Blood-Brain-Barrier Disruption With Cerezyme in Patient's With Parkinson's Disease

  • STATUS
    Recruiting
  • participants needed
    6
  • sponsor
    InSightec
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Updated on 19 February 2024
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parkinson's disease

Summary

This clinical trial focus on the delivery of Cerezyme in Parkinson's Disease (PD) patients using MR-guided focused ultrasound (MRgFUS) induced opening of the blood-brain barrier (BBB).

Description

Leveraging the preclinical evidence and clinical experience with MRgFUS BBB opening in human subjects, the investigators propose a phase I open-label study to determine the safety and feasibility of three biweekly delivery of Cerezyme (an analogue of the human enzyme beta-glucocerebrosidase) via MRgFUS induced BBB opening to unilateral putamen in PD. The putamen contains terminals of dopaminergic neurons from the substantia nigra, and is a critically affected brain region in PD. The primary objective is to determine the feasibility of repeated MRgFUS BBB opening for Cerezyme delivery in unilateral putamen. The secondary objective is to describe the safety and tolerability of the procedure. Another objective is to measure changes in PD relevant clinical scores and Cerezyme relevant biomarkers in the study.

Details
Condition Parkinson's Disease, Parkinson's Disease
Age 35years - 75years
Treatment Exablate BBBD with Cerezyme
Clinical Study IdentifierNCT04370665
SponsorInSightec
Last Modified on19 February 2024

Eligibility

 Yes No Not Sure

Inclusion Criteria

Men or women between age 35 and 75 years, inclusive
Able and willing to give informed consent
Diagnosis of Parkinson's Disease
At least 2 years from initial diagnosis
On stable regiment of PD medications for at least 90 days prior to the study
Able to communicate during the Exablate MRgFUS procedure
Able to attend all study visits

Exclusion Criteria

Previous neurosurgical procedure for PD
Cerezyme hypersensitivity
Patients who had recent intracranial hemorrhage or stroke
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
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)
Patients with a history of a bleeding disorder, blood clotting, coagulopathy or with a history of spontaneous tumor hemorrhage
Patients receiving bevacizumab (Avastin) therapy
Currently participating in another clinical therapeutic trial
Documented myocardial infarction within six months of enrollment
Unstable angina on medication
Congestive heart failure
Unstable cardiac arrhythmia
Cardiac pacemaker
Severe hypertension (diastolic BP > 100 on medication)
Documented cerebral infarction within the past 12 months or TIA in the past 1 month
Insulin-dependent diabetes mellitus that is not well-controlled or that in the Investigator's opinion precludes participation in the study
Known sensitivity to gadolinium
Known sensitivity to DEFINITY ultrasound contrast agent or perflutren
Contraindications to MRI such as non-MRI-compatible implanted devices
Large subjects not fitting comfortably into the MRI scanner
Difficulty lying supine and still for up to 4 hours in the MRI unit or claustrophobia
Untreated, uncontrolled sleep apnea
Positive pregnancy test (for pre-menopausal women)
Known life-threatening systemic disease
Severely impaired renal function and/or on dialysis
Right to left or bi-directional cardiac shunt
Subjects with evidence of cranial or systemic infection
Subjects with uncontrolled chronic pulmonary disorders
Subjects with a history of severe asthma, hay fever, or multiple allergies that is not well-controlled (e.g. with anaphylaxis)
Subjects with a family or personal history of cardiac arrhythmia
Severe liver injury
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