A Phase 1/2 interventional study of Ambroxol Hydrochloride in Parkinson Disease and GBA Gene Mutation, sponsored by Agyany Pharma LTD. Terminated at 1 site in Israel. Open to participants aged 30 Years to 70 Years. Per ClinicalTrials.gov, last updated 2026-11-09.
Sponsored by Agyany Pharma LTD · Phase 1/2, Interventional, and Treatment
Sponsor's decision
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.
Parkinson disease (PD) is the second most common neurodegenerative disease affecting around 10 million people worldwide. It is a debilitating disorder that despite many years and billions of dollars research - there is still no cure and none of the therapeutic options truly reverse its manifestations. The realization that the relationship between Gaucher disease (GD) - the rare lysosomal storage disease - and PD, also exists in carriers of the disease (with a mono-allele mutation in glucocerebrosidase (GBA1)) has led to several attempts to develop new drugs not just for GBA-related PD, but also for PD at large. However, heretofore all pharmaceutical companies have focused on finding new formulations, mostly based on what Agyany believe is not targeting the underlying pathology, but in any case, will require several years before new drugs will reach the market. With the background of three decades working at the world's largest center for GD at Shaare Zedek Medical Center in Jerusalem, and with a different understanding of the pathological processes leading to PD among a significant number of GD patients and carriers, Agyany plans to begin clinical trials in newly diagnosed PD patients using existing generic drugs that would enable a short path for introducing novel therapeutic approach to GBA-related PD and potentially also for so called idiopathic PD (when no genetic cause is known).
Based on our understanding of the underlying mechanism of GBA1-related PD, on research done in animal models and on our own anecdotal experience, Agyany believe that pharmacological chaperons are the most reasonable therapeutic modality to achieve success. Since the misfolding of the mutant enzyme, glucocerebrosidase, is the same both in GBA1-related PD and GD, and that the ambroxol impact is the same as well, Agyany can extrapolate from the success of ambroxol to achieve reversibility of neuronopathic features (that heretofore were considered irreversible, and the best expectation was lack of deterioration), in neuronopathic GD (nGD), to potential success in GBA-related PD.
The plan is to first use generic formulations with a confirmed safety profile and repurpose their indication to PD.
4,573 studies on the registry are indexed under Parkinson Disease; 1,086 are open to participants now.
This study's enrollment of 46 is above the median of 40 across 3,353 interventional studies indexed under Parkinson Disease.
Browse Parkinson Disease studies →Agyany Pharma LTD is the lead sponsor of 2 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
For inclusion into the trial, subjects are required to fulfill all of the following criteria:
Newly diagnosed PD patients:
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:
iii. Hoehn and Yahr staged between I-II, inclusive.
iv. 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:
Exclusion Criteria:
Eligible subjects may not have any of the following exclusion criteria:
Use of dopaminergic treatment under these conditions:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Daily administration of Ambroxol in newly diagnosed GBA1 PD.
Drug: Ambroxol Hydrochloride
75mg slow release (SR), X16/day or 300mg X4/day oral capsules.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs)
The outcome measure involves the assessment of adverse events (AEs) experienced by study participants throughout the duration of the trial. Adverse events will be documented, and their severity will be graded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 5. This comprehensive evaluation aims to provide a clear understanding of the safety profile of the intervention by specifically measuring and reporting the incidence and severity of treatment-emergent adverse events.
Time frame: 12 months
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Criteria for potentially clinically significant abnormalities (PCSA): Hemoglobin: less than or equal to (\<=) 115 grams per liter (g/L) (Male\[M\]) or \<=95 g/L (Female\[F\]), greater than or equal to (\>=) 185 g/L (M) or \>=165 g/L (F), Decrease from baseline (DFB) \>=20 g/L; Hematocrit: \<=0.37 volume/volume (v/v) (M) or \<=0.32 v/v (F), \>=0.55 v/v (M) or \>=0.5 v/v (F); Red blood cells (RBC): \>=6 Tera/L; Platelets: less than (\<) 100 Giga/L, \>=700 Giga/L; White blood cells (WBC): \<3.0 Giga/L (Non-Black \[NB\]) or \<2.0 Giga/L (Black \[B\]), \>=16.0 Giga/L; Neutrophils: \<1.5 Giga/L (NB) or \<1.0 Giga/L (B); Lymphocytes: \<lower limit of normal (LLN), greater than (\>) 4.0 Giga/L; Monocytes: \<LLN, \>0.7 Giga/L; Basophils: \>0.1 Giga/L; Eosinophils: \>0.5 Giga/L or \>upper limit of normal (ULN) (if ULN \>=0.5 Giga/L).
