The goal of this clinical trial is to learn whether a nasal spray medicine called foralumab is safe and easy to tolerate in adults with amyotrophic lateral sclerosis (ALS). ALS is a disease that slowly damages the nerve cells that control movement, causing muscles to grow weaker over time. Foralumab is being studied as a possible way to calm the inflammation and immune system activity that are thought to play a role in ALS.
The main questions it aims to answer are:
1. Is nasal foralumab safe for people with ALS, and how well do they tolerate it over 12 and 24 weeks of treatment? 2. Does foralumab change certain markers in the blood and spinal fluid, and lower inflammation in the brain, compared with a placebo?
Researchers will compare people who take foralumab to people who take a placebo (a matching nasal spray with no medicine in it) to see how the two groups differ in safety and in these markers.
About 44 adults aged 18 to 75 with ALS will take part. For the first 12 weeks, participants will be randomly assigned so that about 3 in 4 use foralumab and about 1 in 4 use the placebo. Neither the participants nor the study staff will know who is using which during this time (this is called "double-blind"). After the first 12 weeks, everyone who continues will use foralumab for the next 12 weeks.
Participants will:
* Spray the study treatment into both nostrils 3 times a week, on alternate days, in cycles of 2 weeks on treatment followed by 1 week off * Learn to use the nasal spray device so they or a caregiver can give most doses at home * Take part for about 8.5 months in total, which includes screening, 24 weeks of treatment, and a safety check-in about 4 weeks after the last dose * Attend some visits at the study clinic and complete others remotely by phone or video * Have study tests that may include blood samples, breathing and muscle-strength checks, and - for those who agree and at certain study centers - spinal fluid samples and brain scans to measure inflammation
Adults ≥18 years of age and ≤75 years of age with a diagnosis of symptomatic ALS by revised El Escorial Criteria (clinically possible, probable, probable lab-supported, or definite).
Participant must be actively enrolled and must have met eligibility criteria for the Healey ALS MyMatch Common Screening Protocol (MCSP) AND determined to have met preliminary eligibility on targeted medical history and review of systems and safety laboratory results required for this trial.
Blood neurofilament (NfL) level of greater than 40 pg/mL as measured by the MyMatch Common Screening Protocol specific NfL assay.
Ability to provide written informed consent.
Clinically significant medical condition that would pose a risk to the participant, according to the PI's judgment and examination prior to baseline.
Active ENT disease (such as allergic rhinitis, chronic rhinitis, active sinusitis treated within the past 30 days or deemed as clinically relevant, clinically significant deviated nasal septum, nasal polyps) that might interfere with safely administering the drug nasally.
Active clinically significant infection or immunocompromised condition.
Treatment with chronic immunosuppressants or other immunosuppressants within the past 90 days.
Parallel
Foralumab Nasal
DrugForalumab is an anti-CD3 monoclonal antibody administered as a nasal spray. Participants will complete eight 3-week dosing cycles over the study. During each cycle, Foralumab will be administered intranasally three times per week for the first two weeks, with no administration in the third week.
Placebo
DrugForalumab placebo nasal solution is a preservative-free, sterile, clear, colorless-to-slightly-yellow solution filled in an Aptar Unidose nasal atomizer device. Participants will complete eight 3-week dosing cycles over the study. During each cycle, Foralumab placebo nasal solution will be administered intranasally three times per week for the first two weeks, with no administration in the third week.
Safety - Occurrence of serious and non-serious treatment emergent adverse events and clinically significant treatment emergent abnormalities.
The occurrence of serious (inclusive of SUSARs) and non-serious treatment emergent adverse events (TEAEs) and clinically significant treatment-emergent abnormalities in clinical, including vital signs (heart rate, blood pressure, weight) and laboratory values (reported as AEs) over both 12 (4 cycles) and 24 (8 cycles) week treatment periods in the Safety population.
Tolerability - Percentage of participant that complete 12 or 24 weeks of study treatment.
the percentage of nasal foralumab treated participants in the Safety population who complete four (12-weeks) or eight (24-weeks) cycles of study treatment without study drug attributed intolerable AEs or SAEs or AESIs that lead to early permanent drug discontinuation.
Immune Reconstitution Changes: change in blood Tregs and functional T cell markers on flow cytometry
Evaluate immune reconstitution changes from baseline following 4 cycles of foralumab 50 μg dosage treatment compared to placebo, as measured by blood Treg number and functional T cell markers including CD4+ T cell subset profiles on flow cytometry
Immunological gene expression changes: single cell RNA sequencing
Evaluate immune reconstitution changes from baseline following 4 cycles of foralumab 50 μg dosage treatment compared to placebo, as measured by blood Treg number and functional T cell markers including CD4+ T cell subset profiles on flow cytometry
Neurofilament light changes
evaluate plasma and CSF neurofilament light (pNfL) changes from baseline following 4 cycles of foralumab 50 μg dosage treatment compared to placebo.
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