Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders
Status unconfirmed
Conditions studied: Lysosomal Storage Diseases
In brief
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.
Key facts
- Study ID
- NCT03812055
- Run by
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- People needed
- 50
- Starts
- 2018-07-06
- Expected to finish
- 2020-07-01
- Last updated by the study team
- 2019-01-22
Who can join
Age: any. Sex: any. Healthy volunteers: accepted.
You may qualify if…
- Subjects with
- confirmed diagnosis of any lysosomal storage disorder
- family members with history of lysosomal storage disorders
You may not qualify if…
- Subjects excluded from the study include those who:
- present with severe cognitive deficits impairing decision making
- are unable to or for whom it is medically unsafe to withdraw from their current medications, such as subjects on SSRI s and other psychoactive drugs. The subjects on SSRIs may be included in the study only with an approval from the prescribing physician to discontinue their medications temporarily for the study.
- are pregnant or nursing. All women of child bearing potential will undergo a pregnancy test.
- have a history of neurologic conditions such as stroke or any focal brain lesion that may result in parkinonian manifestations. Individuals with such MRI findings will be excluded from the study.
Where it is running
- LDRTC — Fairfax, Virginia, United States (enrolling)
Full record on ClinicalTrials.gov
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