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Complement Activation in the Lysosomal Storage Disorders

NCT04189601 · Status: WITHDRAWN · Type: OBSERVATIONAL

Last updated 2021-06-07

No results posted yet for this study

Summary

The lysosomal storage disorders (LSDs) are monogenic disorders associated with inflammation affecting multiple organs, and early death. Few treatments are available that can modify the disease course, and there is an urgent need to identify new steps in pathogenesis that can be targeted therapeutically. The complement system is novel and highly plausible as a primary driver of inflammation and cellular injury in the LSDs. This study assesses the complement activation state in patients with Fabry disease (FD), Gaucher disease (GD) and Niemann-Pick disease, type C (NPC), with comparison to healthy controls. This has the potential for immense clinical benefit through targeted complement inhibition across the full spectrum of lysosomal storage disorders, in which key pathophysiological processes including the inflammatory response to lysosomally 'stored' materials are shared.

Conditions

  • Fabry Disease
  • Gaucher Disease
  • Niemann-Pick Disease, Type C
  • Lysosomal Storage Diseases

Interventions

DIAGNOSTIC_TEST

Complement measurements

Blood and urine tests to assess the complement activation state

Sponsors & Collaborators

  • Sanofi

    collaborator INDUSTRY

  • Melbourne Health

    lead OTHER

Principal Investigators

  • Thomas D Barbour, MBBS · Melbourne Health

Eligibility

Min Age
17 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2020-09-30
Primary Completion
2021-02-26
Completion
2021-04-30

Countries

  • Australia

Study Locations

More Related Trials

Entities

Diseases
Companies

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 NCT04189601 on ClinicalTrials.gov