Time frame: 12 months
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Biochemistry (Metabolic, Electrolytes, Renal and Liver Function)
Criteria for PCSA: Alanine Aminotransferase (ALT): \>3 ULN, \>5 ULN; Aspartate aminotransferase (AST): \>3 ULN; Alkaline phosphatase: \>1.5 ULN; Total Bilirubin: \>1.5 ULN; ALT and Bilirubin: \>3 ULN and \>2 ULN; Direct Bilirubin and Bilirubin: \>35% and \>1.5 ULN. Criteria for PCSA: Creatinine: \>=150 micromoles per liter (mcmol/L) (adults), \>=30% change from baseline, \>=100% change from baseline; Blood urea nitrogen: \>=17 millimoles (mmol)/L. Criteria for PCSA: Glucose: \<=3.9 mmol/L and \<LLN; \>=11.1 mmol/L (unfasted \[unfas\]) or \>=7 mmol/L (fasted \[fas\]); Albumin: \<=25 g/L. Criteria for PCSA: Sodium: \<=129 mmol/L, \>=160 mmol/L; Potassium: \<3 mmol/L, \>=5.5 mmol/L and Chloride: \<80 mmol/L, \>115 mmol/L.
Time frame: 12 months
Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities
Criteria for PCSA: Systolic blood pressure (SBP) supine: \<=95 millimeters of mercury (mmHg) and DFB \>=20 mmHg; \>=160 mmHg and increase from baseline (IFB) \>=20 mmHg; Diastolic blood pressure (DBP) supine: \<=45 mmHg and DFB \>=10 mmHg; \>=110 mmHg and IFB \>=10 mmHg; SBP (Orthostatic): \<=-20 mmHg; DBP (Orthostatic): \<=-10 mmHg; Heart rate (HR) supine: \<=50 beats per minute (bpm) and DFB \>=20 bpm; \>=120 bpm and IFB \>=20 bpm; Weight: \>=5% DFB; \>=5% IFB.
Time frame: 12 months
Number of Participants With Abnormal Physical Examination Findings
Routine physical examinations will be conducted to assess participants for any physical manifestations of adverse effects or safety concerns. In the final report the number of participants with new abnormal physical examination findings will be reported.
Time frame: 12 months
Number of Participants With Significant Change in Transcranial Ultrasonography (TCS) of Substantia Nigra Region
Transcranial Ultrasonography detects the area of hyper-echogenicity in the region of substantia nigra (values greater than 0.2 cm square are considered abnormal. Improvement from baseline will be assessed and considered significant if greater than 15% from baseline.
Time frame: 12 months
Difference of dopamine turnover rate measured by Fluoro-Dopa-PET in the putamen between baseline and after 12 months of treatment.
Change from baseline to end of study (EOS) in F-DOPA imaging will be qualitative as assessed by independent expert in nuclear medicine in a central imaging center.
Time frame: 12 months
Changes in motor evaluations
Using: (Video-recorded unified Parkinson's disease rating scale (MDS-UPDRS) Part II and III.
Time frame: 12 months
Changes in motor evaluations
Perdue pegboard
Time frame: 12 months
Changes in motor evaluations
Trail Making Test (TMT)
Time frame: 12 months
Change in cognitive and mental impairment
Montreal Cognitive Assessment (MoCa)
Time frame: 12 months
Change in cognitive and mental impairment
NeuroTrax
Time frame: 12 months
Change in cognitive and mental impairment
Clinical Global Impression Scale (CGI)
Time frame: 12 months
Change in cognitive and mental impairment
Hooper Visual Organization Test (VOT)
Time frame: 12 months
Change in cognitive and mental impairment
Beck Depression inventory (BDI)
Time frame: 12 months
Change in sleep behavior
REM sleep behavior disorder (RBD)
Time frame: 12 months
Change in sleep behavior
Epworth sleepiness scale (ESS)
Time frame: 12 months
Changes in sensory and autonomic assessments
Using the following tests: the BIT University of Pennsylvania brief Smell Identification Test (UPSIT)
Time frame: 12 months
Changes in sensory and autonomic assessments
Constipation questionnaire
Time frame: 12 months
Changes in sensory and autonomic assessments
Orthostatic hypotension (OH)
Time frame: 12 months
Changes in sensory and autonomic assessments
Color discrimination.
Time frame: 12 months
Plan to share: No — There is no plan to share such data.
Ran from Oct 2023 to primary completion in Mar 2026, 29 months.
Since we started tracking on Sep 25, 2026, the dates moved once, on Nov 9, 2026: primary completion Feb 2025 → Mar 2026 and completion Apr 2025 → Apr 2026.
Earlier changes are on the record history on ClinicalTrials.gov ↗
How these figures are worked out, and which studies are left out of them.
Sponsor's decision
Sponsor's decision
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is terminated, as verified in May 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Ambroxol
Agyany Pharma LTD