NCT06193421TerminatedPhase 1/2
High-Dose Ambroxol in GBA1-Related Parkinson
An Open-Label Pilot Study for Assessing the Safety and Efficacy of High-Dose Ambroxol (HDA) in Newly Diagnosed GBA1 Parkinson Disease (PD)
Source: ClinicalTrials.gov · Last updated 9 Nov 2026
Reason given: Sponsor's decision
Brief summary (registry, in English)
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 glucocerebrosidase (GBA1) mutations, has sparked interest in developing new drugs. Despite pharmaceutical companies focusing on formulations, progress is slow. Agyany, with decades of experience in GD research, plans clinical trials using existing generic drugs for GBA-related PD and idiopathic PD. Their approach targets the misfolded enzyme glucocerebrosidase with pharmacological chaperons, inspired by success in GD using ambroxol. The strategy aims to provide a quicker path to novel therapeutic options for PD.
- Status (registry)
- Terminated
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 46 (actual)
- First submitted
- 7 Nov 2023
- Start date
- 24 Oct 2023
- Primary completion date
- 30 Mar 2026
- Lead sponsor
- Agyany Pharma LTD
- Diseases
- Parkinson disease
- NCT number
- NCT06193421
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
- 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.
Other illness
- 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.
- Medical history of psychosis.
Age
- Pregnancy or lactation; female subjects of a childbearing age who are unwilling to use contraceptive measures.
Other
- Eligible subjects may not have any of the following exclusion criteria:
Who the study is looking for
- 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:
- Hoehn and Yahr staged between I-II, inclusive.
- 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.
Age in the registry: 30 years to 70 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Shaare Zedek Medical Center | Jerusalem | Israel | Unknown status |
The registry text, word for word
This page is information, not medical advice. Discuss any trial with your doctor.