Status:
COMPLETED
Lifetime Impact of Achondroplasia Study in Europe-LIAISE
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Achondroplasia
Eligibility:
All Genders
5-70 years
Brief Summary
Observational study looking at the burden of illness in achondroplasia subjects aged 5-70. The study will include a 5 year review of historical clinical data as well as a single point collection of qu...
Detailed Description
This is a multinational, epidemiological, observational, retrospective, cross-sectional study of individuals with achondroplasia (subjects). This study will be conducted at up to approximately 20 site...
Eligibility Criteria
Inclusion
- Individuals with a documented diagnosis of achondroplasia based on:
- Genetic confirmation of achondroplasia and/or
- Clinical diagnosis of achondroplasia (clinical examination or radiological assessment)
- ≥ five years of age at the time of enrolment
- Has the cognitive and linguistic capacities necessary to complete questionnaires in the language of his/her country (and/or parents/legally acceptable representatives, as applicable)
- Agrees to participate in the study and has read, understood, completed and signed:
- Informed Consent Form (ICF) - for adult subjects
- Informed Assent Form (IAF) - for minor subjects, accompanied by a parental ICF completed by their parents/legally acceptable representatives. The age at which the minor subjects sign the IAF will be subject to local requirements.
- Has medical records available for at least the five years prior to the date of enrolment.
Exclusion
- Currently participating, or participated within the last six months, in
- a clinical trial of a medicinal product or medical device or,
- other non-clinical, low interventional studies
- Currently participating or participated in any BioMarin study at any time.
Key Trial Info
Start Date :
December 17 2017
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
April 29 2020
Estimated Enrollment :
196 Patients enrolled
Trial Details
Trial ID
NCT03449368
Start Date
December 17 2017
End Date
April 29 2020
Last Update
April 5 2021
Active Locations (13)
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1
Medizinische Universitat Wien
Vienna, Austria
2
Aarhus Universitetshospital
Aarhus, Denmark
3
Klinik für Kinder- und Jugendmedizin
Cologne, Germany
4
Medizinische Fakultät, Universitätskinderklinik (KPAE)
Magdeburg, Germany, 44 39120