Cromolyn Sodium for ALS

Recruiting at 2 trial locations

Overseen By Atul Gupta, M.D.

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: PhenoNet, Inc.

Must be taking: Riluzole

Must not be taking: Inhaled proteins

Disqualifiers: Bulbar ALS, Ventilation use, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

105 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called Cromolyn Sodium to determine its effectiveness for people with ALS, a disease affecting nerve cells in the brain and spinal cord. Researchers will assess the treatment's impact on movement, breathing, and specific blood markers. Participants will receive one of three options: a placebo, a low dose, or a high dose of the treatment. Individuals with ALS symptoms like muscle weakness for no more than two years and stable breathing and mouth function are suitable candidates for this trial. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial requires participants to be on a stable dose of Riluzole for at least 4 weeks before joining. The protocol does not specify if you need to stop other medications, so it's best to discuss this with the study team.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Earlier studies have tested Cromolyn Sodium in people with ALS, a disease affecting nerve cells in the brain and spinal cord. Research shows that this treatment is generally well-tolerated. Some studies explored different doses of Cromolyn Sodium taken through a dry powder inhaler and found no serious side effects. Most participants experienced only mild reactions, such as a sore throat or cough.

Since Cromolyn Sodium is already used for other conditions and has been studied in ALS, it appears relatively safe. However, as with any treatment, responses can vary. It is always important to consult a doctor about any concerns before joining a trial. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Most treatments for ALS focus on managing symptoms and slowing disease progression, often through medications like riluzole and edaravone. However, Cromolyn Sodium presents a novel approach by targeting inflammation, which is believed to play a role in ALS progression. This drug is administered via oral inhalation, potentially offering a more direct effect on respiratory symptoms. Researchers are eager to see if this unique method might improve patient outcomes compared to current treatments.

What evidence suggests that PHENOGENE-1A might be an effective treatment for ALS?

Research has shown that cromolyn sodium might help treat ALS, a disease affecting nerve cells in the brain and spinal cord. Animal studies have found that it can delay the onset of symptoms related to nerve damage, suggesting potential benefits for people with ALS. It seems to protect nerve cells from harm. In other tests, cromolyn sodium improved performance on tasks measuring nerve function. Although more research is needed to confirm its effects in humans, these early results are promising. Participants in this trial will receive either a placebo or one of two doses of PHENOGENE-1A, which contains cromolyn sodium, to further investigate its potential benefits for ALS. ^{1 5 6 7 8}

Are You a Good Fit for This Trial?

This trial is for adults aged 18-75 with ALS, showing both upper and lower motor neuron degeneration, a disease duration ≤24 months, and an ALSFRS-R score ≥38. Participants must be able to perform study procedures and give informed consent. Those with other diseases explaining their symptoms or unable to complete the study are excluded.

Inclusion Criteria

I am willing to sign a consent form for study procedures.

My symptoms or signs of disease are getting worse over time.

My tests show no other diseases causing my nerve damage.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive PHENOGENE-1A or placebo for 24 weeks, with doses administered twice daily via dry powder inhaler

24 weeks

Regular visits for assessments and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Cromolyn Sodium

Trial Overview The PHENOGENE-1A study tests Cromolyn Sodium at two doses (34.2 mg BID and 17.1 mg BID) against a placebo, alongside standard treatment Riluzole (100 mg). It measures ALS progression using functional scales, lung function tests, and blood biomarkers for neuro-inflammation.

How Is the Trial Designed?

Group I: Low Dose PHENOGENE-1A (17.1 mg BID) Experimental Treatment 2 Interventions

17.1 mg, BID, oral inhalation via dry powder inhaler

Group II: High Dose PHENOGENE-1A (34.2 mg BID) Experimental Treatment 2 Interventions

34.2 mg, BID, oral inhalation via dry powder inhaler

Group III: Placebo Placebo Group 2 Interventions

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

Who Is Running the Clinical Trial?

PhenoNet, Inc.

Lead Sponsor

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07142291

PHENOGENE-1A (Cromolyn) Treatment in Patients With ... The purpose of this study is to test the effects of PHENOGENE-1A, which is the treatment under investigation in this study.

2. nature.com nature.com/articles/s41598-019-53982-w

Cromolyn sodium delays disease onset and is ... Our results indicate that cromolyn sodium treatment significantly delayed the onset of neurological symptoms, and improved deficits in PaGE performance.

3. medrxiv.org medrxiv.org/content/10.1101/2025.05.25.25328303v1.full.pdf

Oral Inhaled Cromolyn for Mild-to-Moderate Amyotrophic ... This study aims to evaluate the safety and efficacy of two doses of oral inhaled cromolyn in mild to moderate ALS over 12 weeks. The primary ...

4. sciencedirect.com sciencedirect.com/science/article/abs/pii/S014929182030045X

Amyotrophic Lateral Sclerosis, Neuroinflammation, and ... E.J. Granucci et al. Cromolyn sodium delays disease onset and is neuroprotective in the SOD1(G93A) Mouse Model of amyotrophic lateral sclerosis. Sci Rep.

5. massgeneral.org massgeneral.org/news/press-release/cromolyn-sodium-delays-disease-onset-and-is-neuroprotective-in-the-sod1g93a-mouse-model-of-als

Cromolyn sodium delays disease onset and is ... “Our findings demonstrate that cromolyn treatment provides neuroprotection in a mouse model of ALS. It remains to be seen whether these effects ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04428775

A Safety and Biomarker Study of ALZT-OP1a in Subjects ... Each dose of ALZT-OP1a (cromolyn) will be co-administered with a stable dose of ALS standard-of-care treatment as prescribed by their physician. Masking : None ...

7. clinicaltrials.eu clinicaltrials.eu/trial/study-of-cromolyn-inhalation-treatment-for-patients-with-mild-to-moderate-amyotrophic-lateral-sclerosis-als/

Study of Cromolyn Inhalation Treatment for Patients with ... Cromolyn (also known as PHENOGENE-1a) is a medication being studied as an additional treatment for people with ALS (Amyotrophic Lateral ...

8. ctv.veeva.com ctv.veeva.com/study/phase-iib-study-of-phenogene-1a-cromolyn-as-an-adjuvant-therapy-in-mild-to-moderate-als

PHENOGENE-1A (Cromolyn) Treatment in Patients With Mild ... The purpose of this study is to test the effects of PHENOGENE-1A, which is the treatment under investigation in this study.