Intermediate Expanded Access Protocol for ALS

Sponsor

Clene Nanomedicine (Industry)

Overall Status

Available

CT.gov ID

NCT04081714

Collaborator

Massachusetts General Hospital (Other)

Location

Study Details

Study Description

Brief Summary

This is a single-center intermediate expanded access program to provide access to the investigational product, CNM-Au8, up to 30 participants diagnosed with ALS.

Condition or Disease	Intervention/Treatment	Phase
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 thirty (30) participants diagnosed with ALS.

Safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 treatment in ALS participants will be evaluated. Visits will occur at MGH, remotely over telephone, or via tele-visit. Collection of samples for pharmacokinetic and pharmacodynamic analysis may not be feasible for visits completed remotely. 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. Participants may initiate treatment on the same day as the Screening visit, provided all inclusion and exclusion criteria have been fulfilled prior to treatment initiation.

There will be four study periods:

A screening period over up to four (4) weeks (Screening Period);
Initial treatment period of twenty-four (24) weeks (Treatment Period 1);
Up to three consecutive optional follow-on treatment periods of twenty-four (24) week duration:
Treatment Period 2,
Treatment Period 3,
Treatment Period 4;
A four (4) week safety follow-up period (End-of-Study [EOS] Assessment).

Per protocol all participants will receive open-label oral treatment daily up to 24-weeks during Treatment Period 1. Participants may optionally continue on open-label therapy for up to three additional consecutive 24-week treatment periods (e.g., Treatment Period 2, Treatment Period 3, Treatment Period 4) up to a maximum duration of ninety-six (96) weeks.

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. Select visit assessments may be collected remotely, via tele-visit with site staff.

Study Design

Study Type:

Expanded Access

Official Title:

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

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Inclusion Criteria:

Able to understand and give written informed consent.
Male or female participants aged 18 years or greater (inclusive) at the time of ALS diagnosis.
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 subinvestigator at the site).
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.
In the judgement of the Investigator, the participant's expected survival is greater than six- months.
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:

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.
Based on the investigator's judgment, participants who may have difficulty complying with the protocol and/or any study procedures.
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.
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 109 per liter) or eosinophilia (absolute eosinophil count of

500 eosinophils per microliter) at Screening.

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).
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), unless waived at the discretion of the Sponsor (e.g., for other investigational drug trials with no known interaction with CNM-Au8).
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.
Females of child-bearing potential, or men, who are unwilling or unable to use accepted methods of birth control.
History of gold allergy.