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

NCT ID: NCT06868628

Last Updated: 2025-09-23

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 5 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-05-19
• Study Completion Date: 2027-04-01

Brief Summary

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

Conditions

Multiple System Atrophy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Foralumab Nasal

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

Group Type: EXPERIMENTAL

Foralumab Nasal

Intervention Type: DRUG

Foralumab 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.

Interventions

Foralumab Nasal

Foralumab 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.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Subjects with a clinical diagnosis of Clinically Established or Clinically Probable Multiple System Atrophy in accordance with 2022 MDS diagnostic criteria.
• Age 30 to 85 years, at the time of signing the informed consent.
• Stable dopaminergic treatment for at least 4 weeks before enrollment.
• Adequate hematologic parameters without ongoing transfusion support: Hemoglobin (Hb) ≥ 9 g/dL; Platelets ≥ 100 x 109 cells/L.
• Creatinine ≤ 1.5 x the upper limit of normal (ULN), or calculated creatinine clearance ≥ 60 mL/minute x 1.73 m2 per the Cockcroft-Gault formula.
• 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.
• QT interval corrected for rate (QTcF) ≤ 470 msec for women and ≤ 450 msec for men on the ECG obtained at Screening.
• Negative urine pregnancy test within 7 days prior to the first dose of study therapy for women of child-bearing potential (WCBP), defined as a sexually mature woman who has not undergone a hysterectomy or who has not been naturally postmenopausal for at least 24 consecutive months (i.e., who has had menses any time in the preceding 24 consecutive months). Sexually active WCBP and male patients must agree to use highly effective methods to avoid pregnancy (oral, injectable, or implantable hormonal contraceptive; tubal ligation; intra-uterine device; barrier contraceptive with spermicide; or vasectomized partner) throughout the study and for 90 days after the completion of study treatment.
• Patients whose immunizations are fully up to date at the Screening, according to the assessment of their primary care physician and neurologist.
• Ability to provide written informed consent.

Exclusion Criteria

• Diagnosis or suspicion of other cause for Parkinsonism or a known alternate neurologic diagnosis.
• Female patient who is pregnant, lactating, breastfeeding, or planning to become pregnant during study.
• Individuals with claustrophobia who cannot tolerate the study procedures
• Non-MRI-compatible implanted devices.
• Low-affinity binders for translocator protein (TSPO) PET ligands.
• Systemic corticosteroid treatment in the past four weeks (excluding nasal or local treatment).
• Individuals with significant cognitive impairment (i.e., MoCA score less than or equal to 20).
• Brain MRI indicative of significant abnormalities that interfere with PET-MRI co-registration (i.e., large prior hemorrhage or multiple infarcts).
• Serious cardiac condition within the last 6 months, such as uncontrolled arrhythmia, myocardial infarction, unstable angina, or heart disease defined by the New York Heart Association (NYHA) Class III or Class IV or hereditary long QT syndrome.
• Concomitant medication(s) that may cause QTc prolongation or induce Torsades de Pointes, except for antimicrobials that are used as standard of care to prevent or treat infections and other such drugs that are considered by the Investigator to be essential for patient care.
• 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.
• Past medical history of a hematologic or solid malignancy.
• Treatment with chronic immunosuppressives such as interferon, glatiramer acetate, fingolimod, Siponimod, dimethyl fumarate, or natalizumab within the past 90 days.
• Inability to tolerate nasally administered medications.
• Nasal corticosteroids, nasal antihistamines, nasal flu dosing within the past 30 days, or anticipated need during the study.
• Chronic rhinitis, deviated septum, nasal polyps, history of sinusitis treated within the past 8 months.
• Inflammatory bowel disease, rheumatoid arthritis, systemic lupus erythematosus, primary Sjögren's Syndrome, asthma, or type 1 diabetes.
• Neutropenia or an absolute neutrophil count of < 1,000 cells/mL or other indicators of severe immunosuppression.
• Severe lymphopenia or an absolute lymphocyte count of < 500 cells/mL
• Patients with a history of gadolinium allergy.
• A recent clinically significant active infection requiring treatment with antibiotics or other anti-infective agents within the past 15 days.
• Any other medical or surgical intervention or other condition which, in the opinion of the Principal Investigator, could compromise adherence to study requirements or confound the interpretation of study results.
• Unable or unwilling to comply with protocol requirements.
• Active COVID-19 disease.
• COVID-19 vaccine within past 10 days or any other vaccine within past 7 days (at dosing).

• Minimum Eligible Age: 30 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Tiziana Life Sciences LTD

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Brigham and Women's Hospital Movement Research Team

Role: CONTACT

BWHMovementResearch@bwh.harvard.edu

507-491-0272

Facility Contacts

Diego Rodriguez, MD

Role: primary

drodriguez29@bwh.harvard.edu

507-491-0272

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Other Identifiers

2025P000259

Identifier Type: -

Identifier Source: org_study_id