NCT03449368
Achondroplasia
The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.
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 questionnaire data to look at the impact on the following in individuals with achondroplasia versus a normative population:
* Quality of life
* Clinical burden
* Healthcare resource use
* Socio-economic burden
* Psychosocial burden
Up to 300 subjects will be included in sites in Germany, Spain, Italy, Sweden, Austria and Denmark
All
5 Years to 70 Years
No
Observational
196
2017-12-17
2021-04-05
Vienna, , Austria
Aarhus, , Denmark
Cologne, , Germany
Magdeburg, , Germany
Mainz, , Germany
Como, , Italy
Genova, , Italy
Roma, , Italy
Madrid, , Spain
Murcia, , Spain
Málaga, , Spain
Sevilla, , Spain
Solna, , Sweden
1. Individuals with a documented diagnosis of achondroplasia based on:
1. Genetic confirmation of achondroplasia and/or
2. Clinical diagnosis of achondroplasia (clinical examination or radiological assessment)
2. ≥ five years of age at the time of enrolment
3. 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)
4. Agrees to participate in the study and has read, understood, completed and signed:
1. Informed Consent Form (ICF) – for adult subjects
2. 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.
5. Has medical records available for at least the five years prior to the date of enrolment.
1. Currently participating, or participated within the last six months, in
1. a clinical trial of a medicinal product or medical device or,
2. other non-clinical, low interventional studies
2. Currently participating or participated in any BioMarin study at any time
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