•Research study to investigate the safety, tolerability and effects of multiple doses of LTI-291 in patients with Parkinson’s Disease with a GBA1 mutation.
- Conditions
- PD patients with a GBA1 mutation , movement disorder
- Registration Number
- NL-OMON23923
- Lead Sponsor
- •Lysosomal Therapeutics, Incorporated
- Brief Summary
A
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 40
1.Signed informed consent prior to any study-mandated procedure.
2.Minimum age of 18 years.
1.Any active or chronic disease or condition other than PD that could interfere with, or for which the treatment might interfere with, the conduct of the study, or that would pose an unacceptable risk to the subject in the opinion of the investigator (following medical history review, physical examination, vital signs (supine systolic and diastolic blood pressure, pulse rate, body temperature), 12-lead electrocardiogram (ECG), and clinical laboratory parameters (hematology, blood chemistry, and urinalysis)). Minor deviations of laboratory values from the normal range may be accepted, if judged by the investigator to have no clinical relevance.
2.History of recent major surgery (within 60 days of screening) that could interfere with, or for which the treatment might interfere with, the conduct of the study, or that would pose an unacceptable risk to the subject in the opinion of the investigator.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method •Safety and tolerability endpoints
- Secondary Outcome Measures
Name Time Method •Functional outcome measures<br /><br>•Pharmacokinetic endpoints<br /><br>•Pharmacodynamic endpoints<br>