World Data on Ambroxol for Patients With GD and GBA Related PD
NCT ID: NCT04388969
Last Updated: 2023-02-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
300 participants
OBSERVATIONAL
2020-05-06
2024-01-31
Brief Summary
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Detailed Description
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Ambroxol hydrochloride is an oral mucolytic drug, available over-the-counter (OTC) for many years as cough medicine. In 2009 it was found (in-vitro) to also act as a pharmacological chaperone (PC) for mutant glucocerebrosidase (GCase), 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.
In an attempt to provide the proof of concept to the potential use of Ambroxol as PC for patients with GD, the Gaucher Clinic at Shaare Zedek Medical Center in Jerusalem, Israel, has treated 12 adult patients with GD1, all untreated with any specific treatment for GD \[whether ERT or substrate reduction therapy (SRT),\] with Ambroxol at the OTC dose of 150mg/day for a period of 6 months; this was not a formal clinical trial, but rather an audit of a series of patients receiving this drug via the employment of the Israeli Ministry of Health Form 29c to prescribe it for an off-label indication. A single patient, the thinnest of the group with a BMI of 17.1, achieved a robust response relative to baseline similar to ERT.
In 2016, a group from Japan has demonstrated impressive neurological response using high-dose Ambroxol (a marked decrease in seizure frequency, improvement of myoclonus, ability to walk for a bedridden patient, improvement in the latency of I-V waves and threshold of ABR) with dramatic change in daily activities and quality of life (QOL). These encouraging results, that were confirmed in a larger series, yet unpublished, have further led to the conduction of an investigator-initiated-research (IIR) clinical trial in London by Prof A Schapira, which has recently been published (January 13, 2020 JAMA Neurology), wherein newly diagnosed Parkinson patients received high dose Ambroxol, with good safety results
Conditions
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Study Design
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COHORT
RETROSPECTIVE
Study Groups
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Gaucher disease patients
Patients with type 1,2,3 Gaucher disease.
No interventions assigned to this group
GBA carriers with Parkinson disease
Patients with Parkinson disease GBA related (carriers)
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* patients with GBA-related Parkinson disease.
Exclusion Criteria
100 Years
ALL
No
Sponsors
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Shaare Zedek Medical Center
OTHER
Responsible Party
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Ari Zimran
Principal Investigator
Principal Investigators
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Ari Zimran, MD
Role: PRINCIPAL_INVESTIGATOR
Shaare Zedek Medical Center
Locations
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Shaare Zedek Medical Center
Jerusalem, Please Select..., Israel
Shaare Zedek Medical Center
Jerusalem, , Israel
Shaare Zedek Medical Center
Jerusalem, , Israel
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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0097-20-SZMC
Identifier Type: -
Identifier Source: org_study_id
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