Status:
COMPLETED
Core Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis II
Lead Sponsor:
Manchester University NHS Foundation Trust
Conditions:
Mucopolysaccharidosis Type II
Eligibility:
All Genders
Up to 100 years
Brief Summary
The Mucopolysaccharidoses (MPS) are a family of genetic metabolic disorders, caused by specific enzyme deficiencies which result in accumulation of partially degraded glycosaminoglycans (GAGs) within ...
Eligibility Criteria
Inclusion
- Inclusion criteria:
- Confirmed MPS II diagnosis.
- Aged between 12 and 25 years old.
- Parents/ main care givers of child with MPS Type II.
- Adults aged 18 or over.
- Clinicians responsible for direct care and management of at least 2 pediatric MPS Type II patients in the last 12 months.
- Scientists who have worked with MPS Type II within the last 5 years
- Exclusion criteria:
- Unconfirmed MPS Type II diagnosis.
- Aged below 12 and above 25 years old.
- Not directly involved in a day to day care.
- Clinicians who only occasionally look after pediatric MPS Type II patients.
- Scientists who have not worked with MPS Type II within the last 5 years
Exclusion
Key Trial Info
Start Date :
September 28 2020
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
June 30 2021
Estimated Enrollment :
46 Patients enrolled
Trial Details
Trial ID
NCT06022380
Start Date
September 28 2020
End Date
June 30 2021
Last Update
September 1 2023
Active Locations (1)
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1
Manchester University NHS Foundation Trust
Manchester, Lancashire, United Kingdom, M13 9WL