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

NCT07142291 | Recruiting Phase 2 FDA Drug

• 1 other identifier: PHENOALS-001
• Study Type: interventional
• Target: 105
• Locations: 6 countries
• Sites: 17

Brief 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.

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Keywords

Amyotrophic Lateral Sclerosis; Lou Gehrigs Disease; ALS

Outcome Measures

Primary Outcomes (2)

• Absolute change in ALSFRS-R total score from baseline to Week 24

  To evaluate the effects of PHENOGENE-1A (cromolyn) on functional changes in subjects with mild to moderate ALS using the ALS Functional Rating Scale-Revised (ALSFRS-R).

  Baseline to Week 24

• Incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)

  To evaluate the safety of PHENOGENE-1A following 24 weeks of twice daily (BID) treatment.

  Baseline to Week 24

Secondary Outcomes (11)

• 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

Other Outcomes (1)

• Change in plasma neuroinflammatory biomarkers from baseline to Week 24

  Baseline (pre-dose) to Week 24

Study Arms (3)

Low Dose PHENOGENE-1A (17.1 mg BID)

EXPERIMENTAL

17.1 mg, BID, oral inhalation via dry powder inhaler

Drug: Cromolyn Sodium (17.1 mg BID); Drug: Riluzole (100 mg)

High Dose PHENOGENE-1A (34.2 mg BID)

EXPERIMENTAL

34.2 mg, BID, oral inhalation via dry powder inhaler

Drug: Cromolyn Sodium (34.2 mg BID); Drug: Riluzole (100 mg)

Placebo

PLACEBO COMPARATOR

Placebo comparator matched to active treatment, BID, oral inhalation via dry powder inhaler

Drug: Placebo; Drug: Riluzole (100 mg)

Interventions

Cromolyn Sodium (17.1 mg BID) DRUG

34.2 mg, BID, oral inhalation via dry powder inhaler

Also known as: Cromolyn, Sodium Cromoglycate, PHENOGENE-1A

Low Dose PHENOGENE-1A (17.1 mg BID)

Placebo DRUG

Placebo comparator matched to active treatment.

Placebo

Riluzole (100 mg) DRUG

50 mg, oral tablet, BID, standard of care treatment

Also known as: Rilutek

High Dose PHENOGENE-1A (34.2 mg BID); Low Dose PHENOGENE-1A (17.1 mg BID); Placebo

Cromolyn Sodium (34.2 mg BID) DRUG

17.1 mg, BID, oral inhalation

Also known as: Cromolyn, Sodium Cromoglycate

High Dose PHENOGENE-1A (34.2 mg BID)

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of ALS; the diagnosis of ALS defined by revised El Escorial criteria as follows:
• Evidence of lower motor neuron (LMN) degeneration by clinical, electrophysiological, or neuropathological examination.
• Evidence of upper motor neuron (UMN) degeneration by clinical examination.
• Progressive spread of symptoms or signs within a region or to other regions, as determined by clinical examination or the history of disease progression.
• Male or female subjects aged 18 to 75 years inclusive.
• Must provide written informed consent for study-related procedures.
• Must be capable of completing all study-related procedures, assessments, and visits in the judgment of Investigator.
• Disease duration from ALS symptom onset of motor weakness ≤24 months.
• ALSFRS-R total score ≥38 at screening visit.
• ALSFRS-R Breathing subscore should be ≥9 at the time of screening.
• ALSFRS-R Bulbar subscore should be ≥9 at the time of screening.
• Forced vital capacity \>70% of predicted value.
• PIFR ≥100 L/minute.
• Must be receiving a stable dose of standard-of-care treatment Riluzole for 4-weeks before signing informed consent.
• 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:

You may not qualify if:

• ALSFRS-R score change (decrease) by 2.5 or more points between the screening visit and Day 1 (baseline) score.
• Bulbar onset ALS (\<9 bulbar subscore)
• 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.
• Any other significant neurological disorder which can interfere with study assessments, e.g., significant cognitive impairment and/or clinical dementia.
• 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.
• 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).
• Any moderate-to-severe pulmonary disease or difficulty taking inhaled drugs.
• Inability to tolerate the administration of an oral inhaled powder via DPI.
• Has taken any investigational product within 30 days or 5 half lives of the drug, whichever is longer, prior to dosing.
• Taking inhaled protein products on a chronic basis (such as insulin, parathyroid hormone, etc).
• 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.
• 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.
• Moderate-to-severe renal disease: creatinine clearance \<45 mL/min/1.73 m2 (by Cockcroft-Gault calculation).
• 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.
• Pregnant or breast-feeding females.

Sponsors & Collaborators

• PhenoNet, Inc.: lead

Study Sites (17)

Honor Health Neurology - Bob Bove Neuroscience Institute

Scottsdale, Arizona, 85251, United States

RECRUITING

University of California San Diego

La Jolla, California, 92037, United States

RECRUITING

Sutter Health - California Pacific Medical Center Research Institute

San Francisco, California, 94115, United States

RECRUITING

Mayo Clinic Jacksonville

Jacksonville, Florida, 32224, United States

RECRUITING

Lange Neurology

New York, New York, 10065, United States

RECRUITING

NEUROHK s.r.o.

Hradec Králové, 50002, Czechia

RECRUITING

Thomayer University Hospital - Fakultni Thomayerova nemocnice

Prague, 14059, Czechia

RECRUITING

Charité Centrum für Neurologie, Neurochirurgie und Psychiatrie

Berlin, 13353, Germany

RECRUITING

DIAKOVERE Henriettenstift - Klinik für Neurologie und Klinische Neurophysiologie

Hanover, 30171, Germany

RECRUITING

Universitaetsklinikum Schleswig-Holstein

Lübeck, 23538, Germany

RECRUITING

Michalski i Partnerzy Lekarze Spółka Partnerska

Krakow, 31-426, Poland

RECRUITING

SP ZOZ Szpital Uniwersytecki w Krakowie

Krakow, 51-503, Poland

RECRUITING

Centrum Medyczne NeuroProtect (NeuroProtect Medical Center)

Warsaw, 01-684, Poland

RECRUITING

City Clinic Research

Warsaw, 02-473, Poland

RECRUITING

University Clinical Center of Serbia

Belgrade, 11000, Serbia

RECRUITING

Hospital Universitario Vall D Hebron

Barcelona, 8035, Spain

RECRUITING

Hospital Universitario Ramon Y Cajal

Madrid, 28034, Spain

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Cromolyn Sodium; BID protein, human; Riluzole

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Chromones; Benzopyrans; Pyrans; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Thiazoles; Sulfur Compounds; Organic Chemicals; Benzothiazoles; Azoles

Central Study Contacts

David R Elmaleh, PhD

CONTACT

+1 (617) 784-0490; delmaleh@phenonet.us

Atul Gupta, M.D.

CONTACT

+91-9717287654; agupta@phenonet.us

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 19, 2025
• First Posted: August 26, 2025
• Study Start: November 25, 2025
• Primary Completion (Estimated): November 1, 2027
• Study Completion (Estimated): March 1, 2028
• Last Updated: April 1, 2026

Record last verified: 2026-03

Locations

CZ (2); PL (4); DE (3); SE (1); US (5); ES (2)