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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 mutation

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 mutation - The Dutch Parkinson GBA Ambroxol trial

Status
Recruiting
Phases
Phase 2
Study type
Interventional
Source
NL-OMON
Registry ID
NL-OMON52316
Enrollment
80
Registered
2023-03-13
Start date
2023-06-01
Completion date
Unknown
Last updated
2024-04-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Parkinson Parkinson's disease

Interventions

Patients will start with oral tablets of Ambroxol 600mg or placebo, which will increase with 600mg per week, to a maximum of 3 times 600mg a day for a period of 48 weeks. At 48 weeks all participant

Sponsors

Universitair Medisch Centrum Groningen
Lead Sponsor

Eligibility

Age
18 Years to 99 Years

Inclusion criteria

Inclusion criteria: - Diagnosis of Parkinson*s disease, according to Movement Disorders Society (MDS) criteria

Exclusion criteria

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

Design outcomes

Primary

MeasureTime frame
The primary endpoint is the comparison of MDS-UPDRS part III motor subscale in the practically defined OFF medication state at 60 weeks after a 12 week washout period.

Secondary

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

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)