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High-Dose Ambroxol in GBA1-Related Parkinson

NCT06193421 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 40

Last updated 2024-02-08

No results posted yet for this study

Summary

Parkinson's disease (PD), affecting 10 million people globally, lacks a cure, and current therapies only manage symptoms. A link between Gaucher disease (GD) and PD, particularly in carriers of glucocerebrosidase (GBA1) mutations, has sparked interest in developing new drugs. Despite pharmaceutical companies focusing on formulations, progress is slow. Agyany, with decades of experience in GD research, plans clinical trials using existing generic drugs for GBA-related PD and idiopathic PD. Their approach targets the misfolded enzyme glucocerebrosidase with pharmacological chaperons, inspired by success in GD using ambroxol. The strategy aims to provide a quicker path to novel therapeutic options for PD.

Conditions

  • Parkinson Disease
  • GBA Gene Mutation

Interventions

DRUG

Ambroxol Hydrochloride

75mg slow release (SR), X16/day or 300mg X4/day oral capsules.

Sponsors & Collaborators

  • Agyany Pharma LTD

    lead INDUSTRY

Principal Investigators

  • Ari Zimran, MD · Agyany Pharma LTD

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
30 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-10-24
Primary Completion
2025-02-27
Completion
2025-04-30

Countries

  • Israel

Study Locations

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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 NCT06193421 on ClinicalTrials.gov