Intermediate Expanded Access Protocol (EAP) CNMAu8.EAP02

Amyotrophic Lateral Sclerosis Clinical Trial

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

Status Available

Clinical Trial Summary

The primary objective of the intermediate expanded access protocol is to provide access to the investigational product, CNM-Au8, to up to 300 people living with ALS (pALS).

No formal clinical hypotheses are being evaluated with concurrent controls.

Secondary objectives include assessment of the safety of CNM-Au8 treatment in pALS. Safety will be assessed through the frequency of serious adverse events (SAEs), treatment-emergent adverse events (TEAEs) assessed as 'severe', discontinuations due to TEAEs, and laboratory abnormalities assessed as clinically significant during routine clinical monitoring (as applicable).

Clinical Trial Description

This is a multi-center intermediate expanded access program to provide access to the investigational product, CNM-Au8, up to 300 participants diagnosed with ALS.

The safety of CNM-Au8 treatment in ALS participants will be evaluated. Visits will occur at a clinic or remotely via telephone or video-visit. Visits may be conducted remotely due to COVID-19-related pandemic concerns, or if due to ALS disease progression.

Participants who meet the inclusion criteria and none of the exclusionary criteria may be enrolled into the EAP.

There will be three study periods:

1. A treatment period of forty-eight (48) weeks (Treatment Period 1);

2. Additional optional follow-on treatment period(s) of up to forty-eight (48) weeks duration may be added at the discretion of the Sponsor and Site Investigator (e.g., Treatment Period 2, Treatment Period 3);

3. A four (4) week safety follow-up period (End-of-Study [EOS]Assessment).

All participants will receive open-label oral treatment daily up to 48 weeks during Treatment Period 1. Additional 48-week treatment periods may be approved at the discretion of the Sponsor. The EAP may be discontinued at any time at the Sponsor's discretion.

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.

Visit assessments may be collected remotely, via tele-visit with study site staff. Investigational product may be shipped by the site to participants who do attend in-clinic visits. ;

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

• NCT number: NCT05281484
• Study type: Expanded Access
• Source: Clene Nanomedicine
• Contact: Austin Rynders, BS
• Phone: (801)676-9695
• Email: eap@clene.com
• Status: Available