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Trial record 10 of 156 for:    "Skeletal Dysplasias"

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

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ClinicalTrials.gov Identifier: NCT03150069
Recruitment Status : Recruiting
First Posted : May 11, 2017
Last Update Posted : January 24, 2019
Sponsor:
Collaborator:
BioMarin Pharmaceutical
Information provided by (Responsible Party):
Nadia Ali, PhD, Emory University

Brief 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.


Condition or disease Intervention/treatment
Morquio Disease MPS - Mucopolysaccharidosis Other: Interview

Detailed Description:

The present study seeks to interview women with Morquio A and Morquio B syndrome themselves, to explore and discover their perspectives and concerns surrounding pregnancy and having biological children, the impact of the advent of ERT for Morquio A on their perspectives and concerns in comparison with the control group of Morquio B subjects for whom no ERT treatment exists, as well as what each group is being told by the medical community and others in their lives in this regard.

Interviews will be conducted by a health psychologist, either in-person or over the telephone. We will interview 1) those women with Morquio syndrome who are known to have been pregnant and 2) those women with Morquio syndrome who have not been pregnant and/or chose to adopt children. All information will be kept confidential, except as in accordance with Georgia law relating to reporting of child or elder abuse, suicidal and/or homicidal intent.

Data will be analyzed using MAXQDA 12.0 software to develop code which can be used to describe and compare the data using 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.


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Study Type : Observational
Estimated Enrollment : 12 participants
Observational Model: Cohort
Time Perspective: Cross-Sectional
Official Title: Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?
Actual Study Start Date : May 10, 2017
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019


Group/Cohort Intervention/treatment
Morquio A / Biological Mothers
Women with Morquio A who have biological children
Other: Interview
In-person or telephone in-depth interview

Morquio A / No Biological Children
Women with Morquio A who do not have biological children (both adoptive mothers and non-mothers)
Other: Interview
In-person or telephone in-depth interview

Morquio B / Biological Mothers
Women with Morquio B who have biological children
Other: Interview
In-person or telephone in-depth interview

Morquio B / No Biological Children
Women with Morquio B who do not have biological children (both adoptive mothers and non-mothers)
Other: Interview
In-person or telephone in-depth interview




Primary Outcome Measures :
  1. Completion of in-depth interview [ Time Frame: Approximately 1 hour ]
    Completion of in-depth interview



Information from the National Library of Medicine

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Ages Eligible for Study:   15 Years to 100 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
Women with Morquio syndrome, at least 15 years of age, English-speaking
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03150069


Contacts
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Contact: Nadia Ali, Ph.D. 404-778-8613 Nadia.Ali@emory.edu

Locations
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United States, Georgia
Emory University Recruiting
Decatur, Georgia, United States, 30033
Contact: Nadia Ali, Ph.D.    404-778-8613    Nadia.Ali@emory.edu   
Principal Investigator: Nadia Ali, Ph.D.         
Sponsors and Collaborators
Emory University
BioMarin Pharmaceutical

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Responsible Party: Nadia Ali, PhD, Principal Investigator, Emory University
ClinicalTrials.gov Identifier: NCT03150069     History of Changes
Other Study ID Numbers: IRB00088777
First Posted: May 11, 2017    Key Record Dates
Last Update Posted: January 24, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: IPD will not be shared with other researchers

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Nadia Ali, PhD, Emory University:
Pregnancy

Additional relevant MeSH terms:
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Osteochondrodysplasias
Mucopolysaccharidoses
Mucopolysaccharidosis IV
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases