Intermediate Expanded Access Protocol for ALS

Amyotrophic Lateral Sclerosis Clinical Trial

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

Status Available

Clinical Trial Summary

This is a single-center intermediate expanded access program to provide access to the investigational product, CNM-Au8, up to 40 participants diagnosed with ALS.

Clinical Trial Description

This is a single-center intermediate expanded access program to provide access to the investigational product, CNM-Au8, up to forty (40) 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 three study periods:

1. A screening period over up to four (4) weeks (Screening Period);

2. Initial treatment period of twenty-four (24) weeks (Treatment Period 1);

a. Additional consecutive optional follow-on treatment periods of twenty-four (24) weeks duration may be offered at the discretion of the Investigator and Sponsor (e.g., Treatment periods 2, 3, 4, 5, 6+)

3. 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, Treatment Period 5, Treatment Period 6 (n), Treatment Period n+1).

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. ;

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

• NCT number: NCT04081714
• Study type: Expanded Access
• Source: Clene Nanomedicine
• Contact: Austin Rynders, BS
• Phone: +1 (801) 676-9695
• Email: eap@clene.com
• Status: Available