PHENOGENE-1A (Cromolyn) Treatment in Patients With Mild to Moderate ALS
Start Date
11/25/2025
Completion Date
6/1/2027
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.
Detailed 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 Riluzole 50 mg BID (100 mg daily) for at least 4 weeks prior to randomization and must continue their Riluzole regimen, 50 mg BID (100 mg daily), as standard-of-care treatment, throughout the 24 week treatment period.
Eligibility Criteria
Age Range: 18 years to 75 years
Interventions
Cromolyn Sodium (34.2 mg BID)
Cromolyn Sodium (17.1 mg BID)
Placebo
Riluzole (100 mg)
Conditions
Locations
Honor Health Neurology - Bob Bove Neuroscience Institute
Scottsdale, Arizona 85251
United States
University of California San Diego
La Jolla, California 92037
United States
Sutter Health - California Pacific Medical Center Research Institute
San Francisco, California 94115
United States
Mayo Clinic Jacksonville
Jacksonville, Florida 32224
United States
Lange Neurology
New York, New York 10065
United States
NEUROHK s.r.o.
Hradec Králové, 50002
Czechia
Fakultni Nemocnice Hradec Kralove
Hradec Králové, 50005
Czechia
Thomayer University Hospital - Fakultni Thomayerova nemocnice
Prague, 14059
Czechia
Charité Centrum für Neurologie, Neurochirurgie und Psychiatrie
Berlin, 13353
Germany
DIAKOVERE Henriettenstift - Klinik für Neurologie und Klinische Neurophysiologie
Hanover, 30171
Germany
Universitaetsklinikum Schleswig-Holstein
Lübeck, 23538
Germany
Michalski i Partnerzy Lekarze Spółka Partnerska
Krakow, 31-426
Poland
SP ZOZ Szpital Uniwersytecki w Krakowie
Krakow, 51-503
Poland
Centrum Medyczne NeuroProtect (NeuroProtect Medical Center)
Warsaw, 01-684
Poland
City Clinic Research
Warsaw, 02-473
Poland
University Clinical Center of Serbia
Belgrade, 11000
Serbia
Hospital General Universitario Dr. Balmis de Alicante
Alicante, 3010
Spain
Barcelona Sea Hospital (Hospital Del Mar De Barcelona)
Barcelona, 8003
Spain
Hospital Universitario Vall D Hebron
Barcelona, 8035
Spain
Hospital Universitario Ramon Y Cajal
Madrid, 28034
Spain
Hospital Regional Universitario De Malaga
Málaga, 29010
Spain
Hospital Universitario Virgen Del Rocio
Seville, 41013
Spain