SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease

Status unconfirmed

Conditions studied: Gaucher Disease

In brief

This project is expected to elucidate role of different therapeutic interventions: SRT in comparison to ERT in influencing immune aspects of GD pathology, as well as bone involvement.

Key facts

Study ID

NCT02605603

Run by

Lysosomal and Rare Disorders Research and Treatment Center, Inc.

People needed

30

Starts

2015-05-01

Expected to finish

2020-05-01

Last updated by the study team

2019-08-13

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Confirmed diagnosis of Gaucher disease
• Subjects on SRT using Cerdelga ( must fulfil the pharmacogenomics criteria for Cerdelga)

You may not qualify if…

• Unconfirmed diagnosis of Gaucher disease
• Subject or guardian unable to provide consent
• Any chronic immunosuppressive state or therapy

Where it is running

• O&O Alpan, LLC — Fairfax, Virginia, United States

Full record on ClinicalTrials.gov

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