Asset profile
Ambroxol
Small molecule
Evidence outlook · registrational
Parkinson's Disease: registrational readout pending
Entered Phase 3 on partial signal mid-stage evidence. Registrational readout expected around 2029-02.
AMBITIOUS (NCT05287503): The trial randomized 65 participants to ambroxol or placebo with participants, care providers, investigators, and outcome assessors masked. Its primary outcomes were clinical cognitive measures. Registry results were not posted at the time of grading; the key result shown was recovered from the cited external source.
Source is the AMBITIOUS trial preprint (SSRN, not yet peer reviewed); the quote was transcribed from indexed renderings of the abstract because the SSRN page blocks automated retrieval. The direction of the result and the companion levodopa-equivalent figure are corroborated by independent renderings.
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Featured trials
The studies most instrumental to this drug's clinical progression, individually graded on design and result. Pick an indication; Research unlocks the trials, endpoints, key results and grades.
Competitive landscape
64 competitors in this universe: 4 direct rivals (same mechanism and indication), 0 same-class, 60 same-indication; 18 further along in development.
| Drug | Stage | Modality | Mechanism | Evidence signal | Reported efficacygraded featured trials |
|---|---|---|---|---|---|
| Ambroxol: this drug | Phase 3 | Small molecule | GBA1/glucocerebrosidase | ||
| GT-02287 | Phase 2 | Small molecule | GBA1/glucocerebrosidase | ||
| PR001 (LY3884961) | Phase 1/2 | Gene therapy / editing | GBA1/glucocerebrosidase | ||
| VGN-R08b | Phase 1/2 | Gene therapy / editing | GBA1/glucocerebrosidase | ||
| CAP-003 | Not yet studied | Gene therapy / editing | GBA1/glucocerebrosidase | ||
| Amantadine ER (Gocovri / ADS-5102) | Approved | Small molecule | NMDA receptor | ||
| Apomorphine (Apokyn) | Approved | Small molecule | dopamine | ||
| Apomorphine Sublingual Film (Kynmobi) | Approved | Small molecule | dopamine | ||
| Crexont (IPX203) | Approved | Small molecule | dopamine precursor | ||
| Entacapone (Comtess) | Approved | Small molecule | COMT | ||
| Foslevodopa / Foscarbidopa (Vyalev) | Approved | Small molecule | dopamine precursor | ||
| Inbrija (CVT-301) | Approved | Small molecule | dopamine precursor | ||
| Istradefylline (Nourianz) | Approved | Small molecule | adenosine A2A | ||
| Levodopa-Carbidopa Intestinal Gel (Duopa) | Approved | Small molecule | dopamine precursor | ||
| Opicapone (Ongentys) | Approved | Small molecule | COMT, PDE4 | ||
| Parcopa | Approved | Small molecule | dopamine precursor |
…and 49 more. Research shows the complete landscape.
Unlock the full comparison
Research adds each competitor's evidence grade and sourced trial results, aligned per race, so you can see who actually has the data.
Upcoming events
(2 total)
Estimated primary-completion dates from ClinicalTrials.gov. These are registry estimates, never confirmed readout dates; they can move as trials progress.
All registered studies
Every ClinicalTrials.gov study of this drug's own program, grouped by indication and phase. Research unlocks the studies themselves: titles, statuses, dates and featured-trial cross-links.