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

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What is this study about?

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.

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 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 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 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 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 Study?

  • 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 Study?

  • 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

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
City Clinic Research Sp. z o.o. Warsaw Poland

Other Sites

Site Name City Country Status
Hospital Del Mar Barcelona Spain
Virgen del Rocío University Hospital Sevilla Spain
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Fakultni Nemocnice Hradec Kralove Novy Hradec Kralove Czechia
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie Cracow Poland
Fakultni Thomayerova nemocnice Prague Czechia
Neuroprotect Sp. z o.o. Warsaw Poland
Universitätsklinikum Schleswig Holstein Luebeck Germany
Dmwltxijb Hwyvpquctwcpgne Hanover Germany
Hrhwnkwr Vaxp dkkgeukp Barcelona Spain
Mhfbgpxfm i Pcgaghmwd Lvtbuno Scvyjq Psosqrsakiw Cracow Poland

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Czechia Czechia
Recruiting
01.11.2025
Germany Germany
Recruiting
01.11.2025
Poland Poland
Recruiting
01.11.2025
Spain Spain
Recruiting
01.11.2025

Trial locations

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.

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 ID:
2025-520688-42-00
Protocol code:
PHENOALS-001
Trial Phase:
Therapeutic exploratory (Phase II)

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