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Small Molecule

Treatment for Lysosomal Storage Diseases

N/A
Waitlist Available
Led By Margarita M Ivanova, PhD
Research Sponsored by Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 months
Awards & highlights
No Placebo-Only Group

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.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Effect on autophagy-lysosomal pathway
Effect on enzyme activity
Effect on immune and inflammatory response
+2 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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Who is running the clinical trial?

Lysosomal and Rare Disorders Research and Treatment Center, Inc.Lead Sponsor
8 Previous Clinical Trials
100,286 Total Patients Enrolled
1 Trials studying Lysosomal Storage Diseases
100,000 Patients Enrolled for Lysosomal Storage Diseases
Margarita M Ivanova, PhDPrincipal InvestigatorLDRTC
1 Previous Clinical Trials
100,000 Total Patients Enrolled
1 Trials studying Lysosomal Storage Diseases
100,000 Patients Enrolled for Lysosomal Storage Diseases
Renuka Limgala, PhDPrincipal InvestigatorLDRTC
3 Previous Clinical Trials
100,180 Total Patients Enrolled
2 Trials studying Lysosomal Storage Diseases
100,080 Patients Enrolled for Lysosomal Storage Diseases
~7 spots leftby Oct 2025
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