NCT04388969 is a clinical trial in Gaucher Disease, sponsored by Shaare Zedek Medical Center.

Who is sponsoring NCT04388969?

NCT04388969 is sponsored by Shaare Zedek Medical Center.

What condition does NCT04388969 study?

NCT04388969 studies Gaucher Disease, Parkinson Disease, GBA Gene Mutation.

Is NCT04388969 still recruiting?

NCT04388969 is currently status unknown. Last updated 14 February 2023.

Last reviewed 2023-02-14 · How we verify

NCT04388969

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

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

Status unknown Last updated 14 February 2023

What this trial tests

trial in Gaucher Disease in 300 participants. Status unknown.

Timeline

6 May 2020

Primary endpoint
31 December 2023

31 January 2024

Quick facts

Lead sponsor	Shaare Zedek Medical Center
Status	Status unknown
Study type	OBSERVATIONAL
Enrollment	300
Start date	6 May 2020
Primary completion	31 December 2023
Estimated completion	31 January 2024
Sites	3 locations across Israel

Conditions studied

Gaucher Disease — all drugs for Gaucher Disease →
Parkinson Disease — all drugs for Parkinson Disease →
GBA Gene Mutation — all drugs for GBA Gene Mutation →

Sponsor

Shaare Zedek Medical Center

Who can join

Under 100, any sex, with Gaucher Disease or Parkinson Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Therapeutic Approaches in Lysosomal Storage Diseases.
Fernández-Pereira C, San Millán-Tejado B, Gallardo-Gómez M, Pérez-Márquez T, et al · · 2021 · cited 43× · PMID 34944420 · DOI 10.3390/biom11121775
Protein clearance strategies for disease intervention.
Hommen F, Bilican S, Vilchez D. · · 2022 · cited 30× · PMID 34689261 · DOI 10.1007/s00702-021-02431-y
<i>GBA1</i>-Associated Parkinson's Disease Is a Distinct Entity.
Skrahin A, Horowitz M, Istaiti M, Skrahina V, et al · · 2024 · cited 22× · PMID 39000225 · DOI 10.3390/ijms25137102
Lysosomal dysfunction in α-synuclein pathology: molecular mechanisms and therapeutic strategies.
Dai L, Liu M, Ke W, Chen L, et al · · 2024 · cited 17× · PMID 39223418 · DOI 10.1007/s00018-024-05419-5
A review on Gaucher disease: therapeutic potential of β-glucocerebrosidase-targeted mRNA/saRNA approach.
Feng S, Rcheulishvili N, Jiang X, Zhu P, et al · · 2024 · cited 12× · PMID 38617529 · DOI 10.7150/ijbs.87741
Exploring the efficacy and safety of Ambroxol in Gaucher disease: an overview of clinical studies.
Mohamed FE, Al-Jasmi F. · · 2024 · cited 11× · PMID 38414738 · DOI 10.3389/fphar.2024.1335058
Lysosomal Dysfunction: Connecting the Dots in the Landscape of Human Diseases.
Uribe-Carretero E, Rey V, Fuentes JM, Tamargo-Gómez I. · · 2024 · cited 8× · PMID 38248465 · DOI 10.3390/biology13010034
The ANeED study - ambroxol in new and early dementia with Lewy bodies (DLB): protocol for a phase IIa multicentre, randomised, double-blinded and placebo-controlled trial.
Chwiszczuk LJ, Breitve MH, Kirsebom BB, Selnes P, et al · · 2023 · cited 8× · PMID 37304077 · DOI 10.3389/fnagi.2023.1163184

Verify or expand the search:

Other recruiting trials for Gaucher Disease

Currently open trials in the same condition.

NCT06528080 — A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease · EARLY_PHASE1 · active not recruiting
NCT06573723 — Institutional Registry of Rare Diseases · recruiting
NCT06539169 — FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases · recruiting
NCT05843552 — Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease · recruiting
NCT05487599 — A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) · Phase 1, PHASE2 · recruiting

Other Shaare Zedek Medical Center trials

Trials by the same sponsor.

NCT05331209 — Acupuncture With/Without Self-acupressure for Post-oophorectomy Hot Flashes in BRCA Carriers · NA · not yet recruiting
NCT06070753 — A Prospective, Trial About Safety and Efficacy of Combined Treatment With Cerebrolysin in Acute Ischemic Hemispheric Str · NA · unknown
NCT05841953 — The Use of Electrical Stimulation to Increase Anal Pressures · NA · unknown
NCT06777706 — Open Label Study to Assess Safety & Efficacy of QD for Induction of Remission in Pediatric Patients with UC · NA · completed
NCT05838105 — Two Different Types of Luteal Phase Support in Natural Cycle Frozen Embryo Transfer and Its Effect on Pregnancy Rates · NA · unknown

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04388969 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Shaare Zedek Medical Center
Last refreshed: 14 February 2023

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04388969.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing