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PHENOGENE-1A (Cromolyn) Treatment in Patients With Mild to Moderate ALS
NCT07142291 · PhenoNet, Inc.
In plain English
Click the button to translate this study into plain language — what it is, who qualifies, and what participation looks like.
Official title
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)
About this study
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
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.
Study design
Enrollment target: 105 participants
Allocation: randomized
Masking: quadruple
Age groups: adult, older_adult
Timeline
Starts: 2025-11-25
Estimated completion: 2028-03
Last updated: 2026-04-01
Interventions
Drug: Cromolyn Sodium (34.2 mg BID)Drug: Cromolyn Sodium (17.1 mg BID)Drug: PlaceboDrug: Riluzole (100 mg)
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)
Sponsor
PhenoNet, Inc. · industry
Contacts & investigators
ContactDavid R Elmaleh, PhD · contact · delmaleh@phenonet.us · +1 (617) 784-0490
ContactAtul Gupta, M.D. · contact · agupta@phenonet.us · +91-9717287654
All locations (17)
Honor Health Neurology - Bob Bove Neuroscience InstituteRecruiting
Scottsdale, Arizona, United States
University of California San DiegoRecruiting
La Jolla, California, United States
Sutter Health - California Pacific Medical Center Research InstituteRecruiting
San Francisco, California, United States
Mayo Clinic JacksonvilleRecruiting
Jacksonville, Florida, United States
Lange NeurologyRecruiting
New York, New York, United States
NEUROHK s.r.o.Recruiting
Hradec Králové, Czechia
Thomayer University Hospital - Fakultni Thomayerova nemocniceRecruiting
Prague, Czechia
Charité Centrum für Neurologie, Neurochirurgie und PsychiatrieRecruiting
Berlin, Germany
DIAKOVERE Henriettenstift - Klinik für Neurologie und Klinische NeurophysiologieRecruiting
Hanover, Germany
Universitaetsklinikum Schleswig-HolsteinRecruiting
Lübeck, Germany
Michalski i Partnerzy Lekarze Spółka PartnerskaRecruiting
Krakow, Poland
SP ZOZ Szpital Uniwersytecki w KrakowieRecruiting
Krakow, Poland
Centrum Medyczne NeuroProtect (NeuroProtect Medical Center)Recruiting
Warsaw, Poland
City Clinic ResearchRecruiting
Warsaw, Poland
University Clinical Center of SerbiaRecruiting
Belgrade, Serbia
Hospital Universitario Vall D HebronRecruiting
Barcelona, Spain
Hospital Universitario Ramon Y CajalRecruiting
Madrid, Spain