NCT04388969 World Data on Ambroxol for Patients With GD and GBA Related PD
| NCT ID | NCT04388969 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Shaare Zedek Medical Center |
| Condition | Gaucher Disease |
| Study Type | OBSERVATIONAL |
| Enrollment | 300 participants |
| Start Date | 2020-05-06 |
| Primary Completion | 2030-10-31 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 300 participants in total. It began in 2020-05-06 with a primary completion date of 2030-10-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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
Inclusion Criteria: * patients with Gaucher disease type 1,2 or 3(a,b,c). * patients with GBA-related Parkinson disease. Exclusion Criteria: * None.
Contact & Investigator
Ari Zimran, MD
PRINCIPAL INVESTIGATOR
Shaare Zedek Medical Center
Frequently Asked Questions
Who can join the NCT04388969 clinical trial?
This trial is open to participants of all sexes, up to 100 Years, studying Gaucher Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT04388969 currently recruiting?
Yes, NCT04388969 is actively recruiting participants. Contact the research team at joleenist@szmc.org.il for enrollment information.
Where is the NCT04388969 trial being conducted?
This trial is being conducted at Jerusalem, Israel, Jerusalem, Israel, Jerusalem, Israel.
Who is sponsoring the NCT04388969 clinical trial?
NCT04388969 is sponsored by Shaare Zedek Medical Center. The principal investigator is Ari Zimran, MD at Shaare Zedek Medical Center. The trial plans to enroll 300 participants.