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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new nasal spray treatment called Foralumab to determine its effectiveness for people with Multiple System Atrophy (MSA), a condition affecting the nervous system and causing movement and balance issues. The goal is to assess whether Foralumab can improve symptoms and quality of life for those with MSA. Participants must have a confirmed MSA diagnosis and have been on stable medication for at least four weeks before joining. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

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.

Is there any evidence suggesting that Foralumab Nasal is likely to be safe for humans?

Research has shown that Foralumab Nasal has been well-tolerated in earlier studies. One study with healthy volunteers found that taking Foralumab orally at doses up to 5 mg did not cause any drug-related safety issues, indicating likely safety. The treatment also demonstrated promising safety results in studies for multiple system atrophy (MSA), with no major safety concerns reported. Overall, the evidence suggests that Foralumab Nasal is generally safe for humans at the doses tested. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Multiple System Atrophy (MSA), which mainly focus on alleviating symptoms, Foralumab Nasal offers a novel approach by targeting inflammation directly in the central nervous system. Researchers are excited about Foralumab because it is administered nasally, allowing it to reach the brain more effectively and potentially reduce neuroinflammation. This unique delivery method could provide a more direct and efficient way to address the underlying causes of the disease, offering new hope for patients with MSA.

What evidence suggests that Foralumab Nasal might be an effective treatment for Multiple System Atrophy?

Research shows that Foralumab Nasal, the treatment under study in this trial, might help manage Multiple System Atrophy (MSA) by affecting the immune system. Early findings suggest it can reduce inflammation, which is believed to contribute to MSA's progression. Although final results on its effectiveness in people are pending, initial studies indicate that the treatment is safe and well-tolerated. The goal is for Foralumab Nasal to slow the worsening of symptoms by addressing the root causes of the disease. ^{1 3 6 7 8}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Tiziana Life Sciences LTD

Lead Sponsor

Trials

13

Recruited

310+

Published Research Related to This Trial

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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06868628?cond=Multiple%20system%20atrophy%20OR%20%22MSA%22%20OR%20%22%20multisystem%20atrophy%22%20OR%20%22%20Shy-Drager%20syndrome%22&aggFilters=status:not%20rec&viewType=Table&rank=8

NCT06868628 | A Phase 2a Study of Foralumab Nasal in ... Subjects with a clinical diagnosis of Clinically Established or Clinically Probable Multiple System Atrophy in accordance with 2022 MDS diagnostic criteria.

2. tizianalifesciences.com tizianalifesciences.com/fda-approves-tiziana-life-sciences-phase-2-ind-for-multiple-system-atrophy/

FDA Approves Tiziana Life Sciences Phase 2 IND for ... Disease prognosis is poor with a median survival of 6-9 years. “We are pleased that the FDA have approved the IND to treat MSA patients with ...

3. connect.careboxhealth.com connect.careboxhealth.com/en-US/trial/listing/589315

A Phase 2a Study of Foralumab Nasal in Patients With ... Primary outcome measures · Change from Screening to Month 6 in the total MDS-UMSARS score · Change in TSPO activity as measured by [F-18]PBR06 retention ...

4. clinicaltrialvanguard.com clinicaltrialvanguard.com/news/tiziana-doses-first-patient-in-trial-of-intranasal-foralumab-for-msa/

Tiziana Doses First Patient in Trial of Intranasal Foralumab ... While offering initial insights into foralumab's activity and tolerability in MSA, it will not provide definitive efficacy data.

5. tizianalifesciences.com tizianalifesciences.com/wp-content/uploads/2025/09/Tiziana-Neuro-Presentation-Sept-2025-Non-Confidential.pdf

Foralumab MSA is a rapidly progressive neurological disorder. It is caused by degeneration or atrophy (shrinking) of nerve cells in several (or multiple) areas of the ...

6. tizianalifesciences.com tizianalifesciences.com/drug-pipeline/intranasal-foralumab/

Intranasal Foralumab A Phase 1 trial in healthy volunteers demonstrated that oral Foralumab was well-tolerated up to a 5 mg dose, with no drug-related safety issues. ​; Nasal ...

7. clinicaltrialsarena.com clinicaltrialsarena.com/news/tiziana-multiple-system-atrophy-study/

Tiziana Life's multiple system atrophy study gets FDA ... The therapy has so far shown a promising safety profile and has elicited positive clinical outcomes in patients in trials. Tiziana Life Sciences ...

8. neurologylive.com neurologylive.com/view/phase-2a-inform-ms-trial-study-anti-cd3-agent-foralumab-non-active-secondary-progressive-ms

Phase 2a INFORM-MS Trial to Study Anti-CD3 Agent ... Intranasal foralumab remains in development as a treatment for multiple system atrophy (MSA), with an ongoing phase 2a trial (NCT06868628) ...