Clene’s Expanded Access Program for People Living with Amyotrophic Lateral Sclerosis

About Clene’s ACT-EAP

Welcome to Clene Nanomedicine’s ALS Expanded Access Program (EAP) website. Clene’s ACT-EAP is a compassionate use program developed to provide people with amyotrophic lateral sclerosis (ALS) access to CNM-Au8®. This ACT-EAP is a collaborative effort between Clene, Columbia University, and Synapticure, and is supported by funding that was made available by the ACT for ALS. ALS is a devastating neurological disorder without a cure. The investigational drug CNM-Au8 is currently undergoing clinical trials to evaluate CNM-Au8’s efficacy and safety in treating ALS. CNM-Au8 has not yet been approved or cleared by the FDA to be a safe and effective treatment for ALS.

Objectives

Patient Access to Investigational Drug:

We recognize the urgency to treat ALS. Our primary objective is to provide an opportunity for people living with ALS (pALS), who are otherwise not eligible for participation in a clinical trial, to try investigational treatment CNM-Au8. Through our ACT-EAP, eligible participants can gain access to CNM-Au8 while traditional clinical trials are conducted to evaluate CNM-Au8 for its potential efficacy in ALS.

Safety Assessment:

Participants will be closely monitored to gather data on CNM-Au8's safety and tolerability.

Efficacy Assessment:

Participants will be monitored for healthspan and lifespan while taking CNM-Au8. Data on the possible effects of taking CNM-Au8 to slow disease worsening and increase lifespan, including the effects of CNM-Au8 on blood-based biomarkers will be collected.

What to Expect in this ACT-EAP

Our ACT-EAP aims to strike a balance between the rigors of traditional expanded access program participation and the convenience of remote engagement. Participating in our ACT-EAP with CNM-Au8 is a significant decision. Below is information on what participants can expect throughout their involvement in this EAP. Participation in this ACT-EAP is voluntary.

ACT-EAP with CNM-Au8 Design:
There are two ways to participate in our ACT-EAP with CNM-Au8:

  • Through a participating local research institution or
  • Through Synapticure’s virtual clinical site.

While Synapticure will conduct all visits virtually, visits at local research institutions may be both in-person and virtual. Virtual visits will require a stable internet connection.

Each participant will be in the EAP for up to 144 weeks plus a 4-week safety follow-up for a total of 148 weeks.
Participating through a Local Research Institution:
Study
Entry
START
Eligibility Assessments
Week
0
1st Dose of CNM-Au8
Week
12
Week
24
Week
36
Telehealth Visits (Optional)
Week
48
Week
60
Telehealth Visits (Optional)
Week
72
Week
84
Telehealth Visits (Optional)
Week
96
Week
108
Telehealth Visits (Optional)
Week
120
Week
132
Telehealth Visits (Optional)
Week
144
End of
Study
+4 Week Safety Follow-up
Participants will begin visits in-person at a local research institution. Participants will have the option to attend visits virtually starting at week 36 and every 24 weeks thereafter.
Participating through Synapticure:
Study
Entry
START
Eligibility Assessments
Week
0
1st Dose of CNM-Au8
Week
12
Week
24
Week
36
Week
48
Week
60
Week
72
Telehealth Visits
Week
84
Week
96
Week
108
Week
120
Week
132
Week
144
End of
Study
+4 Week Safety Follow-up
Synapticure participants will attend all visits virtually. Virtual participation will require the participant to allow vendors into their houses to perform assessments. Continuous support and resources will be available to aid in data collection and reporting for the entire duration of the EAP.

For the ACT-EAP with CNM-Au8, Clene has partnered with Synapticure, a specialty telehealth clinical care provider specializing in neurodegenerative diseases. Interested people living with ALS who do not reside near one of the participating research clinics, or those located in more rural areas, may enroll through Synapticure to participate in this ACT-EAP completely virtually, including receiving CNM-Au8 directly to your home.

