A Phase IIB Randomized, Double-Blind, Placebo-Controlled, Multi-Dose Study to Evaluate the Effects of PHENOGENE-1A (Cromolyn) as an Adjuvant Treatment in Subjects With Mild to Moderate Amyotrophic Lateral Sclerosis (ALS)

PhenoNet, Inc.

Summary

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.

Description

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…

Eligibility

Age range: 18–75 years
Sex: All
Healthy volunteers: No

Detailed criteria

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…

Interventions

Drug
Cromolyn Sodium (34.2 mg BID)
17.1 mg, BID, oral inhalation
Drug
Cromolyn Sodium (17.1 mg BID)
34.2 mg, BID, oral inhalation via dry powder inhaler
Drug
Placebo
Placebo comparator matched to active treatment.
Drug
Riluzole (100 mg)
50 mg, oral tablet, BID, standard of care treatment

Locations (17)

Honor Health Neurology - Bob Bove Neuroscience Institute
Scottsdale, Arizona
University of California San Diego
La Jolla, California
Sutter Health - California Pacific Medical Center Research Institute
San Francisco, California
Mayo Clinic Jacksonville
Jacksonville, Florida
Lange Neurology
New York, New York
NEUROHK s.r.o.
Hradec Králové