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Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders

NCT03812055 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 50

Last updated 2019-01-22

No results posted yet for this study

Summary

The purpose of this study is to evaluate the effect of small molecule therapy in primary cells derived from patients with lysosomal storage disease. The study will focus on activity of small molecules, in terms of measurements enzymes activity and level of substrates accumulations. Also, the effects of small molecules on cell function, including autophagy-lysosomal pathways, metabolism, mitochondrial function and immune reaction will be investigated.

Conditions

  • Lysosomal Storage Diseases

Sponsors & Collaborators

  • Lysosomal and Rare Disorders Research and Treatment Center, Inc.

    lead OTHER

Principal Investigators

  • Margarita M Ivanova, PhD · LDRTC

  • Ozlem Goker-Alpan, MD · LDRTC

  • Renuka Limgala, PhD · LDRTC

Eligibility

Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2018-07-06
Primary Completion
2020-07-31
Completion
2020-07-31

Countries

  • United States

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 NCT03812055 on ClinicalTrials.gov