Clinical Research Studies

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

• Conditions:
  Parkinson’s Disease (pd) With Glucocerebrosidase 1 (gba1) Mutation
• Age: Between 40 years - 75 years
• Gender: Male or Female

• Other Inclusion Criteria:
  

  1. Subjects diagnosed with Parkinson's Disease
  2. Subjects with body weight range of =40 kg (88 lbs) to =110 kg (242 lbs) and a body mass index (BMI) of 18 to 34 kg/m2
  3. Subjects in Hoehn and Yahr Stage II-IV
  4. Subjects who have stable use of background medication at least 8 weeks prior to study drug dosing
  5. Subjects who have at least 1 pathogenic GBA1 mutation confirmed by the central laboratory
  6. Subjects and/or legally authorized representative (LAR) has the ability to understand the purpose and risks of the study and provide written informed consent and authorization to use protected health information in accordance with national and local privacy regulations.
  7. Subjects has a reliable study partner/informant (e.g., family member, friend) willing and able to participate in the study as a source of information on the patient’s health status and cognitive and functional abilities
  8. Women of nonchildbearing potential must be either surgically sterile or postmenopausal
  9. Subjects of childbearing potential must use a highly effective method of contraception consistently and correctly for the duration of the study including the long-term follow-up.
  10. Male subjects must agree to abstain from sperm donation for the duration of the study
  11. Women of childbearing potential must have a negative result for the serum pregnancy test
  12. Subjects who are generally ambulatory, not dependent on walker or wheelchair
  13. Subjects who are living in the community (i.e., not in nursing home); some levels of assisted living may be permitted at the discretion of the PI

  
  

You may not be eligible for this study if the following are true:

• 1. Subjects diagnosed of a significant CNS disease other than PD that may be a cause for the patient’s PD symptoms or may confound study objectives
  2. Subjects with MoCA score of <14
  3. Subjects with brain MRI/magnetic resonance angiography (MRA) indicating clinically significant abnormality
  4. Subjects with contraindications to corticosteroid use
  5. Concomitant disease or condition within 6 months of Screening that could interfere with, or treatment of which might interfere with, the conduct of the study or that would, in the opinion of the PI, pose an unacceptable safety risk to the patient or interfere with the patient's ability to comply with study procedures
  6. Subjects who has clinically significant abnormalities in laboratory test results as per the protocol
  7. Subjects who participated within 6 months in another therapeutic investigational drug or device study with purported disease-modifying effects on PD
  8. Subjects with history of deep brain stimulator placement, focused ultrasound, or surgery for PD
  9. Subjects who have any type of prior gene or cell therapy
  10. Subjects who received immunizations (live vaccines) in the 4 weeks prior to Screening
  11. Subjects who used ambroxol within 8 weeks of dosing
  12. Subjects who used blood thinners in the 2 weeks prior to Screening or the anticipated need to initiate blood thinners during the study.
  13. Subjects who are using systemic immunosuppressant therapy other than protocol-specified steroids and topicals
  14. Contraindications or intolerance to radiographic visualization methods (MRI, CT, DaT-SPECT) inducing claustrophobia and intolerance to contrast agents used for MRI or CT
  15. Subjects with contraindications to general anesthesia
  16. Positive urine test for drugs of abuse without prescription, or positive breath alcohol test at Screening and Day -1.