MR-guided Focused Ultrasound Plus GCase

Launched by INSIGHTEC · Sep 29, 2022

Keywords

ClinConnect Summary

This clinical trial is investigating a new treatment approach for Parkinson’s disease (PD) using a method called MR-guided Focused Ultrasound (MRgFUS) combined with a medication called GCase. The aim is to see if delivering GCase directly into the brain is safe and effective. In previous studies with animals, this method showed promise in improving symptoms and brain health related to Parkinson's disease.

To participate in the study, individuals need to be between 35 and 80 years old and have been diagnosed with Parkinson's disease for at least two years. They should be currently taking medication for PD and meet specific health criteria. Participants can expect to undergo MRgFUS treatment and will be closely monitored throughout the study. It’s important to note that there are certain conditions that would prevent someone from joining, such as pregnancy or severe kidney problems. If you or a loved one are interested in this study, it could be a chance to contribute to research that may lead to better treatments for Parkinson’s disease.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Men or women between age 35 and 80 years, inclusive.
• • Able and willing to give informed consent.
• • Diagnosis of PD satisfying MDS Clinical Diagnostic Criteria for PD
• • At least 2 years from initial diagnosis
• • Hoehn and Yahr Stage 1-3 on PD medication
• • Dopaminergic deficit by a positive DAT SPECT scan
• • On stable regimen of PD medications for at least 90 days prior to the study
• • American Society of Anesthesiologists (ASA) score 1-3
• • Harbor at least one GBA mutation if enrolled in the GBA PD arm or two wild-type GBA alleles if enrolled in the idiopathic PD arm
• Exclusion Criteria:
• • Positive pregnancy test (for pre-menopausal women).
• • Contraindication to DEFINITY ultrasound contrast agent or perflutren (e.g. hypersensitivity, known left or bidirectional cardiac shunt)
• • Contraindication to MRI or gadolinium-DTPA (e.g. incompatible device, hypersensitivity)
• • Insulin-dependent diabetes mellitus that is not well-controlled or that in the Investigator's opinion precludes participation in the study.
• • Severely impaired renal function with estimated glomerular filtration rate \<30 mL/min/1.73m2 and/or on dialysis.
• • Currently participating in another clinical therapeutic trial
• • Patient receiving bevacizumab (Avastin) therapy
• • Subjects with evidence of cranial or systemic infection.
• • Cerebral or systemic vasculopathy.
• • Documented cerebral infarction within the past 12 months or TIA in the past 1 month.
• • Contraindication to GCase enzyme therapy, specifically previous hypersensitivity reaction to GCase enzyme therapy
• • Parkinsonism plus symptoms, secondary parkinsonism
• • Previous neurosurgical procedure for PD