Participant enrollment is complete for a phase 2 trial (NCT04414345) evaluating the efficacy, safety, pharmacokinetics, and pharmacodynamics of nanocrystalline gold (CNM-Au8; Clene Nanomedicine, Inc., Salt Lake City, UT) intended to improve muscle function for individuals with early symptomatic amyotrophic lateral sclerosis (ALS).
The RESCUE-ALS (NCT04098406) study will evaluate muscle function with nanocrystalline gold vs placebo treatment. Muscle function will be measured with the motor unit number index (MUNIX), which quantitatively reflects loss or preservation of motor neurons in ALS. The study will also assess the affect of nanocrystalline gold on neuronal bioenergetics, reducing reactive oxygen species, and promoting protein homeostasis, thought to be involved in disease progression.
Participants in the randomized placebo-controlled study will receive nanocrystalline gold or placebo in addition to their current standard of care for 36 weeks. Nanocrystalline gold is a concentrated, aqueous suspension of clean-surfaced faceted gold nanocrystals (Au) believed to act catalytically to support intracellular biologic reactions.
“In multiple preclinical models of ALS, CNM-Au8 has been shown to promote motor neuron health, reduce neuron loss, and preserve motor neuron function,” said Robert Glanzman, MD, FAAN, chief medical officer, Clene Nanomedicine. “We believe the unique mechanisms of CNM-Au8 provide the potential to be an effective disease-modifying therapy for patients with ALS.”