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These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 9, 2026, 4:00 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–10 of 10 matching trials from the live ClinicalTrials.gov search.

Conditions: Gaucher Disease Type 1
Interventions: Not listed
Lead sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.; Other
Eligibility: 16 Years to 90 Years
Enrollment: 40 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019 – 2020
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Fabry Disease
Interventions: Not listed
Lead sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.; Other
Eligibility: 18 Years to 80 Years
Enrollment: 40 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2025
U.S. locations: 2
States / cities: Rockville, Maryland • Fairfax, Virginia

Conditions: Lysosomal Storage Diseases
Interventions: Enzyme assay and molecular sequencing; Diagnostic Test
Lead sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.; Other
Eligibility: Not listed
Enrollment: 100,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2019
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Lysosomal Storage Diseases
Interventions: Not listed
Lead sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.; Other
Eligibility: Not listed
Enrollment: 50 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2020
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Gaucher Disease, Bone Diseases
Interventions: Not listed
Lead sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.; Other
Eligibility: 5 Years to 21 Years
Enrollment: 20 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2025
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Sanfilippo Syndrome, MPS3
Interventions: Not listed
Lead sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.; Other
Eligibility: 5 Years to 99 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2024
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Sanfilippo Syndrome, MPS3
Interventions: Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day; Drug
Lead sponsor: Ozlem Goker-Alpan; Other
Eligibility: 18 Years to 99 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Fabry
Interventions: biomarkers; Diagnostic Test
Lead sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.; Other
Eligibility: 18 Years to 70 Years
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2022
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Parkinson Disease, Gaucher Disease
Interventions: Not listed
Lead sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.; Other
Eligibility: 50 Years and older
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2020
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Gaucher Disease
Interventions: Not listed
Lead sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.; Other
Eligibility: 18 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2020
U.S. locations: 1
States / cities: Fairfax, Virginia

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