Ambroxol

← Pipeline

Ambroxol

University College London · Parkinson's disease
Mid-stage completed completed CT.gov grounded

Mechanism-led repurposing program that keeps the Parkinson's pipeline from being only antibody and device narratives.

Open Parkinson's disease hub Filter main pipeline

Program overview

Mechanism
glucocerebrosidase support / lysosomal modulation
Modality
small molecule repurposing
Phase
Mid-stage
Status
completed
Recruitment
completed
Confidence
medium
Watch priority
2
Last verified
20 Apr 2026

Sponsor and trial identifiers

Sponsor
University College London
Trial IDs
NCT05287503, NCT02914366
ClinicalTrials.gov exact matches: NCT05287503, NCT02914366

Regions and recruitment

Active in 2 regions
Italy Canada

Indication tags

Parkinson's GBA-PD

Milestones and catalysts

Last milestone
A prominent UK-linked Parkinson's program testing a mechanistically plausible repurposing path.
Next expected catalyst
Whether clinical and biomarker read-throughs justify a larger disease-modification push.

Related pages

Disease hub Parkinson's disease hub Disease-specific views with programs, mechanisms, and related context.
Research Research agenda Research notes and background around Parkinson's disease.
Key Targets Key targets The mechanisms and intervention levers behind these programs.
Discover Discover Explainers and visual introductions to the science behind neurodegeneration.
Join Get involved Contribute research, writing, design, engineering, or review.

Related programs

Each card explains why it is here so you can read the overlap at a glance.

Priority 2
Mid-stage active active, not recruiting

A genetically targeted Parkinson's program that fits the monitor's mechanism-first framing well.

Shown because it shares same disease bench and shared biology: parkinson's, gba-pd with Ambroxol.
Mechanism
GCase pathway modulation
Modality
small molecule
Next catalyst
Further efficacy and pharmacodynamic data in GBA-associated Parkinson's disease.
Last verified
20 Apr 2026

PR001 (LY3884961)

Prevail Therapeutics / Eli Lilly · Parkinson's disease
Priority 2
Early / Mid-stage active active, not recruiting

Gene-therapy Parkinson's program that gives the watchlist a cleaner exact-grounded GBA1 entry.

Shown because it shares same disease bench and shared biology: parkinson's with Ambroxol.
Mechanism
GBA1 gene replacement
Modality
AAV gene therapy
Next catalyst
Further safety and biomarker interpretation in GBA1-associated Parkinson's disease.
Last verified
20 Apr 2026

Prasinezumab

Prothena / Roche · Parkinson's disease
Priority 2
Mid-stage active recruiting

High-visibility Parkinson's program targeting alpha-synuclein pathology.

Shown because it shares same disease bench and shared biology: parkinson's with Ambroxol.
Mechanism
anti-alpha-synuclein antibody
Modality
monoclonal antibody
Next catalyst
Further progression and functional readouts.
Last verified
20 Apr 2026
Priority 3
Mid-stage completed completed

Parkinson's program with direct mechanistic relevance to alpha-synuclein biology rather than only symptomatic management.

Shown because it shares same disease bench and shared biology: parkinson's with Ambroxol.
Mechanism
alpha-synuclein misfolding inhibition
Modality
small molecule
Next catalyst
Mostly interpretive now, but still important for understanding how far small-molecule alpha-syn strategies can go.
Last verified
20 Apr 2026

Source links

ClinicalTrials.gov NCT05287503 ClinicalTrials.gov NCT02914366 ClinicalTrials.gov search UCL trial news
Scroll to Top