PHENOGENE-1A (Cromolyn) Treatment in Patients With Mild to Moderate ALS
NCT ID: NCT07142291
Last Updated: 2025-11-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE2
105 participants
INTERVENTIONAL
2025-11-30
2028-03-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Keywords
Explore important study keywords that can help with search, categorization, and topic discovery.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Low Dose PHENOGENE-1A (17.1 mg BID)
17.1 mg, BID, oral inhalation via dry powder inhaler
Cromolyn Sodium (17.1 mg BID)
34.2 mg, BID, oral inhalation via dry powder inhaler
Riluzole (100 mg)
50 mg, oral tablet, BID, standard of care treatment
High Dose PHENOGENE-1A (34.2 mg BID)
34.2 mg, BID, oral inhalation via dry powder inhaler
Cromolyn Sodium (34.2 mg BID)
17.1 mg, BID, oral inhalation
Riluzole (100 mg)
50 mg, oral tablet, BID, standard of care treatment
Placebo
Placebo comparator matched to active treatment, BID, oral inhalation via dry powder inhaler
Placebo
Placebo comparator matched to active treatment.
Riluzole (100 mg)
50 mg, oral tablet, BID, standard of care treatment
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Cromolyn Sodium (34.2 mg BID)
17.1 mg, BID, oral inhalation
Cromolyn Sodium (17.1 mg BID)
34.2 mg, BID, oral inhalation via dry powder inhaler
Placebo
Placebo comparator matched to active treatment.
Riluzole (100 mg)
50 mg, oral tablet, BID, standard of care treatment
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
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.
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
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.
18 Years
75 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
PhenoNet, Inc.
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Sutter Health - California Pacific Medical Center Research Institute
San Francisco, California, United States
Lange Neurology
New York, New York, United States
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
PHENOALS-001
Identifier Type: -
Identifier Source: org_study_id