CompletedNot Applicable

A One Visit Follow Up of Adults With Fabry Disease Who Started Long-term Enzyme Replacement Therapy As Children

United States5 participantsStarted 2018-11-10

Plain-language summary

The objective of this study is to obtain follow up data on a cohort of well-studied patients with Fabry disease who have been on ERT since childhood for a total of about 15 years.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Patients who participated in TKT029 and who are willing and able to come to Dallas for 1 visit for standard of care testing.
✓. Sign the protocol informed consent form
✓. Have been on continuous commercial ERT since TKT029 has ended

Exclusion criteria

✕. Patients who are unable to understand the nature, scope, and possible consequences of the study.
✕. Patient does not give his written informed consent to participate in this study
✕. Patient is unable to comply with the protocol, e.g., uncooperative with protocol schedule, refusal to agree to all of the study procedures.
✕. Patient has been off ERT for an extended period of time as assessed by the investigator.

What they're measuring

estimated Glomerular Filtration Rate (eGFR)

Timeframe: Study involves one visit only - assessed Baseline Visit

Trial details

NCT IDNCT04002531

SponsorBaylor Research Institute

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2019-12-13

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