ambroxol hydrochloride; clenbuterol hydrochloride for Parkinson's disease

Inclusion criteria

• {"criterion_text":"- Diagnosis of Parkinson's Disease according to Movement Disorders Society (MDS) criteria"}
• {"criterion_text":"- GBA1-mutation"}
• {"criterion_text":"- Disease duration of PD < 10 years at time of inclusion"}
• {"criterion_text":"- Able to write written informed consent, understanding study protocol and perform protocol related actions"}
• {"criterion_text":"- Willingness and ability to self-administer oral ambroxol or placebo medication"}

Exclusion criteria

• {"criterion_text":"- The refusal to be informed about an unforeseen clinical finding"}
• {"criterion_text":"- Use of an implanted Deep Brain Stimulation (DBS) system"}
• {"criterion_text":"- History of known sensitivity to the study medication"}
• {"criterion_text":"- Pregnant or breastfeeding women"}
• {"criterion_text":"- MRI incompatible implants in the body"}
• {"criterion_text":"- Any clinically significant or unstable medical or surgical condition that in the opinion of the principal investigator may put the participant at risk when participating in the study or may influence the results of the study or affect the participant’s ability to take part in the study, as determined by medical history, physical examinations, electrocardiogram (ECG), or laboratory tests."}

Primary endpoints

• {"endpoint_text":"- Comparison of MDS-UPDRS part III motor subscale in the practically defined OFF medication state at 60 weeks","definition_or_measurement_approach":"MDS-UPDRS part III motor subscale assessed in the practically defined OFF medication state at 60 weeks; comparison between treatment groups."}

Secondary endpoints

• {"endpoint_text":"- Safety and tolerability by examination of adverse events","definition_or_measurement_approach":"Assessment of safety and tolerability by monitoring and recording adverse events."}
• {"endpoint_text":"- F-DOPA PET","definition_or_measurement_approach":"F-DOPA PET imaging to assess relevant brain biochemical/functional parameters."}
• {"endpoint_text":"- MRI","definition_or_measurement_approach":"Magnetic Resonance Imaging (MRI) as an imaging endpoint."}
• {"endpoint_text":"- Montreal Cognitive Assessment (MoCA)","definition_or_measurement_approach":"Cognitive function assessed using the MoCA instrument."}
• {"endpoint_text":"- Non-Motor Symptoms scale (NMSS)","definition_or_measurement_approach":"Assessment of non-motor symptoms using the NMSS questionnaire."}
• {"endpoint_text":"- Parkinson’s Disease Quality of life questionnaire with 39 items (PDQ-39)","definition_or_measurement_approach":"Health-related quality of life measured using the PDQ-39 questionnaire."}
• {"endpoint_text":"- GCase activity measuring sphinglolipids in PBMCs and plasma","definition_or_measurement_approach":"Biochemical measurement of GCase activity and sphingolipids in PBMCs and plasma samples."}

Netherlands

Earliest CTIS Part Ii Submission Date

17-09-2024

Latest Decision Or Authorization Date

23-09-2024

Processing Time Days

6

Number Of Sites

1

Number Of Participants

80

Primary sponsor

Full Name

Universitair Medisch Centrum Groningen

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Netherlands