NCT07142291

PHENOGENE-1A (Cromolyn) Treatment in Patients With Mild to Moderate ALS

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)

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 105 (estimated)
• Sponsor: PhenoNet, Inc. · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Amyotrophic Lateral Sclerosis (ALS)

Interventions

Type	Name	Description
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

Timeline

• Start date: 2025-11-25
• Primary completion: 2027-11-01
• Completion: 2028-03-01
• First posted: 2025-08-26
• Last updated: 2026-04-01

Locations

17 sites across 6 countries: United States, Czechia, Germany, Poland, Serbia, Spain

Regulatory

• FDA-regulated drug study