Recruiting
ALSFUS003

Intravenous Immunoglobulin with Focused Ultrasound for ALS

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What is being tested

Next Generation Dome Helmet Focused Ultrasound

+ Intravenous immunoglobulin (IVIG), 10% solution for infusion
+ Definity® Vial for (Perflutren Lipid Microsphere) Injectable Suspension
Device
Drug
Who is being recruted

Amyotrophic Lateral Sclerosis
+8

+ Central Nervous System Diseases
+ Metabolic Diseases
Over 18 Years
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 1
Interventional
Study Start: April 2025
See protocol details

Summary

Principal SponsorSunnybrook Health Sciences Centre
Study ContactNir Lipsman, MD, PhD, FRCPCMore contacts
Last updated: December 11, 2025
Sourced from a government-validated database.Claim as a partner
Study start date: April 15, 2025Actual date on which the first participant was enrolled.

This study aims to explore a new approach to treating amyotrophic lateral sclerosis (ALS) by enhancing the delivery of a medication called intravenous immunoglobulin (IVIg) directly to the brain's motor cortex. ALS is a serious disease that affects nerve cells in the brain and spinal cord, leading to loss of muscle control. The study focuses on six patients with ALS to see if this method is safe and feasible. By potentially improving the delivery of IVIg, the study hopes to find a more effective way to manage ALS symptoms and slow the disease's progression. Participants in the study will receive IVIg in two different doses. The first group of three patients will receive a lower dose, while the second group of three will receive a higher dose. The treatment involves a specialized procedure where, during the second dose, the blood-brain barrier is temporarily opened using focused ultrasound through a helmet-like device, allowing the IVIg to reach the motor cortex more effectively. This procedure takes place two weeks after the first dose and uses a combination of ultrasound and a special solution called microbubbles. The study will monitor the participants for safety and how well the treatment is working over 24 weeks through regular follow-up visits.

Official TitleTargeting Neuroinflammation With Enhanced Delivery of Intravenous Immunoglobulin to the Motor Cortex Using Next Generation Dome Helmet Focused Ultrasound in Amyotrophic Lateral Sclerosis: A Phase I, Open Label Trial 
Principal SponsorSunnybrook Health Sciences Centre
Study ContactNir Lipsman, MD, PhD, FRCPCMore contacts
Last updated: December 11, 2025
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details
6 patients to be enrolledTotal number of participants that the clinical trial aims to recruit.
Treatment Study
These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

How participants are assigned to different groups/arms
In this clinical study, all participants receive the same treatment. Since there is only one group, there is no need for randomization or assignment to different arms. This type of study is often used to test a new treatment without comparing it to another.

Other Ways to Assign Participants
Randomized allocation
: Participants are assigned randomly, like flipping a coin, to ensure fairness and reduce bias.

Non-randomized allocation
: Participants are assigned based on specific factors, such as their medical condition or a doctor's decision.

How treatments are given to participants
Participants receive treatments one after another in a pre-planned sequence. The next treatment may depend on how the participant responds to the previous one.

Other Ways to Assign Treatments
Single-group assignment
: Everyone gets the same treatment.

Parallel assignment
: Participants are split into separate groups, each receiving a different treatment.

Cross-over assignment
: Participants switch between treatments during the study.

Factorial assignment
: Participants receive different combinations of treatments.

Other assignment
: Treatment assignment does not follow a standard or predefined design.

How the effectiveness of the treatment is controlled
In a non placebo-controlled study, no participants receive an inert substance (placebo) to compare outcomes. Instead, all participants receive either the experimental treatment or an alternative treatment (often the Standard of Care). This method allows researchers to compare the effects of the experimental treatment with those of a different active intervention, rather than a placebo.

Other Options
Placebo-Controlled
: A placebo is used to compare the effects of the experimental treatment with those of an inert substance, isolating the true treatment effect.

How the interventions assigned to participants is kept confidential
Everyone involved in the study knows which treatment is being given. This is typically used when it's not possible or necessary to hide the treatment details from participants or researchers.

Other Ways to Mask Information
Single-blind
: Participants do not know which treatment they are receiving, but researchers do.

Double-blind
: Neither participants nor researchers know which treatment is given.

Triple-blind
: Participants, researchers, and outcome assessors do not know which treatment is given.

