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Trial record 230 of 637 for:    Amyotrophic Lateral Sclerosis

Intermediate Expanded Access Protocol for ALS

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ClinicalTrials.gov Identifier: NCT04081714
Expanded Access Status : Available
First Posted : September 9, 2019
Last Update Posted : September 9, 2019
Sponsor:
Collaborator:
Massachusetts General Hospital
Information provided by (Responsible Party):
Clene Nanomedicine

Brief Summary:
This is a single-center intermediate expanded access program to provide access to the investigational product, CNM-Au8, up to twenty participants diagnosed with ALS who are otherwise ineligible for participation in clinical studies with CNM-Au8.

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

Detailed Description:
This is a single-center intermediate expanded access program to provide access to the investigational product, CNM-Au8, up to twenty participants diagnosed with ALS. Safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 treatment in ALS participants will be evaluated. Visits will occur at Massachusetts General Hospital or via tele-visit. Participants will be screened over up to a 4-week period prior to treatment initiation. Participants who meet the inclusion criteria and none of the exclusionary criteria may be enrolled into the EAP. Per protocol all participants will receive open label oral treatment daily up to 24-weeks during Treatment Period 1.

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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
Official Title: An Intermediate Expanded Access Protocol for Amyotrophic Lateral Sclerosis With CNM-Au8



Intervention Details:
  • Drug: CNM-Au8
    CNM-Au8 is a dark red/purple-colored liquid formulation consisting of a stable suspension of faceted clean surfaced elemental gold nanocrystals in buffered USP water with a concentration of up to 0.5 mg/mL of gold. The formulation is buffered by sodium bicarbonate present at a concentration of 0.546 mg/mL. There are no other excipients. The drug product is formulated to be taken orally and will be provided in single dose HDPE containers.
    Other Name: Nanocrystalline gold

Information from the National Library of Medicine

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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 whose conditions are defined as "definite ALS" or "probable ALS" or "possible ALS" diagnostic criteria by the revised El Escorial Criteria as determined by a neurologist specializing in ALS (e.g., the Principal Investigator or sub-investigator at the site).
  4. For participants presently taking riluzole or edaravone, stable dosing over the prior 4-weeks.
  5. Participant is able to daily consume 60 mL of the investigational drug suspension without substantial dysphagia, OR can intake the drug through a gastrostomy tube.
  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.

Exclusion Criteria:

  1. Participant with a history of any clinically significant or unstable medical condition based on the Investigator's judgment that may interfere with assessment of the study objectives.
  2. Based on the investigator's judgment, participants who may have difficulty complying with the protocol and/or any study procedures.
  3. Participant with clinically significant abnormalities in hematology, blood chemistry, ECG, or physical examination not resolved by the Baseline visit which according to Investigator can interfere with EAP participation.
  4. Participants with clinically significant hepatic or renal dysfunction or clinical laboratory findings that would limit the interpretability of change in liver or kidney function, or those with low platelet counts (< 150 x 10^9 per liter) or eosinophilia (absolute eosinophil count of ≥ 500 eosinophils per microliter) at Screening.
  5. Participants with a prior history of, or positive serological assay for the presence of HIV infection, or laboratory evidence of active or chronic infection with hepatitis C (HCV) or hepatitis B (HBV). Note, participants who have been vaccinated for HBV and have detectable HB antibodies are not excluded unless positive for surface antigen (HBsAg).
  6. Participant has participated in any other investigational drug trial (within 4-weeks prior to screening or at least five-half lives of the investigational product), and thereafter.
  7. Females who are pregnant or nursing or who plan to get pregnant during the course of this clinical trial or within 6 months of the end of this trial.
  8. Females of child-bearing potential, or men, who are unwilling or unable to use accepted methods of birth control.
  9. History of gold allergy.

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): NCT04081714


Contacts
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Contact: Robert Glanzman, MD +1 (801) 676-9695 info@clene.com
Contact: Austin Rynders, BS +1 (801) 676-9695 info@clene.com

Locations
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United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Contact: Judith Carney, RN    617-724-8995    jcarey8@partners.org   
Principal Investigator: Merit Cudkowicz, MD         
Sponsors and Collaborators
Clene Nanomedicine
Massachusetts General Hospital

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Responsible Party: Clene Nanomedicine
ClinicalTrials.gov Identifier: NCT04081714     History of Changes
Other Study ID Numbers: CNMAu8.EAP01
First Posted: September 9, 2019    Key Record Dates
Last Update Posted: September 9, 2019
Last Verified: September 2019
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