NCT04098406

Therapeutic Nanocatalysis to Slow Disease Progression of Amyotrophic Lateral Sclerosis (ALS)

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study in Early Symptomatic Amyotrophic Lateral Sclerosis Patients on Stable Background Therapy to Assess Bioenergetic Catalysis With CNM-Au8 to Slow Disease Progression in ALS.

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 45 (actual)
• Sponsor: Clene Nanomedicine · Industry
• Sex: All
• Age: 30 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

The objective of this trial was to assess the efficacy, safety, and PK/PD effects of CNM-Au8 as a disease-modifying agent for the treatment of ALS by utilizing electrophysiological measures to detect preservation of motor neuron function. The primary endpoint was the mean change in the average difference between active treatment and placebo from Baseline through Week 36 evaluated by electromyography.

Detailed description

This was a multi-centre randomized, double-blind, parallel group, placebo-controlled study of the efficacy, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 in patients who are newly symptomatic within 24-months of Screening and with a clinically probable or possible or definite ALS diagnosis per the Awaji-Shima criteria. Patients were screened over up to a 6-week period. Patients who met the inclusion criteria and none of the exclusionary criteria were enrolled into the clinical study. Patients were randomized 1:1 into one of two groups: either active treatment with CNM-Au8 30 mg or Placebo. All patients received their randomized oral treatment daily over thirty-six (36) consecutive weeks during the Treatment Period. There were up to four study periods: 1. Up to a six (6) week screening period (Screening Period); 2. A thirty-six (36) week blinded randomized treatment period (Treatment Period); 3. Up to a forty-eight (48) week optional open-label extension period (Open-Label Period); 4. A four (4) week safety follow-up period following completion of either the Treatment or Open-Label period or in the case of Early Termination (Safety Follow-Up Period). Per protocol, all patients received their blinded and randomized oral treatment daily over at least 36 consecutive weeks during the Treatment Period. For those patients not transitioning into the optional OLE period, patients completed a safety follow-up visit 4-weeks following study drug discontinuation. An independent DSMB was responsible for monitoring the safety of the study on a quarterly basis and ad hoc at the request of the DSMB or the Sponsor (e.g., in the event of unexpected SAEs) to review data throughout the Treatment Period. The DSMB may make recommendations on the conduct of the study, including study termination. Appropriate procedures will be detailed in a DSMB Charter.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	CNM-Au8	CNM-Au8 is a dark red/purple-colored liquid formulation consisting of a stable suspension of faceted clean surfaced elemental gold nanocrystals in buffered deionized water with a nominal concentration of 0.5 mg/mL of gold. The formulation is buffered by sodium bicarbonate present at a concentration of 0.546 mg/mL. There are no other excipients. The drug product is formulated to be taken orally and will be provided in single dose 60 mL HDPE containers.
DRUG	Placebo	Placebo was liquid with identical color and taste

Timeline

• Start date: 2019-12-19
• Primary completion: 2021-07-13
• Completion: 2021-07-13
• First posted: 2019-09-23
• Last updated: 2024-07-03
• Results posted: 2024-07-03

Locations

2 sites across 1 country: Australia

Regulatory

• FDA-regulated drug study