Foralumab Nasal for Multiple System Atrophy

Overseen By Brigham and Women's Hospital Movement Research Team

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Brigham and Women's Hospital

Must be taking: Dopaminergic treatment

Must not be taking: Systemic corticosteroids, Nasal corticosteroids

Disqualifiers: Pregnancy, Claustrophobia, Cognitive impairment, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Compensation: Varies

Number of Visits: Unknown

Duration: 6 months

5 Participants Needed

Trial Summary

What is the purpose of this trial?

A Phase 2a Study of Foralumab Nasal in Patients with Multiple System Atrophy (MSA)

Will I have to stop taking my current medications?

The trial requires that you have been on a stable dopaminergic treatment for at least 4 weeks before joining. You cannot take systemic corticosteroids or certain medications that affect heart rhythm. Other medications may be allowed if considered essential by the study doctor.

How is Foralumab Nasal different from other drugs for multiple system atrophy?

Foralumab Nasal is unique because it is administered nasally, which may offer a novel way to deliver treatment directly to the central nervous system, potentially reducing inflammation and targeting the disease more effectively than current options, which are limited and often ineffective. ^{1 2 3 4 5}

Eligibility Criteria

This trial is for adults aged 30-85 with Multiple System Atrophy (MSA) who meet specific health criteria like normal blood counts, liver function, and heart rhythm. They must not have significant cognitive impairment or recent serious infections and should be on stable MSA treatment. Pregnant women and those with certain medical conditions or treatments are excluded.

Inclusion Criteria

Adequate hematologic parameters without ongoing transfusion support: Hemoglobin (Hb) ≥ 9 g/dL; Platelets ≥ 100 x 10^9 cells/L

Total bilirubin ≤ 2 times the upper limit of normal (ULN) unless due to Gilbert's disease

Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 times ULN

Exclusion Criteria

Individuals with significant cognitive impairment (i.e., MoCA score less than or equal to 20)

Patients who test positive for human immunodeficiency virus (HIV), hepatitis B virus surface antigen (HBsAg), hepatitis C virus (HCV) or positive Epstein-Barr virus (EBV) IgM at the Screening Visit

History of gadolinium allergy

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Observational Lead-in

Participants are observed for baseline measurements before treatment

6 months

Treatment

Participants receive Foralumab Nasal in an open-label treatment phase

6 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Foralumab Nasal

Trial Overview The study tests Foralumab Nasal spray in patients with MSA to see if it's safe and effective. It's a Phase 2a trial, which means it focuses on the drug's effectiveness while monitoring side effects in people who have the condition.

Participant Groups

Group I: Foralumab Nasal Experimental Treatment 1 Intervention

This study includes a 6-month observational lead-in phase followed by a 6-month open-label treatment phase with Foralumab Nasal.

Who Is Running the Clinical Trial?

Brigham and Women's Hospital

Lead Sponsor

Trials

1,694

Recruited

14,790,000+

Findings from Research

Multiple system atrophy (MSA) is a severe neurodegenerative disorder with a mean survival of only 9 years, characterized by autonomic failure and either parkinsonism or cerebellar ataxia, with limited treatment options available.

While pharmacological treatments for motor symptoms are largely ineffective, early identification and treatment of autonomic and urogenital symptoms can be beneficial, and ongoing multicenter trials are exploring potential neuroprotective therapies like riluzole and human recombinant growth hormone.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Multiple system atrophy: an update. Wenning, GK., Geser, F., Stampfer-Kountchev, M., et al.[2013]

In a study involving PLP-α-syn transgenic mice, treatment with ATH434 for 6 months preserved motor performance and protected against neuron loss, indicating its potential as a disease-modifying therapy for multiple system atrophy (MSA).

ATH434 treatment led to a significant reduction in α-synuclein aggregates and oligomers, increased microglial lysosomal activity, and lower iron levels in the substantia nigra, suggesting a mechanism of action that enhances α-syn clearance without increasing inflammation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

ATH434 Reduces α-Synuclein-Related Neurodegeneration in a Murine Model of Multiple System Atrophy. Heras-Garvin, A., Refolo, V., Schmidt, C., et al.[2022]

References

1. United States pubmed.ncbi.nlm.nih.gov

Multiple system atrophy: an update. [2013]

2. England pubmed.ncbi.nlm.nih.gov

Combination of alpha-synuclein immunotherapy with anti-inflammatory treatment in a transgenic mouse model of multiple system atrophy. [2018]

3. England pubmed.ncbi.nlm.nih.gov

Multiple system atrophy in Hokkaido, Japan: a prospective registry study of natural history and symptom assessment scales followed for 5 years. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

ATH434 Reduces α-Synuclein-Related Neurodegeneration in a Murine Model of Multiple System Atrophy. [2022]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Present and future of disease-modifying therapies in multiple system atrophy. [2021]