Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruitingRare disease

Study of Cromolyn Inhalation Treatment for Patients with Mild to Moderate Amyotrophic Lateral Sclerosis (ALS)

Verified siteInvestigational
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on people with Amyotrophic Lateral Sclerosis (ALS), a condition that affects nerve cells controlling muscle movement, leading to progressive weakness. The research examines a medication called PHENOGENE-1a (also known as cromolyn) used together with standard treatments for mild to moderate ALS. The purpose is to evaluate how this inhaled medication affects changes in physical function over 24 weeks.

The treatment involves using inhalation powder contained in hard capsules that are breathed in through a special inhaler device. Some participants will receive PHENOGENE-1a, while others will receive placebo through the same type of inhaler. The maximum daily amount of medication is 64.8 mg, and treatment continues for 24 weeks.

During the study, participants will need to continue taking their regular ALS medication called riluzole. The study will monitor various aspects of the disease, including breathing function and overall physical abilities. All participants must be between 18 and 75 years old and have been diagnosed with ALS within the past 24 months.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    You will begin participation in a 24-week study testing PHENOGENE-1a, an inhaled medication for ALS (Amyotrophic Lateral Sclerosis)

    Your breathing capacity will be measured to confirm it is above 70% of normal values

    Your ALSFRS-R score (a measure of ALS symptoms) must be 38 or higher

  2. Step 2

    Medication assignment

    You will be randomly assigned to receive either PHENOGENE-1a or a placebo through inhalation

    The medication comes in powder form in hard capsules for inhalation

    You must continue taking your regular Riluzole medication throughout the study

  3. Step 3

    Treatment period

    The treatment period lasts for 24 weeks

    You will use an inhaler device to take the study medication

    Your breathing strength must show a Peak Inspiratory Flow Rate (PIFR) of at least 100 liters per minute

  4. Step 4

    Regular assessments

    Your ALS symptoms will be monitored using the ALSFRS-R scale

    Your breathing function will be regularly tested

    The study will track changes in your condition over the 24-week period

  5. Step 5

    Safety monitoring

    Your breathing capacity will be monitored throughout the study

    Your overall health status will be regularly assessed

    Any changes in your condition will be documented and monitored

Who can join the trial?

14 criteria

  • Age between 18 to 75 years old
  • Must have a confirmed diagnosis of ALS (Amyotrophic Lateral Sclerosis) according to specific medical criteria
  • Disease symptoms must have started no more than 24 months ago
  • Must have a PIFR (Peak Inspiratory Flow Rate) of at least 100 liters per minute
  • Must be taking Riluzole (a standard ALS medication) at a stable dose for at least 4 weeks before joining the study
  • Must have an ALSFRS-R total score (a scale measuring ALS symptoms) of 38 or higher when screened
  • Must have a breathing score of 9 or higher on the ALSFRS-R scale
  • Must have a bulbar score (measuring speech and swallowing) of 9 or higher on the ALSFRS-R scale
  • Must have a FVC (Forced Vital Capacity - a breathing test) greater than 70% of expected value
  • Must be able to complete all study procedures and visits
  • Must provide written consent to participate in the study
  • Women who can become pregnant must use effective birth control during the study and for 3 months after
  • Men with partners who can become pregnant must use effective birth control during the study and for 3 months after
  • Must not donate eggs (women) or sperm (men) during the study and for 3 months after

Who cannot join the trial?

15 criteria

  • Age below 18 or above 80 years
  • Pregnant or breastfeeding women
  • History of severe allergic reactions to medications
  • Participation in other clinical trials within the past 30 days
  • Significant liver or kidney disease (conditions affecting how your body processes medications)
  • Current use of medications that could interact with the study drug
  • Inability to follow study procedures or attend scheduled visits
  • Severe respiratory problems requiring permanent ventilation support
  • Diagnosis of ALS (Amyotrophic Lateral Sclerosis) for more than 5 years
  • Mental conditions that could interfere with providing informed consent
  • History of drug or alcohol abuse within the past year
  • Severe heart conditions or uncontrolled high blood pressure
  • Active infections or major health conditions that could affect study results
  • Previous adverse reactions to drugs similar to cromolyn
  • Unable to take oral medications
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

Cromolyn (also known as PHENOGENE-1a) is a medication being studied as an additional treatment for people with ALS (Amyotrophic Lateral Sclerosis). This medication is being tested to see if it can help slow down the progression of symptoms in patients with mild to moderate ALS. Cromolyn has been previously used in other medical conditions, and researchers are now investigating its potential benefits when used alongside other ALS treatments to improve patients' daily functioning.

What is already known about the treatment

Cromolyn – An anti-inflammatory medication administered through inhalation or nasal spray that is being investigated as a potential treatment for Amyotrophic Lateral Sclerosis (ALS) under the name PHENOGENE-1a. This drug belongs to the class of mast cell stabilizers and works by preventing the release of inflammatory chemicals in the body's cells. Currently undergoing Phase IIb clinical trials to evaluate its effectiveness as an adjuvant therapy for mild to moderate ALS patients, with researchers specifically monitoring its impact on functional changes using the ALSFRS-R scoring system over a 24-week treatment period. While traditionally used for asthma and allergic conditions, its potential neuroprotective properties are being explored in neurodegenerative diseases.

Investigated diseases

Amyotrophic Lateral Sclerosis (ALS) - A progressive neurological disease that affects nerve cells responsible for controlling voluntary muscle movement. The condition causes muscle weakness that starts in specific areas, such as hands, arms, or legs, and gradually spreads to other parts of the body. As motor neurons degenerate, they stop sending messages to the muscles, which leads to muscle weakening, twitching, and inability to move the arms, legs, and body. The disease affects the muscles needed for speaking, swallowing, and breathing. Over time, people with ALS experience increasing difficulty with daily activities requiring muscle control.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2025-520688-42-00Protocol codePHENOALS-001Estimated enrolment160 patientsSponsorPhenonet Inc.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.