Status:
RECRUITING
High-Dose Ambroxol in GBA1-Related Parkinson
Lead Sponsor:
Agyany Pharma LTD
Conditions:
Parkinson Disease
GBA Gene Mutation
Eligibility:
All Genders
30-70 years
Phase:
PHASE1
PHASE2
Brief Summary
Parkinson's disease (PD), affecting 10 million people globally, lacks a cure, and current therapies only manage symptoms. A link between Gaucher disease (GD) and PD, particularly in carriers of glucoc...
Detailed Description
Parkinson disease (PD) is the second most common neurodegenerative disease affecting around 10 million people worldwide. It is a debilitating disorder that despite many years and billions of dollars r...
Eligibility Criteria
Inclusion
- For inclusion into the trial, subjects are required to fulfill all of the following criteria:
- Newly diagnosed PD patients:
- Individuals who exclusively carry at least one single GBA1 variant without any additional genetic variants.
- Confirmed diagnosis of PD, by a movement disorder specialist, according to MDS PD criteria, within a maximum of three years from the date of diagnosis, coupled with the following conditions:
- iii. Hoehn and Yahr staged between I-II, inclusive.
- iv. No motor fluctuations or L-dopa induced dyskinesia.
- Stable anti-PD medications for ≥ 4 weeks:
- Subjects can take PD medications including NMDA glutamate antagonists, monoamine oxidase B (MAO-B) inhibitors, dopamine agonists, and L-Dopa.
- Male or female, age 30-70 years; however, if female:
- must be using contraception measures if of childbearing potential.
- must not be lactating.
- Complying with study protocol.
Exclusion
- Eligible subjects may not have any of the following exclusion criteria:
- Presence of any medical, emotional, behavioral, or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study (such as clinical depression).
- Any other disorder that may interfere with the results of the efficacy endpoints.
- Currently taking another investigational drug for any condition.
- Use of dopaminergic treatment under these conditions:
- L-Dopa equivalent daily dose \> 400mg
- L-Dopa daily dose \> 300mg
- L-Dopa equivalent and L-Dopa daily dose has been changed in the past 4 weeks prior to screening visit.
- Medical history of psychosis.
- Exposure to ambroxol in the last 24 months prior to screening and/or history of adverse events to ambroxol.
- Exposure to dopamine receptor blocking agents, lithium, cinnarizine, amiodarone or valproic acid in the last 12 months prior to screening.
- Pregnancy or lactation; female subjects of a childbearing age who are unwilling to use contraceptive measures.
Key Trial Info
Start Date :
October 24 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
April 30 2025
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT06193421
Start Date
October 24 2023
End Date
April 30 2025
Last Update
February 8 2024
Active Locations (1)
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1
Shaare Zedek Medical Center
Jerusalem, Israel, 9103102