Intermediate Expanded Access Protocol (EAP) CNMAu8.EAP02

UCI Specialty Area: Neurology

Principal Investigator: Namita Goyal

Official Title: A Second Intermediate Expanded Access Protocol for Amyotrophic Lateral Sclerosis with CNM-Au8

A Study On:

Amyotrophic Lateral Sclerosis ALS

Brain - Neurologic/ Psychologic

Study Description

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:

A treatment period of forty-eight (48) weeks (Treatment Period 1);
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);

A3. 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.

Eligibility

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.

OR Prior participation in the HEALEY Platform ALS trial (Regimen C) Open Label Extension (NCT04414345) will be considered an automatic inclusion.

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.
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).

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University of California, Irvine - Study Information Sheet

UCI Center for Clinical Research Potential Participant Database

Lead Researcher
Maheswari Senthil, MD
UCI Center for Clinical Research

In the instance of parental permission, “You” refers to “Your child.”

Please read the information below and ask questions about anything that you do not understand. A
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You are being asked to participate in a clinical research potential participant database to identify
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time. If you decide to withdraw from this database you should notify the research team
immediately.

You are eligible to participate in this study if you may be interested in participating in a research trial at UCI Health

We would like to interview you to learn more about your eligibility for current clinical research studies at UCI. The interview will last about 5-30 minutes, depending on the extent of your medical history.

Possible risks/discomforts associated with the database are hearing about potential clinical research studies that may not be of interest to you, invasion of your privacy, or a potential breach of confidentiality.

There are no direct benefits from participation in the database. However, this database may help inform you on clinical research studies that you may be eligible for. Participation in UCI clinical research studies help us gain important data and knowledge that may lead to improved treatment options for people with similar conditions to you in the future.

There are no alternative procedures available. The only alternative is not to participate in this study.

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The research team, authorized UCI personnel, and regulatory entities, may have access to your study records to protect your safety and welfare.

While the research team will make every effort to keep your personal information confidential, it is possible that an unauthorized person might see it. We cannot guarantee total privacy.

Future Research Use
Researchers will use your information to conduct this study. Once the study is done using your information, we may share them with other researchers so they can use them for other studies in the future. We will not share your name or any other private identifiable information that would let the researchers know who you are. We will not ask you for additional permission to share this de-identified information

Data Retention - In accordance with UC Office of the President policy, information will be retained for 10 years after the end of the calendar year in which the research is completed.

If, during the course of this study, significant new information becomes available that may relate to your willingness to continue to participate, this information will be provided to you by the research team listed at the top of the form.

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