Quadruple-blind
: Participants, researchers, outcome assessors, and care providers all do not know which treatment is given.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria
Any sexBiological sex of participants that are eligible to enroll.
Over 18 YearsRange of ages for which participants are eligible to join.
Healthy volunteers not allowedIf individuals who are healthy and do not have the condition being studied can participate.
Conditions
Pathology
Amyotrophic Lateral Sclerosis
Central Nervous System Diseases
Metabolic Diseases
Nervous System Diseases
Neuromuscular Diseases
Nutritional and Metabolic Diseases
Spinal Cord Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Proteostasis Deficiencies
TDP-43 Proteinopathies
Criteria

Inclusion Criteria: 1. Diagnosed with ALS as per the Gold Coast Criteria; 2. Aged 18 years or older; 3. Capable of providing informed consent and complying with study procedures; 4. If taking Riluzole, on a stable dose for at least 4 weeks prior to Baseline; 5. If taking Edaravone, on a stable dose of one completed cycle prior to Baseline; 6. Respiratory Function Criterion: * Able to lie supine without BiPAP or breathing discomfort; OR * Forced vital capacity or slow vital capacity ≥50% of the predicted value for sex, height and age, if available 7. Able to communicate sensations during the Dome FUS procedure. 8. Qualified fit for the anesthesia by an anesthesiologist, ASA I-III. Exclusion Criteria: 1. MRI findings: 1. Active infection/inflammation 2. Acute or chronic hemorrhages, specifically \> 4 lobar microbleeds, and no siderosis or macrohemorrhages 3. Tumor/space occupying lesion causing significant mass effect 4. Meningeal enhancement 5. Intracranial hypotension 2. More than 30% of the skull area traversed by the sonication pathway is covered by scars, scalp disorders (e.g., eczema), or atrophy of the scalp 3. Clips or other metallic implanted objects in the skull or the brain, except shunts 4. Significant cardiac disease or unstable hemodynamic status including: 1. Documented myocardial infarction within six months of screening 2. Unstable angina on medication 3. Unstable or worsening congestive heart failure 4. Left ventricular ejection fraction below the lower limit of normal 5. History of a hemodynamically unstable cardiac arrhythmia 6. Cardiac pacemaker 7. Severe hypertension (diastolic BP \> 100 on medication) 8. Patient has right-to-left, bidirectional, or transient right-to-left cardiac shunts 9. QT prolongation observed on screening ECG (QTc \> 450 for men and \> 470 for women) 5. Uncontrolled hypertension (systolic \> 150 and diastolic BP \> 100 on medication) 6. Patients should not take medications known to increase risk of hemorrhage (e.g., aspirin or class I and III anticoagulants) for at least 7 days prior to treatment or lumbar puncture; patients should not take Avastin for 30 days prior to treatment 7. History of a bleeding disorder, coagulopathy or a history of spontaneous hemorrhage or use of anticoagulants, specifically recent thrombosis or stroke in past 3 months; abnormal coagulation profile (PLT \< 100,00/μl), PT (\> 14 sec) or PTT (\> 36 sec), and INR \> 1.3 8. No more than 1 non-strategic lacune \&lt;1.5 cm 9. Known cerebral or systemic vasculopathy 10. Significant depression and at potential risk of suicide 11. Known sensitivity/allergy to gadolinium (an alternative product may be used) and DEFINITY®. 12. Any contraindications to MRI scanning, including: 1. Large participants not fitting comfortably into the scanner 2. Difficulty lying supine and still for up to 3 hours in the MRI unit or claustrophobia 13. Any contraindication to lumbar puncture for collection of cerebral spinal fluid, including: a. Intracranial hypotension 14. Untreated, uncontrolled sleep apnea 15. Impaired renal function with estimated glomerular filtration rate \< 30 mL/min/1.73m2 or on dialysis. 16. IVIg use in the previous 6 months. 17. Live viral vaccination within the 30 days before study entry 18. Currently, or in the last 3 months participated in a clinical trial delivering an investigational product or non-approved use of a drug or device or in any other type of medical research. 19. Respiratory: chronic pulmonary disorders e.g. severe emphysema, pulmonary vasculitis, or other causes of reduced pulmonary vascular cross-sectional area, patients with a history of drug allergies, uncontrolled asthma or hay fever, and multiple allergies where the benefit/risk of administering DEFINITY® is considered unfavorable by the study physicians in relation to the product monograph for DEFINITY®. 20. Motor cortex atrophy deemed severe enough to limit targeting 21. Previous major allergic or anaphylactic reaction to IVIg 22. Known IgA deficiency with anti-IgA. 23. Known frontotemporal dementia; 24. Definitely or possibly pregnant (if applicable); 25. Known auto-immune condition with or without neurological manifestations (e.g., multiple sclerosis (MS), systemic lupus erythematous (SLE), Rheumatoid arthritis). 26. Current, planned or previous use of oral, intramuscular or intravenous steroid drugs (such as prednisone, prednisolone, dexamethasone, triamcinolone, methylprednisolone, oxandrolone, and others), immunosuppressant drugs (azathioprine, mycophenolate, tacrolimus, sirolimus, cyclophosphamide, and others) or NSAIDs (ibuprofen, naproxen, celecoxib, and others) in the past 30 days; 27. Other unspecified reasons that, in the opinion of the Investigator or the Sponsor, make the participant unsuitable for enrollment


Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives
One single intervention group 

is designated in this study

This study does not include a placebo group 

Treatment Groups
Group I
Experimental
ALS patients will be assigned to receive Intravenous Immunoglobulin (IVIg) and DEFINITY® microbubbles. The first three enrolled patients will participate in the first cohort, receiving 0.4g/kg divided in two doses. After a preliminary safety assessment of the first cohort, the next three successfully screened patients will be enrolled in the second cohort, receiving 0.8g/kg divided in two doses. The second IVIg dose in each cohort will be administered in combination with Next Generation Dome Helmet (NGDH) focused ultrasound (FUS) to transiently open the blood-brain barrier and enhance IVIg delivery to the primary motor cortex.

Two doses of IVIg will be administered 2 weeks apart. The first dose at Week 0 will be a standalone administration. The second dose at Week 2 will be combined with Next Generation Dome Helmet (NGDH) Focused Ultrasound (FUS) blood brain barrier (BBB) opening.

Two doses of IVIg will be administered 2 weeks apart. The first dose at Week 0 will be a standalone administration. The second dose at Week 2 will be combined with Next Generation Dome Helmet (NGDH) Focused Ultrasound (FUS) blood brain barrier (BBB) opening. Cohort I will receive 0.2g/kg of IVIg at each dose. Cohort II will receive 0.4g/kg of IVIg at each dose. Privigen® IVIg comes in vials containing 10% active ingredient. It is supplied in 2.5 g (25 mL bottle), 5 g (50 mL bottle), 10 g (100 mL bottle), 20 g (200 mL bottle) or 40 g (400 mL bottle). The IVIg dose will be determined based on the patient's ideal body weight.

DEFINITY® Perflutren Injectable Microbubbles is an ultrasound contrast imaging agent that will be used for blood brain barrier opening during focused ultrasound. These microbubbles will be injected during the focused ultrasound procedure on the day of the second IVIg dose.
Study Objectives
Primary Objectives

This will be assessed up through the incidence of drug-related adverse events, serious adverse events, and discontinuations due to adverse events after Intravenous Immunoglobulin (IVIg) administration.

This will be assessed up through the incidence of drug-related adverse events, serious adverse events, and discontinuations due to adverse events after DEFINITY® infusion during focused ultrasound (FUS) blood brain barrier (BBB) opening.

This will be measured as detectable gadolinium enhancement at the arm, leg and bulbar regions of the motor cortex bilaterally following FUS with posterior normalization.

Incidence of BBB opening-related and FUS-related adverse events, serious adverse events, incidence of asymptomatic or symptomatic radiologic complication, such as evidence of bleeding or swelling after FUS, incidence of electrographic complication, such as epileptiform discharges on EEG, or accelerated ALS disease progression, defined as ≥ 6-point decline in the ALSFRS-R scores from Baseline to week 8.
Secondary Objectives

Change in concentration of serum neurofilaments from Baseline to Week 24

Change in cerebrospinal fluid (CSF) and blood inflammatory markers, including but not limited to TNF-α, IL-1β, IL-2, IL-6, IL-8, IL-17, CHIT1, CHI3L1, CHI3L2, TGF-β, IL-10, and CRP

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.
This study has 1 location
Recruiting
Sunnybrook Health Sciences CentreToronto, CanadaSee the location

Recruiting
One Study Center
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