Intermediate Expanded Access Protocol (EAP) CNMAu8.EAP02

• ClinicalTrials.gov Identifier: NCT05281484
• Expanded Access Status: Available
• First Posted: March 16, 2022
• Last Update Posted: May 3, 2024
• Sponsor: Clene Nanomedicine
• Information provided by (Responsible Party): Clene Nanomedicine

Study Description

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).

Condition or disease	Intervention/treatment
Amyotrophic Lateral Sclerosis	Drug: CNM-Au8

Detailed 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:

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.

Study Design

• Study Type: Expanded Access
• Expanded Access Type: Individual Patients
• Official Title: A Second Intermediate Expanded Access Protocol for Amyotrophic Lateral Sclerosis With CNM-Au8

Interventions

Intervention Details:

• Drug: CNM-Au8

  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

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All

Criteria

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).

Contacts and Locations

Contacts

Contact: Austin Rynders, BS; (801)676-9695; info@clene.com

Contact: Jeremy Evan, PA-C; (801)676-9695; info@clene.com

Locations

United States, Arizona

Barrow Neurological Institute; Available

Phoenix, Arizona, United States, 85013

Contact: Nicole Turcotte 602-406-4775 fulton.research@dignityhealth.org

Principal Investigator: Shafeeq Ladha, MD

United States, California

UC Irvine; Available

Orange, California, United States, 92868

Contact: Kaelyn McCloud 714-456-8520 klmcclou@hs.uci.edu

Principal Investigator: Namita Goyal, MD

Sutter Health; Available

San Francisco, California, United States, 94107

Contact: Bethany Parrett 415-654-1022 clinicalresearch@sutterhealth.org

Principal Investigator: Jonathan Katz, MD

United States, Connecticut

Hospital for Special Care; Available

New Britain, Connecticut, United States, 06053

Contact: Honora Dalamagas 860-827-1958 hdalamagas@hfsc.org

Principal Investigator: Charles Whitaker, MD

United States, Florida

Nova Southeastern University; Available

Davie, Florida, United States, 33314

Contact: Eduardo Locatelli, MD 954-262-6387 eduardo.locatelli@nova.edu

United States, Illinois

Northwestern; Available

Chicago, Illinois, United States, 60611

Contact: Emma Schmidt 312-503-4362 emma.schmidt@northwestern.edu

Principal Investigator: Senda Ajroud-Driss, MD

United States, Kansas

University of Kansas; Available

Fairway, Kansas, United States, 66205

Contact: Katie Lillig, CCRP 913-945-9932 kjennens2@Kumc.edu

Principal Investigator: Omar Jawdat, MD

United States, Michigan

Henry Ford Health Systems; Available

Detroit, Michigan, United States, 48322

Contact: Beverley Duthie, RN, CRC 313-916-3359 bduthie1@hfhs.org

Principal Investigator: Ximena Arcila-Londono, MD

United States, Nebraska

University of Nebraska Medical Center; Available

Omaha, Nebraska, United States, 68198

Contact: Deb Heimes 402-559-4504 deb.heimes@unmc.edu

Principal Investigator: Joseph Fernandez, MD

United States, North Carolina

DUKE University Medical Center; Available

Durham, North Carolina, United States, 27705

Contact: Michelle Ward, RN, BSN alsresearch@duke.edu

Contact: Hailey Zampa, CRC alsresearch@duke.edu

Principal Investigator: Richard Bedlack, MD

United States, Oregon

Providence Health; Available

Portland, Oregon, United States, 97225

Contact: Ashley Adamo 503-962-1171 ashley.adamo@providence.org

Principal Investigator: Nicholas Olney, MD

United States, Pennsylvania

Penn State Health; Available

Hershey, Pennsylvania, United States, 17033

Contact: Wint Nandar, Ph.D. 717-531-8257 nervemuscle@pennstatehealth.psu.edu

Principal Investigator: Zachary Simmons, MD

University of Pennsylvania; Available

Philadelphia, Pennsylvania, United States, 19104

Contact: Adreeja GuhaRay, MPH 215-313-3966 adreeja.guharay@pennmedicine.upenn.edu

Principal Investigator: Lauren Elman, MD

Jefferson Hospital; Available

Philadelphia, Pennsylvania, United States, 19107

Contact: Amir Moghadam 267-582-6061 Amir.Moghadamahmadi@jefferson.edu

Principal Investigator: Hristelina Ilieva, MD

United States, Texas

Texas Neurology; Available

Dallas, Texas, United States, 75206

Contact: Angela Coriddi 214-827-3610 acoriddi@texasneurology.com

Principal Investigator: Daragh Heitzman, MD

United States, Washington

University of Washington; Available

Seattle, Washington, United States, 98195

Contact: Kinsey Chapman kinseyc@uw.edu

Principal Investigator: Michael Weiss, MD

Sponsors and Collaborators

Clene Nanomedicine

More Information

• Responsible Party: Clene Nanomedicine
• ClinicalTrials.gov Identifier: NCT05281484
• Other Study ID Numbers: CNMAu8.EAP02
• First Posted: March 16, 2022
• Last Update Posted: May 3, 2024
• Last Verified: May 2024

Keywords provided by Clene Nanomedicine:

Expanded access; ALS; Amyotrophic Lateral Sclerosis; CNM Au8; Au8

Additional relevant MeSH terms:

Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis; Pathologic Processes; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Spinal Cord Diseases; Central Nervous System Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases