Morquio DiseaseMorqPregNCT03150069
Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?
An observational study of Morquio Disease and MPS - Mucopolysaccharidosis, run by Emory University at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 15 to 100 years, women onlyHealthy volunteers can take part
- Conditions the study is for
- Morquio Disease, MPS - Mucopolysaccharidosis
- What is being tested?
- Interview
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- 10 May 2017to 17 Aug 2018
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 15 to 100 years
- Women only
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Women with Morquio syndrome, at least 15 years of age, English-speaking
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the 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.
- Conditions
- Morquio Disease, MPS - Mucopolysaccharidosis
- Being tested
- Interview
- Keywords
- Pregnancy
- Medical subject headings
- Mucopolysaccharidosis IV, Mucopolysaccharidoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
InterviewOther
In-person or telephone in-depth interview
| Study group | Role | What they receive |
|---|---|---|
| Morquio A / Biological Mothers | Women with Morquio A who have biological children Receives: Other: Interview | |
| Morquio A / No Biological Children | Women with Morquio A who do not have biological children (both adoptive mothers and non-mothers) Receives: Other: Interview | |
| Morquio B / Biological Mothers | Women with Morquio B who have biological children Receives: Other: Interview | |
| Morquio B / No Biological Children | Women with Morquio B who do not have biological children (both adoptive mothers and non-mothers) Receives: Other: Interview |
What is being measured
The primary outcome is the main question the study is designed to answer.
Completion of in-depth interview
Completion of in-depth interview
Measured: Approximately 1 hour
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 May 2017
- Study start10 May 2017
- Primary completion17 Aug 2018
- Study completion17 Aug 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Morquio DiseaseStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- BioMarin Pharmaceutical
- Responsible party
- Principal investigator, Nadia Ali, PhD, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03150069
- Sponsor's protocol ID
- IRB00088777
- Acronym
- MorqPreg
- Status last verified
- Jan 2021
- First submitted
- 10 May 2017
- Last updated
- 20 Jan 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?
- ClinicalTrials.gov ID
- NCT03150069
- Acronym / sponsor ID
- MorqPreg / IRB00088777
- Conditions
- Morquio Disease, MPS - Mucopolysaccharidosis
- Keywords
- Pregnancy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- BioMarin Pharmaceutical
- Study start
- 10 May 2017 (actual)
- Primary completion
- 17 Aug 2018 (actual)
- Study completion
- 17 Aug 2018 (actual)
- First posted
- 11 May 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Morquio A / Biological Mothers | – | Other: InterviewWomen with Morquio A who have biological children |
| Morquio A / No Biological Children | – | Other: InterviewWomen with Morquio A who do not have biological children (both adoptive mothers and non-mothers) |
| Morquio B / Biological Mothers | – | Other: InterviewWomen with Morquio B who have biological children |
| Morquio B / No Biological Children | – | Other: InterviewWomen with Morquio B who do not have biological children (both adoptive mothers and non-mothers) |
Who may qualify
- Ages
- 15 to 100 years
- Sex
- Female only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Women with Morquio syndrome, at least 15 years of age, English-speaking
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| InterviewIn-person or telephone in-depth interview | Other | Morquio A / Biological Mothers, Morquio A / No Biological Children, Morquio B / Biological Mothers, Morquio B / No Biological Children |
Primary outcome
Completion of in-depth interview
Time frame: Approximately 1 hour
Completion of in-depth interview
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jan 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Morquio Disease and MPS - Mucopolysaccharidosis, run by Emory University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Completion of in-depth interview (time frame: Approximately 1 hour). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2021.