Taking an already FDA approved medication or another investigational therapy before the start of this ACT-EAP, may not prevent your participation in this EAP. Once the EAP has started, participants are asked to abstain from starting new treatments for ALS.

What will Clene’s ACT-EAP measure to evaluate the safety and efficacy of CNM-Au8?

Because this ACT-EAP supports the collection of real-world evidence while taking CNM-Au8, participants in the ACT-EAP will be contributing to our understanding of the potential effects of CNM-Au8 on the course of the disease. We are deeply grateful to all those living with ALS who wish to contribute to science in this manner.

Data collection starts at week 0 and continues every 12 weeks, both during telehealth and in-person visits.

The following data will be analyzed and reported as part of this study:

Safety and Tolerability

Long-term Survival Benefits

Clinical Function

The Revised Amyotrophic Lateral Sclerosis Rating Scale (ALSFRS-R) will be used to assess and monitor key measures of physical function, such as: speech, swallowing, walking, and respiratory function.

Changes in Clinical Care (need for tracheostomy, ventilatory support, or feeding tube)

Changes in Fatigue

Breathing: Forced Vital Capacity (FVC)*

Blood Biomarkers*

Proteins and other biological substances (metabolites) can be measured from a small sample of blood. Such biomarkers may provide another view on the possible effectiveness of CNM-Au8.

Quality of Life Measures*

*Collected at weeks 0, 12 and 24 and then collected every 24 weeks thereafter

Eligibility Criteria:

Our ACT-EAP has specific eligibility criteria to ensure the program enrolls the intended patient population. The following are select criteria for the participation in our ACT-EAP. For a full list of eligibility criteria for Clene’s ACT-EAP, please visit www.clinicaltrials.gov/NCT06408727.

Select criteria include:

  • Age of at least 18 years old or older
  • Diagnosis of ALS (based on Gold Coast criteria by a physician who is experienced in management of ALS)
  • Ability to consume 60mL of liquid by mouth or feeding tube
  • Not eligible for any current ongoing ALS clinical trial at the research institution of enrollment
A description of Clene’s ACT-EAP is available at www.clinicaltrials.gov/NCT06408727 and includes up to date listings of participating research institutions. This entry on www.clinicaltrials.gov will be updated as more research institutions are activated. Check back periodically for updated information.

Locations:

This ACT-EAP with CNM-Au8 is only available at U.S. research institutions and through Synapticure’s virtual clinical site. Locations will be updated as more research institutions are activated. Check back periodically for updated information.

EAP Locations:

Duke University
Durham, North Carolina
Northwestern University
Chicago, Illinois
NOVA University
Ft. Lauderdale, Florida
Texas Neurology
Dallas, Texas
Penn State University
Hershey, Pennsylvania
University of Iowa
Iowa City, Iowa

Columbia University

Duke University

Northwestern University

NOVA University

Texas Neurology

Penn State University

University of Iowa

Synapticure Tele-neurology Clinic

A full description of Clene’s ACT-EAP with CNM-Au8 is available at www.clinicaltrials.gov/NCT06408727.

What is CNM-Au8:

CNM-Au8 is a suspension of very tiny gold particles that is being studied as a treatment for ALS. This investigational medication has been shown to be effective in human cell and animal models of ALS, and preliminary findings from clinical studies in pALS have demonstrated evidence of a survival benefit and delayed clinical worsening. CNM-Au8 is thought to improve mitochondrial function, reduce oxidative stress, and lower levels of misfolded proteins in neurodegenerative diseases like ALS. There have been over 600 patient years of CNM-Au8 exposure without identified safety signals.

CNM-Au8 has been shown to be safe and well-tolerated in clinical trials. However, CNM-Au8 has not yet been approved by the U.S. Food and Drug Administration (FDA) for the treatment of ALS. This means that CNM-Au8 can only be used in investigational studies and EAPs like this one. You may learn more about CNM-Au8 on our webpage at www.clene.com/about-cnm-au8.

