CompletedNot Applicable

Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?

United States18 participantsStarted 2017-05-10

Plain-language summary

The present study seeks to interview women with Morquio A and Morquio B syndrome, to explore their concerns surrounding pregnancy and the impact of ERT on their perspectives, in comparison with the control group of Morquio B subjects for whom no ERT treatment exists. Interviews will be conducted by a health psychologist, in-person or over the telephone. Data will be analyzed using MAXQDA 12.0 software and Grounded Theory. Differences in thematic trends between Morquio A subjects, for whom treatment exists, and a control group of Morquio B subjects, for whom there is no treatment, will be compared.

Who can participate

Age range15 Years – 100 Years

SexFEMALE

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Inclusion criteria

✓. Documented clinical diagnosis of either MPS IVA or MPS IVB, based on clinical signs and symptoms, documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis.
✓. Subject is at least 15 years old.
✓. Subject is female.
✓. Subject is fluent enough in English to complete in-depth interview with PI.

Exclusion criteria

✕. Patient has a clinically significant disease other than Morquio which would confound the effects of Morquio upon study variables.
✕. Any condition that, in the view of the Investigator, places the patient at high risk of poor compliance or of not completing the study.

What they're measuring

Completion of in-depth interview

Timeframe: Approximately 1 hour

Trial details

NCT IDNCT03150069

SponsorEmory University

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2018-08-17

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