Ambroxol to Slow Progression in Parkinson Disease
ASPro-PD
1 other identifier
interventional
330
1 country
15
Brief Summary
This is a UK only clinical trial in patients with Parkinson's disease (PD) of a drug called ambroxol hydrochloride, which is an already licensed drug for the treatment of respiratory conditions (such as a common cold) in many European countries. The aim of this trial is to find out whether ambroxol hydrochloride can slow down the progression of Parkinson's disease and to evaluate it's safety and tolerability.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3 parkinson-disease
Started Feb 2025
Typical duration for phase_3 parkinson-disease
15 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 16, 2023
CompletedFirst Posted
Study publicly available on registry
March 21, 2023
CompletedStudy Start
First participant enrolled
February 25, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 1, 2029
ExpectedStudy Completion
Last participant's last visit for all outcomes
September 1, 2029
May 1, 2025
April 1, 2025
3.9 years
February 16, 2023
April 30, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change in Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I- III score from baseline to Week 104.
The MDS-UPDRS is a comprehensive 50 question assessment of both motor and non-motor symptoms associated with Parkinson's. Comparison of MDS-UPDRS Parts I-III total score at 104 weeks between participants according to treatment allocation will be made. Parts I and II are measured in the practically defined ON medication state and Part III is measured in the practically defined OFF medication state. Parts I and II are historical data assessed by an examiner and are designed to rate mentation, behaviour and mood; Part III is done as a motor examination at the time of a visit. Participants will undergo MDS-UPDRS assessment at baseline, week 20, week 40, week 60, week 80, week 104. The MDS-UPDRS measures patient status on a scale 0, which is normal or none, to 4, which is severe or the worst scenario. MDS-UPDRS score = sum of Parts I, II and III (Range: 0 to 236). Higher score indicative of worse outcome.
Baseline; Week 104
Secondary Outcomes (43)
Motor signs of PD using the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) part III score in the OFF medication state from baseline to Week 104.
Baseline; Week 20; Week 40; Week 60; Week 80; Week 104.
Motor complications of PD using the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) part IV score in the ON medication state from baseline to Week 104.
Baseline; Week 20; Week 40; Week 60; Week 80; Week 104.
The non-motor impact of PD on patients using the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) part I score in the ON medication state baseline to Week 104.
Baseline; Week 20; Week 40; Week 60; Week 80; Week 104.
The motor aspect impact of PD of patients experiences on daily living using the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) part II score in the ON medication state from baseline to Week 104.
Baseline; Week 20; Week 40; Week 60; Week 80; Week 104.
The Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) tremor score from baseline to Week 104.
Baseline; Week 20; Week 40; Week 60; Week 80; Week 104.
- +38 more secondary outcomes
Other Outcomes (7)
The association between ambroxol, the glucocerebrosidase enzyme and other proteins associated with the development of PD measured by glucocerebrosidase enzyme activity and other protein markers in blood and cerebrospinal fluid (CSF) samples.
Baseline; Week 20; Week 40; Week 60; Week 80; Week 104
The association between ambroxol and alpha-synuclein levels in blood and cerebrospinal fluid (CSF) samples from baseline to Week 104.
Baseline; Week 20; Week 40; Week 60; Week 80; Week 104
The association between ambroxol and glycerylceramide, neurofilament light chain and lipid levels in cerebrospinal fluid (CSF) samples from baseline to Week 104.
Baseline; Week 20; Week 40; Week 60; Week 80; Week 104
- +4 more other outcomes
Study Arms (2)
Ambroxol hydrochloride
ACTIVE COMPARATORParticipants will receive ambroxol hydrochloride (tablets) for 104 weeks (blinded treatment period). Participants will receive ambroxol hydrochloride 420mg daily for Days 1-5, then 840mg daily for Days 6-10, then 1260mg for the remainder of the blinded treatment period. Participants will then enter the open-label extension and will receive ambroxol hydrochloride 420mg daily for Days 1-5, then 840mg daily for Days 6-10, then 1260mg for the remainder of the open-label extension phase. n=165
Placebo
PLACEBO COMPARATORParticipants will receive ambroxol hydrochloride matching placebo (tablets) for 104 weeks (blinded treatment period). Participants will receive ambroxol hydrochloride matching placebo 420mg daily for Days 1-5, then 840mg daily for Days 6-10, then 1260mg for the remainder of the blinded treatment period. Participants will then enter the open-label extension and will receive ambroxol hydrochloride 420mg daily for Days 1-5, then 840mg daily for Days 6-10, then 1260mg for the remainder of the open-label extension phase. n=165
Interventions
Eligibility Criteria
You may qualify if:
- A diagnosis of Parkinson's disease (in accordance with the MDS diagnostic criteria) within 7 years of the screening visit confirmed by year of diagnosis.
