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New treatment option for Amyotrophic Lateral Sclerosis (ALS)
Official title PHENOGENE-1A (Cromolyn) Treatment in Patients With Mild to Moderate ALS
ClinicalTrials.gov ID: NCT07142291
What this study is testing
What is Cromolyn Sodium (34.2 mg BID)?
Cromolyn Sodium (34.2 mg BID) is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for amyotrophic lateral sclerosis (als).
Also referred to as Cromolyn, Sodium Cromoglycate.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 75
You may be able to join 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...
- Absence of electrophysiological, neuroimaging, or pathological evidence of other diseases that might explain the UMN or LMN degeneration and...
You likely can't join 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...
- Any other significant neurological disorder which can interfere with study assessments, e.g., significant cognitive impairment and/or clinical...
- Significant psychiatric illness like schizophrenia, bipolar disorder etc. people with depression can be included, only if the depression has been...
- Severe cardiac disease (e.g., QTc\>500 ms), Torsade de Pointes, evidence of significant heart failure (New York Heart Association [NYHA] Class 3 or...
See the full eligibility criteria
- 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.
- Absence of electrophysiological, neuroimaging, or pathological evidence of other diseases that might explain the UMN or LMN degeneration and exclusion of other causes.
- Male or female people 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 people 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...
- Combined (estrogen and progestogen containing) or progestogen-only hormonal contraceptives;
- Intrauterine device or intrauterine hormone-releasing system; OR
- Post-menopausal status must have experienced their last menstrual period minimum of 1 year prior to study drug administration; OR
- 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.
- Male people 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...
- 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...
- 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. people 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...
- 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).
- people with a body weight of 32 kg or less, or a body mass index of \ 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.; people with hepatic diseases such as hepatic...
- 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...
- Pregnant or breast-feeding females.
The study team makes the final eligibility decision.
Where it's taking place
- Scottsdale, Arizona, United States
- La Jolla, California, United States
- San Francisco, California, United States
- Jacksonville, Florida, United States
- New York, New York, United States
- Hradec Králové, Czechia
- Prague, Czechia
- Berlin, Germany
- Hanover, Germany
- Lübeck, Germany
- Krakow, Poland
- Warsaw, Poland
- Belgrade, Serbia
- Alicante, Spain
- Barcelona, Spain
- Madrid, Spain
- Málaga, Spain
- Seville, Spain
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Scottsdale, Arizona, United States; La Jolla, California, United States; San Francisco, California, United States; Jacksonville, Florida, United States; New York, New York, United States; Hradec Králové, Czechia and 12 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.