted methods of birth control.
You may not qualify if:
- For Protocol CNMAu8.EAP02
- Able to understand and give written informed consent.
- Male or female participants aged 18 years or greater (inclusive) at the time of ALS diagnosis.
- Participants with a confirmed diagnosis of ALS per Gold Coast criteria as determined by a neurologist specializing in ALS (e.g., the site principal investigator or sub-investigator for this study).
- Participant is able to daily consume up to 240 mL of the investigational drug suspension without substantial dysphagia, OR can intake the investigational product through a gastrostomy tube.
- Participant must have completed standard clinical safety labs within the prior 90 days from the Baseline visit including a chemistry panel (e.g., CMP) and a hematology panel (e.g., CBC).
- In the judgement of the Investigator the participant's expected survival is greater than six-months.
- Participants who have established care with a neurologist at the specialized ALS center involved in the study and will maintain this clinical care throughout the duration of the EAP within the United States.
- Participant is eligible for participation in: (i) the HEALEY ALS Platform trial (NCT04297683), or (ii) any active study investigating CNM-Au8 for the treatment of ALS.
- Participant has a history of any clinically significant or unstable medical condition (other than ALS) that may interfere with assessment of safety or compromise the study objectives.
- Based on the investigator's judgment, participants who may have difficulty complying with the protocol and/or any study procedures.
- Within the prior 90-days the participant has had clinically significant findings on standard hepatic, hematologic, or renal safety assays.
- Participant is currently involved in another placebo-controlled clinical trial (note: concomitant therapy with other investigational products is permitted with certain restrictions.
- Females who are pregnant or nursing or who plan to get pregnant during the EAP or within 6 months of the end of this trial.
- Females of child-bearing potential, or men, who are unwilling or unable to use accepted methods of birth control.
- +1 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (16)
Barrow Neurological Institute
Phoenix, Arizona, 85013, United States
UC Irvine
Orange, California, 92868, United States
Sutter Health (Enrollment is full, not recruiting)
San Francisco, California, 94107, United States
Hospital for Special Care (Enrollment is full, not recruiting)
New Britain, Connecticut, 06053, United States
Nova Southeastern University
Davie, Florida, 33314, United States
Northwestern (Enrollment is full, not recruiting)
Chicago, Illinois, 60611, United States
University of Kansas (Enrollment is full, not recruiting)
Fairway, Kansas, 66205, United States
Massachusetts General Hospital (Enrollment is full, not recruiting)
Boston, Massachusetts, 02114, United States
Henry Ford Health Systems (Enrollment is full, not recruiting)
Detroit, Michigan, 48322, United States
University of Nebraska Medical Center (Enrollment is full, not recruiting)
Omaha, Nebraska, 68198, United States
DUKE University Medical Center (Enrollment is full, not recruiting)
Durham, North Carolina, 27705, United States
Providence Health (Enrollment is full, not recruiting)
Portland, Oregon, 97225, United States
Penn State Health (Enrollment is full, not recruiting)
Hershey, Pennsylvania, 17033, United States
Jefferson Hospital (Enrollment is full, not recruiting)
Philadelphia, Pennsylvania, 19107, United States
Texas Neurology
Dallas, Texas, 75206, United States
University of Washington
Seattle, Washington, 98195, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 7, 2022
First Posted
March 16, 2022
Last Updated
March 27, 2026
Record last verified: 2026-03