Therapeutic Nanocatalysis to Slow Disease Progression of Amyotrophic Lateral Sclerosis (ALS)
Condition: Amyotrophic Lateral Sclerosis
MeSH terms: Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis; Disease Progression
Study type: Interventional
Phase: Phase 2

Summary: 
      The objective of this trial is 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 is the mean
      change in the average difference between active treatment and placebo from Baseline through
      Week 36 evaluated by electromyography.
    

Detailed description: 
      This is 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 may be screened over up to a 6-week period. Patients who meet the inclusion criteria
      and none of the exclusionary criteria will be enrolled into the clinical study. Patients will
      be randomized 1:1 into one of two groups: either active treatment with CNM-Au8 30 mg or
      Placebo.

      All patients will receive their randomized oral treatment daily over thirty-six (36)
      consecutive weeks during the Treatment Period.

      There will be 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 will receive 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 will complete a
      safety follow-up visit 4-weeks following study drug discontinuation.

      An independent DSMB will be 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.
    

Intervention name(s): CNM-Au8; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): 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.; Placebo is liquid with identical color and taste
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: randomized, double-blind, parallel group, placebo-controlled study
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Gold; Nanocrystal; electromyography; ALS; Nanoparticle