Join Us in the Fight Against ALS

By participating in our ALS ACT-EAP, pALS, caregivers, and healthcare providers become integral partners in advancing our understanding of potential treatments for ALS. Together, we strive to make a meaningful impact on the lives of those affected by this challenging disease.

Frequently Asked Questions

EAP stands for Expanded Access Program, also known as “Compassionate Use” program. An EAP is an FDA-regulated pathway that allows people with a serious and life-threatening disease to access an investigational drug that is not yet approved by the U.S. Food and Drug Administration (FDA).

People living with ALS who are interested in participating in Clene’s ACT-EAP with CNM-Au8 should discuss the option with their healthcare provider. If you are seen at one of the participating research institutions, further information about this ACT-EAP may be able to be provided by the research team.

Alternatively, people living with ALS who are seen at a research institution that is not participating in Clene’s ACT-EAP with CNM-Au8 may reach out directly, or have their healthcare provider contact, the lead investigator of the nearest participating research institution. If you do not live near a participating research institution, you may contact Synapticure through their website at www.Synapticure.com or call to book a consultation at 708-630-1534.

Participating research institutions can be found at www.clinicaltrials.gov/NCT06408727.

In general, participants in one EAP may not typically enroll and participate in another EAP at the same time. This is mainly due to safety concerns when using multiple investigational drug options at the same time. It is possible, however, to withdraw from one EAP and then enroll in a different EAP. Each program will have different requirements on how long to wait before entering another EAP. It is best to discuss this option with your healthcare provider and with the research institution of the EAP of interest.

If you meet the outlined Eligibility Criteria above and would like to be evaluated for participation in the ACT-EAP with CNM-Au8, contact the lead investigator of the research institution nearest you listed on the Clene ACT-EAP entry at www.clinicaltrials.gov/NCT06408727. If you do not live near a participating research institution, you may contact Synapticure through their website at www.Synapticure.com or call to book a consultation at 708-630-1534.

EAPs provide access to investigational therapies to people living with a serious and immediately life-threatening disease, such as ALS, who do not meet criteria for clinical trial enrollment. In an EAP, there is no placebo arm; all participants get access to the investigational drug. In a clinical trial, there is usually a placebo arm in which some portion of enrolled participants are given a matched placebo instead of investigational drug.
The National Institute of Health (NIH) has granted funds to Columbia, Clene, and Synapticure to administer and conduct this ACT-EAP at no cost to study participants. Please note your health insurer may be billed for, among other things, routine items and services that you would have received even if you did not take part in this program. You will be responsible for payment of any deductibles and co-payments required by your insurer for routine care or other billable expenses. If you have any questions about costs to you that may result from taking part in this program, please speak with the site doctors and site staff.
Like any medical intervention, there are potential risks and benefits associated with experimental treatments. Patients and their healthcare providers should thoroughly discuss potential risks and benefits before deciding to participate.
This ACT-EAP with CNM-Au8 is subject to FDA regulatory oversight to ensure patient safety and proper program conductance.
Eligibility for clinical trials may be influenced by participation in an EAP, and vice versa. It’s crucial for patients and their healthcare providers to carefully consider the implications of EAP participation on future trial eligibility and discuss these aspects with the relevant research teams. Once a participant has started Clene’s ACT-EAP with CNM-Au8, the participant may not be allowed to start a new treatment for ALS for the duration in this ACT-EAP.
Your decision to participate in this program is voluntary. You may choose to not participate or you may withdraw from the program for any reason without penalty or loss of benefits to which you are otherwise entitled and without any effect on your future medical care. If you decide to withdraw, please notify a member of the site staff before you withdraw. If you do withdraw your consent during the treatment plan, the site doctor and relevant site staff will follow withdrawal procedures.
Geographical, operational, and financial constraints were incorporated into the decision-making process regarding a research institution’s participation in this ACT-EAP with CNM-Au8. The design of the ACT-EAP aimed to maximize total number of participants while providing access to clinical sites at representative geographic locations throughout the US (and including Synapticure for virtual access) while minimizing operational costs for each site. If you are interested in participating and do not live near a research institution, and you are not eligible for any clinical trials within reasonable distance, you may wish to determine if you are eligible to participate via Synapticure.
This ACT-EAP is a partnership between Synapticure, Columbia University, and Clene Nanomedicine. One aspect that makes this ACT-EAP unique is Synapticure’s participation. Synapticure is a tele-neurology clinic that will allow individuals from remote areas of the US to participate via fully virtual visits. Participants who enroll through Synapticure will participate by having blood draws and the collection of other clinical data occurring from their home. This four-year ACT-EAP with CNM-Au8 will therefore differ from other EAPs in terms of the investigational drug being studied, the frequency of clinical visits, and the overall design of the program.

