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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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2 August 2023 |
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Main ID: |
NCT03204370 |
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Date of registration:
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07/06/2017 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Natural History of Atypical Morquio A Disease
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Scientific title:
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Natural History of Atypical Morquio A Disease: a 5-years Prospective Study in a Series of 9 Adult Patients Followed in a Single Expert Center |
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Date of first enrolment:
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February 1, 2018 |
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Target sample size:
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10 |
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Recruitment status: |
Recruiting |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT03204370 |
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Study type:
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Observational |
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Study design:
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Phase:
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Countries of recruitment
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France
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Contacts
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Name:
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Christophe Richez, MD, PhD |
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Address:
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Telephone:
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0033(0)556795556 |
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Email:
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christophe.richez@chu-bordeaux.fr |
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Affiliation:
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- MPS4A affected patients
- Height more than 1 m (atypical phenotypes)
- Treatment by ERT or not
- Followed in our expert center
- Having signed an inform consent form
- Being affiliated to a health insurance system
Exclusion Criteria:
- Patients affected by another disease
- Patients refusing to participate to the study
Age minimum:
18 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Mucopolysaccharidosis IV A
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Intervention(s)
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Drug: Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM]
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Primary Outcome(s)
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6-minute-walking test
[Time Frame: through study completion, an average of 5 years]
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Secondary ID(s)
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BMRN58492
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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