PhenoNet, Inc.
The purpose of this study is to test the effects of PHENOGENE-1A, which is the treatment under investigation in this study. This research will investigate if PHENOGENE-1A can help people with ALS by measuring their function using the ALS Functional Rating Scale Revised (ALSFRS-R), measuring lung function using pulmonary function tests (PFTs), such as forced vital capacity (FVC), and measuring neuro-inflammatory biomarkers in the blood.
This is a Phase IIB, randomized, double-blind, placebo-controlled study designed to assess the effects of PHENOGENE-1A (oral inhalation via dry powder inhaler \[DPI\]) in subjects with mild to moderate ALS disease. Eligible subjects will be randomized to receive either low dose PHENOGENE-1A (34.2 mg per day: in 2 doses of 17.1 mg and matching placebo BID), high dose PHENOGENE-1A (68.4 mg per day: 34.2 mg BID), or placebo (2 matching placebo capsules BID) (see table below), in a 2:2:1 ratio. Subjects will receive treatment for a duration of 24 weeks. All subjects must be on a stable dose of Ril…
Inclusion Criteria: 1. Diagnosis of ALS; the diagnosis of ALS defined by revised El Escorial criteria as follows: 1. Evidence of lower motor neuron (LMN) degeneration by clinical, electrophysiological, or neuropathological examination. 2. Evidence of upper motor neuron (UMN) degeneration by clinical examination. 3. Progressive spread of symptoms or signs within a region or to other regions, as determined by clinical examination or the history of disease progression. 4. Absence of electrophysiological, neuroimaging, or pathological evidence of other diseases that might explain the…
17.1 mg, BID, oral inhalation
34.2 mg, BID, oral inhalation via dry powder inhaler
Placebo comparator matched to active treatment.
50 mg, oral tablet, BID, standard of care treatment