The Dutch Parkinson GBA Ambroxol trial (DUPARG-AMBROXOL): A randomised, double-blind, placebo-controlled, single-center trial with Ambroxol in Parkinson patients with a GBA mutatio

Phase 2

Recruiting

• Conditions: 10028037; Parkinson; Parkinson's disease

• Registration Number: NL-OMON52316
• Lead Sponsor: niversitair Medisch Centrum Groningen

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Not specified
• Target Recruitment: 80

Inclusion Criteria

- Diagnosis of Parkinson*s disease, according to Movement Disorders Society
(MDS) criteria <10 years
- PD patients carrying a GBA1 mutation
- Willingness to cooperate and sign written informed consent
- Willing and able to self-administer oral ambroxol medication

Exclusion Criteria

- The refusal to be informed about an unforeseen clinical finding
- Pregnant or breast feeding women
- Use of an implanted Deep Brain Stimulation (DBS) system
- MRI imcompatible implants in the body

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The primary endpoint is the comparison of MDS-UPDRS part III motor subscale in<br /><br>the practically defined OFF medication state at 60 weeks after a 12 week<br /><br>washout period.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Secondary endpoints are:<br /><br>- Safety and tolerability by examine adverse events<br /><br>- F-DOPA PET<br /><br>- MRI<br /><br>- Montreal Cognitive Assessment (MoCA)<br /><br>- Non-Motor Symptoms scale (NMSS)<br /><br>- Parkinson*s Disease 39 item Quality of life questionnaire (PDQ-39)<br /><br>- GCase activity measured using a flow cytometry based assay of peripheral<br /><br>blood mononuclear cells (PBMCs)</p><br>