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Intermediate Expanded Access Protocol (EAP) CNMAu8.EAP02

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05281484
Expanded Access Status : Available
First Posted : March 16, 2022
Last Update Posted : October 26, 2023
Sponsor:
Information provided by (Responsible Party):
Clene Nanomedicine

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.

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



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).


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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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).


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05281484


Contacts
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Contact: Austin Rynders, BS (801)676-9695 info@clene.com
Contact: Jeremy Evan, PA-C (801)676-9695 info@clene.com

Locations
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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
Holy Cross Hospital Available
Davie, Florida, United States, 33314
Contact: Gustavo Alameda, MD    954-414-9750    gustavo.alameda@holy-cross.com   
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    919-613-2681    Rachel.m.ward@duke.edu   
Contact: Hailey Zampa, CRC    919-684-8267    Hailey.zampa@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: Yesasvini Devabhaktuni    267-582-6061    yesasvini.devabhaktuni@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         
Sponsors and Collaborators
Clene Nanomedicine
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Responsible Party: Clene Nanomedicine
ClinicalTrials.gov Identifier: NCT05281484    
Other Study ID Numbers: CNMAu8.EAP02
First Posted: March 16, 2022    Key Record Dates
Last Update Posted: October 26, 2023
Last Verified: October 2023
Keywords provided by Clene Nanomedicine:
Expanded access
ALS
Amyotrophic Lateral Sclerosis
CNM Au8
Au8
Additional relevant MeSH terms:
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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