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NCT07142291PHASE2Recruiting

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

PhenoNet, Inc.

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

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 UMN or LMN degeneration and exclusion of other causes. 2. Male or female subjects aged 18 to 75 years inclusive. 3. Must provide written informed consent for study-related procedures. 4. Must be capable of completing all study-related procedures, assessments, and visits in the judgment of Investigator. 5. Disease duration from ALS symptom onset of motor weakness ≤24 months. 6. ALSFRS-R total score ≥38 at screening visit. 7. ALSFRS-R Breathing subscore should be ≥9 at the time of screening. 8. ALSFRS-R Bulbar subscore should be ≥9 at the time of screening. 9. Forced vital capacity \>70% of predicted value. 10. PIFR ≥100 L/minute. 11. Must be receiving a stable dose of standard-of-care treatment Riluzole for 4-weeks before signing informed consent. 12. Female subjects who are of childbearing potential must agree to use of highly effective methods of contraception consistent with local regulations during the study, and for 3 months after the study drug administration. Examples include the following, but not limited to: 1. Combined (estrogen and progestogen containing) or progestogen-only hormonal contraceptives; 2. Intrauterine device or intrauterine hormone-releasing system; OR 3. Post-menopausal status must have experienced their last menstrual period minimum of 1 year prior to study drug administration; OR 4. Surgically sterilized. Female subject should be willing to not donate egg during the trial and for 3 months after the last dose of the study drug. 13. Male subjects who are sexually active with a female of childbearing potential must agree to use highly effective contraception as described above, or a combination of 2 acceptable methods of contraception (e.g., a barrier method along with a female partner using a hormonal contraceptive method), in accordance with local regulations, throughout the duration of the study, and for 3 months after the last dose of the study drug. (Male subject should be willing to not donate sperm during the trial and for 3 months after the last dose of the study drug.) Exclusion Criteria: 1. ALSFRS-R score change (decrease) by 2.5 or more points between the screening visit and Day 1 (baseline) score. 2. Bulbar onset ALS (\<9 bulbar subscore) 3. Any use of non-invasive ventilation (e.g., continuous positive airway pressure, non-invasive bi-level positive airway pressure or non-invasive volume ventilation) for any portion of the day, or mechanical ventilation via tracheostomy, or on any form of oxygen supplementation. 4. Any other significant neurological disorder which can interfere with study assessments, e.g., significant cognitive impairment and/or clinical dementia. 5. Significant psychiatric illness like schizophrenia, bipolar disorder etc. Subjects with depression can be included, only if the depression has been stable and no episode of major depression has occurred in the past year. 6. Severe cardiac disease (e.g., QTc\>500 ms), Torsade de Pointes, evidence of significant heart failure (New York Heart Association \[NYHA\] Class 3 or greater, myocardial infarction or unstable angina in the 6 months prior to screening). 7. Any moderate-to-severe pulmonary disease or difficulty taking inhaled drugs. 8. Inability to tolerate the administration of an oral inhaled powder via DPI. 9. Has taken any investigational product within 30 days or 5 half lives of the drug, whichever is longer, prior to dosing. 10. Taking inhaled protein products on a chronic basis (such as insulin, parathyroid hormone, etc). 11. Subjects with a body weight of 32 kg or less, or a body mass index of \<17.5 or \>35.0 at time of screening. 12. Moderate-to-severe liver disease: aspartate aminotransferase (AST), alanine aminotransferase (ALT) \>3 times the upper limit of normal; total bilirubin \> 1.5 x ULN.; subjects with hepatic diseases such as hepatic cirrhosis, hepatic cancer and active hepatitis. 13. Moderate-to-severe renal disease: creatinine clearance \<45 mL/min/1.73 m2 (by Cockcroft-Gault calculation). 14. Any clinically significant disorder or laboratory abnormality that, in the Investigator's opinion, could interfere with the subject's participation in the study, place the subject at increased risk, or confound interpretation of the study results. 15. Pregnant or breast-feeding females.

Interventions

DRUG

Cromolyn Sodium (34.2 mg BID)

DRUG

Cromolyn Sodium (17.1 mg BID)

DRUG

Placebo

DRUG

Riluzole (100 mg)

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Conditions

Amyotrophic Lateral Sclerosis (ALS)

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