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New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis

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Trial Status Unknown

Trial Identifier

NCT02583152

Condition

Mucopolysaccharidoses

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

Mucopolysaccharidoses (MPS) are currently treated with Enzyme replacement therapy and Bone Marrow Transplantation (BMT). No current evidence on the effectiveness on these therapies on the eye in this systemic disease is avalible. Using new imaging techniques; previously subjective data can be quantified and compared to determine if there is an improvment in the vision of patients with MPS.

Eligibility Criteria

Birth Sex

All

Age

From 3 Years

Healthy Volunteers

No

Study Type:

Observational

Number of Participants:

50

Study Started:

2015-11

Study Updated:

2017-11-28

Trial Locations

  • Manchester Royal Eye Hospital

    Manchester, , United Kingdom

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

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