Status:
COMPLETED
Lifetime Impact Study for Achondroplasia
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Achondroplasia
Eligibility:
All Genders
3-100 years
Brief Summary
Observational study looking at the burden of illness in achondroplasia subjects aged 3 and above. The study will include a 3 year review of historical clinical data as well as a single point collectio...
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 4 sites...
Eligibility Criteria
Inclusion
- Individual with a documented diagnosis of achondroplasia based on:
- Genetic confirmation of achondroplasia and/or
- Clinical diagnosis of achondroplasia (clinical examination and/or radiological assessment)
- ≥ three years of age at the time of enrollment
- 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 the three years prior to the date of enrollment.
Exclusion
- Currently participating, or participated in the last six months, in
- a clinical trial of a medicinal product or medical device or
- other non-clinical or low interventional studies
- Currently participating or has participated in any BioMarin study at any time.
Key Trial Info
Start Date :
March 31 2019
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
February 6 2021
Estimated Enrollment :
173 Patients enrolled
Trial Details
Trial ID
NCT03872531
Start Date
March 31 2019
End Date
February 6 2021
Last Update
April 5 2021
Active Locations (4)
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1
Hospital de Pediatría "Prof. Dr. Juan P. Garrahan"
Buenos Aires, Argentina
2
Instituto Nacional Fernandes Figueira (IFF), Fundacao Osvaldo Cruz
Rio de Janeiro, Brazil
3
Centro de Pesquisa Clínica do Instituto da Criança HC - FMUSP
São Paulo, Brazil
4
Fundacion Cardioinfantil-instituto de cardiologia
Bogotá, Colombia