World Data on Ambroxol for Patients With GD and GBA Related PD

ConditionsGaucher Disease Parkinson Disease GBA Gene Mutation

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

AgeUp to 100

SponsorShaare Zedek Medical Center

About this trial

Ambroxol hydrochloride is an oral mucolytic drug available over-the-counter for many years as cough medicine. In 2009 it was found to also act as a pharmacological chaperone (PC) for mutant glucocerebrosidase, albeit in a several-fold higher dose. Unfortunately, due to its low cost, there have been no pharma-driven clinical trials to establish the use of ambroxol. Thus, data are needed on the safety and efficacy of ambroxol for patients with Gaucher disease (GD).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

patients with Gaucher disease type 1,2 or 3(a,b,c).

patients with GBA-related Parkinson disease.

Disqualifiers

None.

Trial population

Patients with Gaucher disease type 1,2,3 (a,b,c) and patients with GBA-related Parkinson disease.

Trial design

Design model

Cohort

Time perspective

Retrospective

Treatments tested in this trial

Not listed

Trial groups

300 Participants

are grouped into 2 trial groups

Group A: Gaucher disease patients

Group B: GBA carriers with Parkinson disease

Trial outcomes

Primary outcomes

Establishing a registry to be used as a reference for patients,parents and physicians, who consider using Ambroxol

With no specific treatment to offer for patients (particularly children and young adults) with nGD, and with all the above-mentioned encouraging preliminary reports, in our current era of rapidly spreading information (in many meetings worldwide, in the internet and via the social media) individual patients in many countries have started to receive off-label Ambroxol by their treating physicians and a few case reports have been published from Korea , Israel and Canada . In order to upscale the level of evidence from anecdotal reports to an observational study, we herein suggest to establish a specific IIR, wherein the "R" stands for a "Registry", that may be used as a reference for patients , parents and physicians, who consider using Ambroxol.

Time frame

10 years

Safety Outcome

By reporting adverse events

Time frame

10 years

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Shaare Zedek Medical Center

Lead sponsor

Source ClinicalTrials.gov

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