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Treatment for Lysosomal Storage Disorders

N/A
Waitlist Available
Led By Ozlem Goker-Alpan, MD
Research Sponsored by O & O Alpan LLC
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 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial aims to study how different genetic mutations that cause lysosomal storage diseases (LSDs) result in different severity of the disease, age of onset, and clinical presentation.

Eligible Conditions
  • Lysosomal Storage Disorders

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Correlating genetic mutations with clinical signs and symptoms
Secondary study objectives
Associated Immune Pathophysiology

Awards & Highlights

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

Find a Location

Who is running the clinical trial?

O & O Alpan LLCLead Sponsor
6 Previous Clinical Trials
20,445 Total Patients Enrolled
1 Trials studying Lysosomal Storage Disorders
20,000 Patients Enrolled for Lysosomal Storage Disorders
Ozlem Goker-Alpan, MDPrincipal InvestigatorLysosomal & Rare Disorders Research & Treatment Center (LDRTC)
9 Previous Clinical Trials
100,300 Total Patients Enrolled
Renuka Limgala, PhDPrincipal InvestigatorLDRTC
3 Previous Clinical Trials
100,150 Total Patients Enrolled
~7 spots leftby Oct 2025
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