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Ultrasound opens brain barrier to deliver Parkinson's drug in tiny trial

NCT ID NCT04370665

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This pilot study tested whether a focused ultrasound device could temporarily open the blood-brain barrier to deliver the drug Cerezyme into the brains of 4 people with Parkinson's disease. The main goal was to see if the procedure was safe and could successfully get the drug into a specific brain region. Because it is a very early, small study, it does not yet show whether this approach helps Parkinson's symptoms.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Cerezyme (imiglucerase)
What this could lead to
If this works, it could show a new way to deliver drugs directly to the brain for Parkinson's disease.
What could go wrong
This is a very small pilot study with only 4 participants, focused on safety and feasibility. It is too early to know if it will improve symptoms or change the course of the disease.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

4 people

The number who actually took part.

Started

Jul 2020

Finished

Feb 2022

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

35 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Men or women between age 35 and 75 years, inclusive. 2. Able and willing to give informed consent. 3. Diagnosis of Parkinson's Disease. 4. At least 2 years from initial diagnosis 5. On stable regiment of PD medications for at least 90 days prior to the study 6. Able to communicate during the Exablate MRgFUS procedure. 7. Able to attend all study visits. Exclusion Criteria: 1. Previous neurosurgical procedure for PD 2. Cerezyme hypersensitivity 3. Patients who had recent intracranial hemorrhage or stroke 4. Skull covered by scars, scalp disorders (e.g., eczema), or atrophy of the scalp. Patients who have clips or other metallic implanted objects in the skull or the brain. 5. Patients who are on blood thinners within washout period prior to treatment (i.e., antiplatelet or vitamin K inhibitor anticoagulants within 7 days, non-vitamin K inhibitor anticoagulants within 72 hours, or heparin-derived compounds within 48 hours of treatment). 6. Patients with a history of a bleeding disorder, blood clotting, coagulopathy or with a history of spontaneous tumor hemorrhage. 7. Patients receiving bevacizumab (Avastin) therapy. 8. Currently participating in another clinical therapeutic trial 9. Documented myocardial infarction within six months of enrollment. 10. Unstable angina on medication. 11. Congestive heart failure. 12. Unstable cardiac arrhythmia. 13. Cardiac pacemaker. 14. Severe hypertension (diastolic BP \> 100 on medication). 15. Documented cerebral infarction within the past 12 months or TIA in the past 1 month. 16. Insulin-dependent diabetes mellitus that is not well-controlled or that in the Investigator's opinion precludes participation in the study. 17. Known sensitivity to gadolinium 18. Known sensitivity to DEFINITY ultrasound contrast agent or perflutren. 19. Contraindications to MRI such as non-MRI-compatible implanted devices. 20. Large subjects not fitting comfortably into the MRI scanner. 21. Difficulty lying supine and still for up to 4 hours in the MRI unit or claustrophobia. 22. Untreated, uncontrolled sleep apnea. 23. Positive pregnancy test (for pre-menopausal women). 24. Known life-threatening systemic disease. 25. Severely impaired renal function and/or on dialysis. 26. Right to left or bi-directional cardiac shunt. 27. Subjects with evidence of cranial or systemic infection. 28. Subjects with uncontrolled chronic pulmonary disorders. 29. Subjects with a history of severe asthma, hay fever, or multiple allergies that is not well-controlled (e.g. with anaphylaxis). 30. Subjects with a family or personal history of cardiac arrhythmia. 31. Severe liver injury.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Sunnybrook Health Sciences Centre

    Toronto, Ontario, M4N 3M5, Canada

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Other studies related to the condition(s) this trial covers.