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)

Official 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.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: RANDOMIZED
• Model: PARALLEL
• Masking: QUADRUPLE
• Enrollment: 105 participants

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

Primary Outcomes

• Absolute change in ALSFRS-R total score from baseline to Week 24 (Baseline to Week 24)
• Incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) (Baseline to Week 24)

Secondary Outcomes

• Mean rank for CAFS at Week 24 (Baseline to Week 24)
• Time to event requiring full-time or nearly full-time respiratory support (Randomization to Week 24)
• Mean change in percent predicted forced vital capacity (%FVC) from baseline to Week 24 (Baseline to Week 24)
• Mean change in peak inspiratory flow rate (PIFR) from baseline to Week 24 (Baseline to Week 24)
• Absolute Values and Changes From Baseline in Vital Signs From Baseline to Week 24 by Treatment Arm (Baseline to Week 24)

Trial Locations

• Honor Health Neurology - Bob Bove Neuroscience Institute, Scottsdale, Arizona, United States
• University of California San Diego, La Jolla, California, United States
• Sutter Health - California Pacific Medical Center Research Institute, San Francisco, California, United States
• Mayo Clinic Jacksonville, Jacksonville, Florida, United States
• Lange Neurology, New York, New York, United States
• NEUROHK s.r.o., Hradec Králové, Czechia
• Thomayer University Hospital - Fakultni Thomayerova nemocnice, Prague, Czechia
• Charité Centrum für Neurologie, Neurochirurgie und Psychiatrie, Berlin, Germany
• DIAKOVERE Henriettenstift - Klinik für Neurologie und Klinische Neurophysiologie, Hanover, Germany
• Universitaetsklinikum Schleswig-Holstein, Lübeck, Germany
• ...and 7 more locations

More Amyotrophic Lateral Sclerosis (ALS) Trials

View all Amyotrophic Lateral Sclerosis (ALS) clinical trials