UnknownNot Applicable

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

United States30 participantsStarted 2015-05

Plain-language summary

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.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

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

Exclusion criteria

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

What they're measuring

Delineate T-, B- and NK cell fractions as well as dendritic cell and monocyte numbers and compare between patients undergoing ERT vs. SRT.

Timeframe: 2 Years

Trial details

NCT IDNCT02605603

SponsorLysosomal and Rare Disorders Research and Treatment Center, Inc.

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2020-02

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