Intermediate Expanded Access Protocol CNMAu8.EAP04
- Conditions
- ALSAmyotrophic Lateral SclerosisPALS
- Registration Number
- NCT06408727
- Lead Sponsor
- Clene Nanomedicine
- Brief Summary
An Intermediate Expanded Access Protocol (EAP) with CNM-Au8 for Amyotrophic Lateral Sclerosis for NIH Grant RFA-NS-23-012
- Detailed Description
An Intermediate Expanded Access Protocol (EAP) with CNM-Au8 for Amyotrophic Lateral Sclerosis for NIH Grant RFA-NS-23-012. The primary objective of this intermediate EAP is to provide access to the investigational product, CNM-Au8 30mg, for up to 180 people living with ALS.
Recruitment & Eligibility
- Status
- AVAILABLE
- Sex
- All
- Target Recruitment
- Not specified
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Able to understand and give written informed consent.
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Male or female participants aged 18 years or greater (inclusive) at the time of informed consent completion.
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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).
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Participant is able to daily consume up to 60 mL of the investigational drug suspension without substantial dysphagia, OR can intake the investigational product through a feeding tube.
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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).
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Participant has a baseline score by the TRICALS risk calculator that is less than -2 (i.e., participant is not at increased risk of early death; https://tricals.shinyapps.io/risk-profile/).
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Participant meets the following criteria:
- Baseline Vital Capacity >15% predicted,
- Baseline ALSFRS-R Score >8, and,
- Baseline BMI >17.5 kg/m2
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Participants have established care with a neurologist at the specialized ALS center or remotely enrolling site involved in the study and will maintain this clinical care throughout the duration of the EAP within the United States.
- Participant is eligible for participation in any double-blind placebo-controlled study the treatment of ALS at the same research site.
- 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.
- Based on the investigator's judgment, participants who may have difficulty complying with the protocol and/or any study procedures.
- Within the prior 90-days the participant has had clinically significant findings on standard hepatic, hematologic, or renal safety assays, including but not limited to: (i) ALT or AST ≥ 3 times upper limits of normal, (ii) direct (conjugated) with bilirubin ≥2 times upper limits of normal, (iii) low platelet counts (< 150 x 109 per liter) or eosinophilia (absolute eosinophil count of ≥ 500 eosinophils per microliter), (iv) serum creatinine >1.2 mg/dL, or (v) eGFR < 45 ml/min per 1.73 m2.
- Participant is currently involved in another placebo-controlled clinical trial (note: concomitant therapy with other investigational products is permitted with certain restrictions-see concomitant medications below).
- 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.
- Females of child-bearing potential, or men, who are unwilling or unable to use accepted methods of birth control.
- History of gold allergy.
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (5)
Texas Neurology
🇺🇸Dallas, Texas, United States
Synapticure
🇺🇸Chicago, Illinois, United States
Pennsylvania State University
🇺🇸Hershey, Pennsylvania, United States
Nova Southeastern University
🇺🇸Fort Lauderdale, Florida, United States
Duke University
🇺🇸Durham, North Carolina, United States