Intermediate Expanded Access Protocol (EAP) CNMAu8.EAP02

October 25, 2023 updated by: Clene Nanomedicine

A Second Intermediate Expanded Access Protocol for Amyotrophic Lateral Sclerosis With CNM-Au8

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).

Study Overview

Status

Available

Conditions

Intervention / Treatment

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 Type

Expanded Access

Expanded Access Type

  • Individual Patients

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Jeremy Evan, PA-C
  • Phone Number: (801)676-9695
  • Email: info@clene.com

Study Contact Backup

  • Name: Austin Rynders, BS
  • Phone Number: (801)676-9695
  • Email: info@clene.com

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85013
        • Available
        • Barrow Neurological Institute
        • Contact:
        • Principal Investigator:
          • Shafeeq Ladha, MD
    • California
      • Orange, California, United States, 92868
        • Available
        • UC Irvine
        • Contact:
        • Principal Investigator:
          • Namita Goyal, MD
      • San Francisco, California, United States, 94107
    • Connecticut
      • New Britain, Connecticut, United States, 06053
        • Available
        • Hospital for Special Care
        • Contact:
        • Principal Investigator:
          • Charles Whitaker, MD
    • Florida
      • Davie, Florida, United States, 33314
      • Davie, Florida, United States, 33314
        • Available
        • Nova Southeastern University
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Available
        • Northwestern
        • Contact:
        • Principal Investigator:
          • Senda Ajroud-Driss, MD
    • Kansas
      • Fairway, Kansas, United States, 66205
        • Available
        • University of Kansas
        • Principal Investigator:
          • Omar Jawdat, MD
        • Contact:
    • Michigan
      • Detroit, Michigan, United States, 48322
        • Available
        • Henry Ford Health Systems
        • Contact:
        • Principal Investigator:
          • Ximena Arcila-Londono, MD
    • Nebraska
      • Omaha, Nebraska, United States, 68198
        • Available
        • University of Nebraska Medical Center
        • Contact:
        • Principal Investigator:
          • Joseph Fernandez, MD
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Available
        • Duke University Medical Center
        • Contact:
        • Contact:
        • Principal Investigator:
          • Richard Bedlack, MD
    • Oregon
      • Portland, Oregon, United States, 97225
        • Available
        • Providence Health
        • Contact:
        • Principal Investigator:
          • Nicholas Olney, MD
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
      • Philadelphia, Pennsylvania, United States, 19104
      • Philadelphia, Pennsylvania, United States, 19107
    • Texas
      • Dallas, Texas, United States, 75206
        • Available
        • Texas Neurology
        • Contact:
        • Principal Investigator:
          • Daragh Heitzman, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Description

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).

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Clene Nanomedicine

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

March 7, 2022

First Submitted That Met QC Criteria

March 7, 2022

First Posted (Actual)

March 16, 2022

Study Record Updates

Last Update Posted (Actual)

October 26, 2023

Last Update Submitted That Met QC Criteria

October 25, 2023

Last Verified

October 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CNMAu8.EAP02

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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