An Intermediate Expanded Access Protocol (EAP) with CNM-Au8 for Amyotrophic LateralSclerosis for NIH Grant RFA-NS-23-012
An Intermediate Expanded Access Protocol (EAP) with CNM-Au8 for Amyotrophic Lateral
Sclerosis for NIH Grant RFA-NS-23-012. The primary objective of this intermediate EAP is
to provide access to the investigational product, CNM-Au8 30mg, for up to 180 people
living with ALS.
Drug: CNM-Au8
CNM-Au8 (cellular energetic nanocatalyst). Faceted clean-surfaced catalytically-active Au
nanocrystals.
Inclusion Criteria:
1. Able to understand and give written informed consent.
2. Male or female participants aged 18 years or greater (inclusive) at the time of
informed consent completion.
3. 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).
4. Participant is able to daily consume up to 60 mL of the investigational drug
suspension without substantial dysphagia, OR can intake the investigational product
through a feeding tube.
5. 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).
6. Participant has a baseline score by the TRICALS risk calculator that is less than -2
(i.e., participant is not at increased risk of early death;
https://tricals.shinyapps.io/risk-profile/).
7. Participant meets the following criteria:
1. Baseline Vital Capacity >15% predicted,
2. Baseline ALSFRS-R Score >8, and,
3. Baseline BMI >17.5 kg/m2
8. Participants have established care with a neurologist at the specialized ALS center
or remotely enrolling site involved in the study and will maintain this clinical
care throughout the duration of the EAP within the United States.
Exclusion Criteria:
1. Participant is eligible for participation in any double-blind placebo-controlled
study the treatment of ALS at the same research site.
2. 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.
3. Based on the investigator's judgment, participants who may have difficulty complying
with the protocol and/or any study procedures.
4. Within the prior 90-days the participant has had clinically significant findings on
standard hepatic, hematologic, or renal safety assays, including but not limited to:
(i) ALT or AST ≥ 3 times upper limits of normal, (ii) direct (conjugated) with
bilirubin ≥2 times upper limits of normal, (iii) low platelet counts (< 150 x 109
per liter) or eosinophilia (absolute eosinophil count of ≥ 500 eosinophils per
microliter), (iv) serum creatinine >1.2 mg/dL, or (v) eGFR < 45 ml/min per 1.73 m2.
5. Participant is currently involved in another placebo-controlled clinical trial
(note: concomitant therapy with other investigational products is permitted with
certain restrictions-see concomitant medications below).
6. 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.
7. Females of child-bearing potential, or men, who are unwilling or unable to use
accepted methods of birth control.
8. History of gold allergy.
Nova Southeastern University
Fort Lauderdale, Florida, United States
Northwestern University
Chicago, Illinois, United States
Synapticure
Chicago, Illinois, United States
University of Iowa
Iowa City, Iowa, United States
Columbia Unniversity
New York, New York, United States
Duke University
Durham, North Carolina, United States
Pennsylvania State University
Hershey, Pennsylvania, United States
Texas Neurology
Dallas, Texas, United States
Austin Rynders, BS
(801)676-9695
eap@clene.com
Jeremy Evan, PA-C
(801)676-9695
eap@clene.com
Not Provided