Intermediate Expanded Access Protocol (EAP) CNMAu8.EAP02

Sponsor

Clene Nanomedicine (Industry)

Overall Status

No longer available

CT.gov ID

NCT05281484

Collaborator

(none)

Locations

Study Details

Study Description

Brief Summary

The primary objective of the intermediate expanded access protocol is to provide access to the investigational product, CNM-Au8, to up to fifteen (15) participants with ALS.

No formal clinical hypotheses are being tested. Secondary objectives include assessment of the safety in participants diagnosed with ALS.

Safety will be assessed through the frequency of treatment-emergent adverse events, serious adverse events, discontinuations due to adverse events, and changes in ALS disease-specific measures, such as the ALS Functional Rating Scale-Revised (ALSFRS-R) scores and Slow Vital Capacity (SVC), as practically feasible.

Condition or Disease	Intervention/Treatment	Phase
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 fifteen (15) participants diagnosed with ALS.

The safety of CNM-Au8 treatment in ALS participants will be evaluated. Visits will occur remotely over telephone, via tele-visit or at a clinic visit, if possible. The Screening Visit and Baseline Visits must be conducted onsite. Other visits may be conducted remotely due to COVID-19 related pandemic concerns, or if due to ALS disease progression. 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 forty-eight (48) weeks (Treatment Period 1);
An additional optional follow-on treatment period of up to forty-eight (48) weeks duration may be added at the discretion of the Sponsor, Healey Coordination Center (HCC), and Site Investigator (Treatment Period 2);
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 48-weeks during Treatment Period 1. Participants who continue on open-label therapy during the additional consecutive 48-week treatment periods (e.g., Treatment Period 2) will have 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 study site staff.

Study Design

Study Type:

Expanded Access

Official Title:

A Second 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 "lab supported 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 for this study).
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.
Slow vital capacity greater than or equal to twenty-precent predicted (>=20%). For participants with bulbar onset ALS who may have difficulty performing the assessment, or who have a tracheostomy, enrollment may be approved on a case-by-case basis upon discussion with, and written approval from, the Sponsor's Medical Representative.
In the judgement of the Investigator, OR as documented by the ENCALS prediction model (http://encalssurvivalmodel.org/, (Westeneng et al. 2018), 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 is eligible for participation in the HEALEY ALS Platform trial (NCT04297683).
Participant has 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, 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.
Participant is involved in another clinical trial or expanded access program, OR has participated in any other investigational drug trial (within 4-weeks prior to screening or at least five-half lives of the investigational product).
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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Barrow Neurological Institute	Phoenix	Arizona	United States	85013
2	Hospital for Special Care	New Britain	Connecticut	United States	06053
3	Holy Cross Hospital	Davie	Florida	United States	33314
4	Nova Southeastern University	Davie	Florida	United States	33314