PHENOGENE-1A (Cromolyn) Treatment in Patients With Mild to Moderate ALS
ConditionAmyotrophic Lateral Sclerosis (ALS)
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-75
SponsorPhenoNet, Inc.
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Parallel
Treatments tested in this trial
Cromolyn Sodium (34.2 mg BID)
Drug17.1 mg, BID, oral inhalation
Cromolyn Sodium (17.1 mg BID)
Drug34.2 mg, BID, oral inhalation via dry powder inhaler
Placebo
DrugPlacebo comparator matched to active treatment.
Riluzole (100 mg)
Drug50 mg, oral tablet, BID, standard of care treatment
Treatment groups
105 Participants
are divided into 3 treatment groupsGroup A: Low Dose PHENOGENE-1A (17.1 mg BID)Experimental treatment 2 interventions
Group B: High Dose PHENOGENE-1A (34.2 mg BID)Experimental treatment 2 interventions
Group C: PlaceboPlacebo comparator 2 interventions
Trial outcomes
Primary outcomes
1
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).
Time frame
Baseline to Week 242
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.
Time frame
Baseline to Week 24Secondary outcomes
1
Mean rank for CAFS at Week 24
To evaluate the effects of PHENOGENE-1A on function and survival using the Combined Assessment of Function and Survival (CAFS).
Time frame
Baseline to Week 242
Time to event requiring full-time or nearly full-time respiratory support
Time from randomization to the first of 7 consecutive days on which permanent assisted ventilation (invasive or non-invasive) was used for \>22 hours/day due to ALS progression.
Time frame
Randomization to Week 243
Mean change in percent predicted forced vital capacity (%FVC) from baseline to Week 24
To evaluate the effect of PHENOGENE-1A on respiratory changes.
Time frame
Baseline to Week 244
Mean change in peak inspiratory flow rate (PIFR) from baseline to Week 24
To evaluate changes in PIFR (L/min) during treatment.
Time frame
Baseline to Week 24Other outcomes
1
Change in plasma neuroinflammatory biomarkers from baseline to Week 24
To evaluate the impact of two different doses of PHENOGENE-1A on neuroinflammation.
Time frame
Baseline (pre-dose) to Week 24Sponsors and contacts
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Source ClinicalTrials.gov