Lysosomal and Rare Disorders Research and Treatment Center, Inc. | Fairfax, VA
Site insights
Top conditions
Top treatments
Curious how this site stacks up to its peers?
Data sourced from clinicaltrials.gov
Work at this site?
CTV for research professionals allows users to enhance their site’s profile, add contact information, and more.
Active trials
This Phase 1/2 first-in-human (FIH) study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of HM15421 in patient...
An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects...
An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Amenable GLA Variants and Severe...
This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat ve...
This is a 12-month, parallel treatment, Phase 3, double-blind, randomized, placebo controlled study to evaluate the effect of venglustat on neuropath...
A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. This study includes a sub-study e...
The purpose of this study is to evaluate the pharmacokinetics and safety of Immune Globulin Intravenous (Human) GC5107 in pediatric subjects with Pri...
Study ABX1100-1001 is a first-in-human (FIH), phase 1 study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of a single asc...
Part 1: Biomarker evaluation/screening phasePrimary Objectives:* Evaluate cerebrospinal fluid (CSF) biomarkers in adult Gaucher disease Type 3 (GD3)...
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treat...
Trial sponsors
Clinical trials
Research sites
Resources
Legal