Pregnancy With Morquio Syndrome – What Are Patients’ Perspectives and Has ERT Changed Them?

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Trial Status Completed

Trial Identifier

NCT03150069

Condition

MPS IVA, Morquio Disease, MPS - Mucopolysaccharidosis

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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.

Eligibility Criteria

Birth Sex

Female

Age

15 Years to 100 Years

Healthy Volunteers

Yes

Drug/Treatment:

Interview

Study Type:

Observational

Number of Participants:

18

Study Started:

2017-05-10

Study Updated:

2021-01-20

Trial Locations

  • Emory University

    Decatur, Georgia, United States

Inclusion Criteria

1. 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.
2. Subject is at least 15 years old.
3. Subject is female.
4. Subject is fluent enough in English to complete in-depth interview with PI.

Exclusion Criteria

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

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