Status:
COMPLETED
Ambroxol as a Disease-modifying Treatment in GBA-PD
Lead Sponsor:
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Collaborating Sponsors:
IRCCS National Neurological Institute "C. Mondino" Foundation
University of Campania Luigi Vanvitelli
Conditions:
Parkinson Disease
GBA Gene Mutation
Eligibility:
All Genders
21-80 years
Phase:
PHASE2
Brief Summary
The present multicenter, randomized, double-blind, placebo-controlled clinical trial will investigate whether the prolonged administration of high-dose oral Ambroxol over 52 weeks is safe, tolerable, ...
Detailed Description
Glucocerebrosidase (GCase) is a lysosomal enzyme encoded by the GBA1 beta-glucosylceramidase gene (GBA). Heterozygous GBA mutations are recognized as the most frequent genetic risk factor for Parkinso...
Eligibility Criteria
Inclusion
- Age 21-80 years
- Diagnosis of idiopathic PD
- Duration of motor symptoms \>5 years
- Heterozygous carrier of a GBA1 mutation.
- Capable of complying with all study procedures, including fasting lumbar puncture
- All male and female participants of childbearing age must agree with their partners to use double barrier birth control or total abstinence during study participation and for 2 weeks after the last dose of study drug.
- Male participants who have received bilateral vasectomy are permanently sterile.
- A woman can participate if she is of:
- Non-childbearing potential
- Women of childbearing potential must have a negative pregnancy test at the screening visit and use accepted contraceptive methods defined as highly effective.
Exclusion
- Secondary and primary atypical parkinsonism
- Diagnosis of Parkinson-Dementia (MDS Level II criteria) or other conditions that result in inability to understand and sign the informed consent
- Hoehn \& Yahr stage ≥ 4/5 in the medication-ON condition
- Deep Brain Stimulation
- Any clinically significant or unstable medical condition, which, in the opinion of the principal investigator or the clinician delegated by the principal investigator, may put the participant at risk when participating in the study (e.g. previous gastric/duodenal peptic ulcer, chronic obstructive pulmonary disease, severe liver or kidney changes, major cardiovascular event (e.g. myocardial infarction, decompensated congestive heart failure, pulmonary embolism occurring within 6 months prior to the screening visit), neoplastic diseases).
- Bronchial asthma
- Abnormalities that could preclude safe completion of the spinal cord in the investigator's opinion, including: treatment with anticoagulants; severe abnormalities or malformations of the lower spine or other spinal disorders; bleeding diathesis (e.g. clinically significant coagulopathies or thrombocytopenia); hypersensitivity to lidocaine.
- Pregnant or breastfeeding women.
- All participants of childbearing age who disagree to use double barrier or abstinence birth control while participating in the study and for 2 weeks after the last dose of study drug;
- Known hypersensitivity to the active substance Ambroxol or to any of its excipients.
Key Trial Info
Start Date :
March 9 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 20 2024
Estimated Enrollment :
65 Patients enrolled
Trial Details
Trial ID
NCT05287503
Start Date
March 9 2022
End Date
December 20 2024
Last Update
August 14 2025
Active Locations (3)
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1
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Italy, 20133
2
University of Campania "Luigi Vanvitelli"
Naples, Italy
3
IRCCS National Neurological Institute "C. Mondino" Foundation
Pavia, Italy, 27100