If you’re not located near a participating research institution for our ACT-EAP, there may still be an option for you to participate. Our aim with this ACT-EAP with CNM-Au8 is to improve and broaden access to potential treatments for people living with ALS regardless of geographical location. We understand that access to ALS clinical centers of excellence can be challenging for some participants.

To accommodate individuals who aren’t close to ALS clinics, Synapticure tele-neurology consultations or remote monitoring may aid to facilitate participation in Clene’s ACT-EAP. Through these means, you can still receive necessary assessments and follow-up care from healthcare professionals involved in the program, ensuring that you can access treatment and support regardless of your location.

For more information on how to participate from a remote location or to inquire about alternative arrangements, please contact Synapticure through their website at www.Synapticure.com or call to book a consultation at 708-630-1534. Synapticure will initiate the process and contact the appropriate care team coordinators on your behalf to facilitate your participation in this ACT-EAP with CNM-Au8.

Clene has been committed to supporting the ALS community. One of the ways we have demonstrated this commitment has been to offer three independent Expanded Access Programs (EAPs) in ALS. These programs allow participants with ALS, who do not qualify for clinical trials, access to Clene’s investigational drug, CNM-Au8. To learn more about how CNM-Au8 works, please click here.

The first EAP with Massachusetts General Hospital (MGH), started in 2019, is run in collaboration with Dr. Merit Cudkowicz MD, MSc of Harvard University, to grant people with ALS access to Clene’s investigational drug CNM-Au8. This EAP is enrolling at the discretion of the investigators at MGH.

The second EAP with MGH and 16 centers across the United States, started in 2023, is ongoing. This EAP was initiated to allow people with ALS who were participants in the Healey Platform Trial continued access to CNM-Au8 once the long- term extension of the regimen ended.

The ACT-EAP has not yet started but is slated to begin in spring of 2024. This EAP is a collaborative effort among Clene, Columbia University, and Synapticure. This ACT-EAP is largely supported with funds from the NIH as a result of the ACT-EAP for ALS and sponsored by Clene.

CNM-Au8® is a federally registered trademark of Clene Nanomedicine, Inc.
IMPORTANT NOTICE

CNM-Au8 is an investigational drug and is not currently approved by the U.S. Food and Drug Administration (FDA). CNM-Au8 is undergoing evaluation and testing as part of clinical trials. Its safety and efficacy have not yet been confirmed by regulatory authorities.

Our commitment to providing access to this investigational drug is rooted in the understanding that some people living with ALS may have exhausted conventional treatment options. However, it’s crucial to recognize that participation in the EAP involves the use of an investigational drug that has not received official approval from the FDA.

We believe in transparency and informed decision-making. We encourage all participants in the ACT-EAP to consult with their healthcare providers to thoroughly understand the potential risks and benefits associated with taking an investigational drug. As CNM-Au8 progresses along the drug development pipeline, we will keep our community informed about any changes in its approval status.

Please be aware that participation in the ACT-EAP is voluntary, and individuals considering this option should carefully weigh the potential risks and benefits in consultation with their healthcare professionals. Clene will continue to prioritize safety, ethics, and the pursuit of effective ALS treatments.

Clene Nanomedicine, Inc. is a wholly owned subsidiary of Clene Inc. © 2024 Clene Nanomedicine, Inc. - All rights reserved.
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