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Quick Info
Status
Available
Phase
-
Trial Type
Expanded Access
Treatment Type
Drug Trials
Randomization
EAP
Enrollment
300
Start Date
Contact Information
Locations
United States, Arizona
Barrow Neurological Institute, Phoenix, AZ, 85013, United States
Contact: Nicole Turcotte   602-406-4775   fulton.research@dignityhealth.org
United States, California
UC Irvine, Orange, CA, 92868, United States
Contact: Kaelyn McCloud   714-456-8520   klmcclou@hs.uci.edu
Sutter Health, San Francisco, CA, 94107, United States
Contact: Bethany Parrett   415-654-1022   clinicalresearch@sutterhealth.org
United States, Connecticut
Hospital for Special Care, New Britain, CT, 06053, United States
Contact: Honora Dalamagas   860-827-1958   hdalamagas@hfsc.org
United States, Florida
Holy Cross Hospital, Davie, FL, 33314, United States
Contact: Gustavo Alameda, MD   954-414-9750   gustavo.alameda@holy-cross.com
Nova Southeastern University, Davie, FL, 33314, United States
Contact: Eduardo Locatelli, MD   954-262-6387   eduardo.locatelli@nova.edu
United States, Illinois
Northwestern, Chicago, IL, 60611, United States
Contact: Emma Schmidt   312-503-4362   emma.schmidt@northwestern.edu
United States, Kansas
University of Kansas, Fairway, KS, 66205, United States
Contact: Katie Lillig, CCRP   913-945-9932   kjennens2@Kumc.edu
United States, Michigan
Henry Ford Health Systems, Detroit, MI, 48322, United States
Contact: Beverley Duthie, RN, CRC   313-916-3359   bduthie1@hfhs.org
United States, Nebraska
University of Nebraska Medical Center, Omaha, NE, 68198, United States
Contact: Deb Heimes   402-559-4504   deb.heimes@unmc.edu
United States, North Carolina
DUKE University Medical Center, Durham, NC, 27705, United States
Contact: Michelle Ward, RN, BSN   919-613-2681   Rachel.m.ward@duke.edu
United States, Oregon
Providence Health, Portland, OR, 97225, United States
Contact: Ashley Adamo   503-962-1171   ashley.adamo@providence.org
United States, Pennsylvania
Penn State Health, Hershey, PA, 17033, United States
Contact: Wint Nandar, Ph.D.   717-531-8257   nervemuscle@pennstatehealth.psu.edu
University of Pennsylvania, Philadelphia, PA, 19104, United States
Contact: Adreeja GuhaRay, MPH   215-313-3966   adreeja.guharay@pennmedicine.upenn.edu
Jefferson Hospital, Philadelphia, PA, 19107, United States
Contact: Yesasvini Devabhaktuni   267-582-6061   yesasvini.devabhaktuni@jefferson.edu
United States, Texas
Texas Neurology, Dallas, TX, 75206, United States
Contact: Angela Coriddi   214-827-3610   acoriddi@texasneurology.com
Trial Goal
Tests to Expect
Update Notes
Sites updated
6/29/2023
Site updates
6/16/2023
Site contact update
6/5/2023
Site contact update
5/22/2023
Site contact update
5/15/2023
Site updates
5/3/2023
Sites updated
4/18/2023
Site updates
4/3/2023
EAP availible
2/16/2023
Site updates
6/15/2022
Sites removed/added
6/6/2022
Expanded access no longer available
4/22/2022
Study added
3/17/2022

Other Information

Purpose
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).
Eligibility
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 ALS diagnosis.
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 240 mL of the investigational drug suspension without substantial dysphagia, OR can intake the investigational product through a gastrostomy 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. In the judgement of the Investigator the participant's expected survival is greater than six-months.
7. 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.
OR Prior participation in the HEALEY Platform ALS trial (Regimen C) Open Label Extension (NCT04414345) will be considered an automatic inclusion.
Exclusion Criteria:
1. 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.
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.
5. Participant is currently involved in another placebo-controlled clinical trial (note:
concomitant therapy with other investigational products is permitted with certain restrictions.
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.
OR These exclusion criteria will not be applied if the participant was previously enrolled in the HEALEY Platform ALS trial (Regimen C) Open Label Extension (NCT04414345).
Details
This is a multi-center intermediate expanded access program to provide access to the investigational product, CNM-Au8, up to 300 participants diagnosed with ALS. The safety of CNM-Au8 treatment in ALS participants will be evaluated. Visits will occur at a clinic or remotely via telephone or video-visit. Visits may be conducted remotely due to COVID-19-related pandemic concerns, or if due to ALS disease progression. Participants who meet the inclusion criteria and none of the exclusionary criteria may be enrolled into the EAP. There will be three study periods: 1. A treatment period of forty-eight (48) weeks (Treatment Period 1); 2. Additional optional follow-on treatment period(s) of up to forty-eight (48) weeks duration may be added at the discretion of the Sponsor and Site Investigator (e.g., Treatment Period 2, Treatment Period 3); 3. A four (4) week safety follow-up period (End-of-Study [EOS]Assessment). All participants will receive open-label oral treatment daily up to 48 weeks during Treatment Period 1. Additional 48-week treatment periods may be approved at the discretion of the Sponsor. The EAP may be discontinued at any time at the Sponsor's discretion. At treatment discontinuation or following the end of the participant's final Treatment Period, participants will complete an end-of-study (EOS) assessment 4 weeks following discontinuation of the investigational drug product. Visit assessments may be collected remotely, via tele-visit with study site staff. Investigational product may be shipped by the site to participants who do attend in-clinic visits.
Collaborator(s)
Trial Protocol as Published on Clinicaltrials.gov
NCT05281484 (First Published: 3/7/2022)