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Ambroxol as a Disease-modifying Treatment in GBA-PD

NCT05287503 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 65

Last updated 2025-08-14

No results posted yet for this study

Summary

The present multicenter, randomized, double-blind, placebo-controlled clinical trial will investigate whether the prolonged administration of high-dose oral Ambroxol over 52 weeks is safe, tolerable, able to change Glucocerebrosidase enzyme activity and alpha-synuclein levels in the central nervous system and, ultimately, to reduce the progression of cognitive decline and motor disability in 60 individuals with Parkinson's disease with mutations of the glucocerebrosidase gene (GBA1; OMIM 606463).

Participants will undergo clinical, biomarker blood and cerebrospinal fluid analysis, neuropsychological, neuroimaging assessment throughout the course of the study.

Conditions

  • Parkinson Disease
  • GBA Gene Mutation

Interventions

DRUG

Ambroxol Hydrochloride

Drug Ambroxol hydrochloride 200 mg plus excipients. The manufacturing process of the 200 mg Ambroxol hydrochloride tablets will be performed by wet granulation, drying, mixing and compression to the target final weight.

DRUG

Placebo

Excipients

Sponsors & Collaborators

  • IRCCS National Neurological Institute "C. Mondino" Foundation

    collaborator OTHER

  • University of Campania Luigi Vanvitelli

    collaborator OTHER

  • Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

    lead OTHER

Principal Investigators

  • Roberto Cilia · Fondazione IRCCS Istituto Neurologico Carlo Besta

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
21 Years
Max Age
80 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-03-09
Primary Completion
2024-12-15
Completion
2024-12-20

Countries

  • Italy

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05287503 on ClinicalTrials.gov