- Adults aged ≥ 35 and ≤ 75 years.
- Hoehn and Yahr stage between 1-2.5, inclusive (in ON stage) at screening visit.
- Known glucocerebrosidase gene (GBA1) status, positive or negative (status MUST be confirmed prior to screening).
- On stable dopaminergic treatment for at least 3 months before enrolment.
- Able and willing to provide informed consent prior to any study related assessments and/or procedures.
- Able and willing to attend trial visits and comply with all study procedures for the duration of the trial.
- Willing and able to self-administer oral ambroxol medication or placebo.
You may not qualify if:
- Participation in another interventional clinical trial of an Investigational Medicinal Product (IMP) and use of an Investigational Medicinal Product (IMP) within 90 days prior to the first dose of trial treatment.
- Use of an Investigational Medicinal Product (IMP) within 90 days prior to the first dose of trial treatment.
- Participation in another clinical trial of an Investigational New Drug being tested for PD disease modifying potential within 12 months prior to the first dose of trial treatment.
- Past surgical history of deep brain stimulation.
- Use of ambroxol in the past 12 months.
- Exposure to Exenatide within 12 months prior to the first dose in this current trial.
- Concomitant medications that in the opinion of the Investigator would preclude participation in the study e.g., exenatide or other GLP1 agonist for diabetes.
- Confirmed dysphagia that would preclude self-administration of ambroxol.
- History of known sensitivity to the study medication, ambroxol or its excipients (lactose monohydrate, granulated microcrystalline cellulose, copovidone and magnesium stearate) in the opinion of the investigator that contraindicates their participation.
- History of known rare hereditary disorders of galactose intolerance, Lapp lactase deficiency or glucose-galactose malabsorption.
- Presence of the LRRK2 G2019S mutation (status to be confirmed prior to screening).
- History of drug abuse or alcoholism in the opinion of the Investigator that would preclude participation in the trial.
- Pregnant (or planned pregnancy during the trial) and/or breastfeeding.
- Women of childbearing potential (WOCBP) and male participants with a partner of childbearing potential not willing to use highly effective contraception or abstinence for the duration of the trial treatment and for 2 weeks following the last dose of the study drug.
- Any clinically significant or unstable medical or surgical condition that in the opinion of the Investigator may; put the participant at risk when participating in the study, influence the results of the study or affect the participants ability to take part in the study, as determined by medical history, physical examinations, electrocardiogram (ECG) or laboratory tests. Such conditions may include:
- +9 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (15)
University Hospitals Birmingham
Birmingham, United Kingdom
Southmead Hospital Bristol
Bristol, United Kingdom
Addenbrookes NHS Trust
Cambridge, United Kingdom
Western General Hospital
Edinburgh, United Kingdom
Kings College London
London, United Kingdom
Royal London Hospital
London, United Kingdom
University College London Hospital's
London, United Kingdom
Newcastle
Newcastle, United Kingdom
Northumbria
Newcastle upon Tyne, United Kingdom
The John Radcliffe Hospital
Oxford, United Kingdom
Derriford Hospital
Plymouth, United Kingdom
Fairfield General Hospital
Salford, United Kingdom
Salford Royal Hospital
Salford, United Kingdom
Southampton General Hospital
Southampton, United Kingdom
Prince Philip Hospital
Wales, United Kingdom
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Anthony Schapira
University College, London
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Masking Details
- Double Blind
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 16, 2023
First Posted
March 21, 2023
Study Start
February 25, 2025
Primary Completion (Estimated)
February 1, 2029
Study Completion (Estimated)
September 1, 2029
Last Updated
May 1, 2025
Record last verified: 2025-04