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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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16 December 2017 |
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Main ID: |
NCT02583152 |
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Date of registration:
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10/09/2015 |
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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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New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis
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Scientific title:
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Use of New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis |
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Date of first enrolment:
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November 2015 |
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Target sample size:
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50 |
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Recruitment status: |
Recruiting |
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URL:
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https://clinicaltrials.gov/show/NCT02583152 |
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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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N/A
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Countries of recruitment
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United Kingdom
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Contacts
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Name:
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Jane Ashworth |
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Address:
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Telephone:
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Email:
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jane.ashworth@mft.nhs.uk |
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Affiliation:
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Name:
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Jane Ashworth, MBChB, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Central Manchester Foundation Trust |
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Name:
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Jane Ashworth, MBChB |
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Address:
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Telephone:
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Email:
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Jane.Ashworth@cmft.nhs.uk |
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Affiliation:
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Adult and paediatric participants with MPS and corneal opacification will be
potentially eligible for this study, including those untreated, treated with previous
haematopetic stem cell transplant, and treated with ERT.
- Participants who have a confirmed diagnosis of mucopolysaccharosisis type I (Hurler,
Hurler/Scheie and Scheie), MPS type II (Hunter), type III (Sanfilippo) type IV
(Morquio) and type VI (MaroteauxLamy), type VII (Sly) will be potentially eligible. In
order to cooperate with the examinations, the participant needs to be able to hold
relatively still while seated at an instrument with a head rest and hold fixation for
several seconds for this reason participants over the age of 3 years will be eligible.
Exclusion Criteria:
- Those who are aged under 3 years or who have significant neurological involvement
which would influence understanding and/or cooperation.
Age minimum:
3 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Mucopolysaccharidoses
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Primary Outcome(s)
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Corneal densitometry scores in participants on treatment
[Time Frame: 60 months study period]
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Secondary Outcome(s)
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Corneal clouding score over time in patients on treatment.
[Time Frame: 60 months study period]
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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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