Lifetime Impact Study for Achondroplasia

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Trial Status Completed

Trial Identifier

NCT03872531

Condition

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.

Trial 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 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 175 subjects will be enrolled in sites in Argentina, Colombia and Brazil.

Eligibility Criteria

Birth Sex

All

Age

3 Years to 100 Years

Healthy Volunteers

No

Study Type:

Observational

Number of Participants:

173

Study Started:

2019-03-31

Study Updated:

2021-04-05

Trial Locations

  • Hospital de Pediatría "Prof. Dr. Juan P. Garrahan"

    Buenos Aires, , Argentina

  • Instituto Nacional Fernandes Figueira (IFF), Fundacao Osvaldo Cruz

    Rio de Janeiro, , Brazil

  • Centro de Pesquisa Clínica do Instituto da Criança HC - FMUSP

    São Paulo, , Brazil

  • Fundacion Cardioinfantil-instituto de cardiologia

    Bogotá, , Colombia

Inclusion Criteria

1. Individual with a documented diagnosis of achondroplasia based on:

1. Genetic confirmation of achondroplasia and/or
2. Clinical diagnosis of achondroplasia (clinical examination and/or radiological assessment)
2. ≥ three years of age at the time of enrollment
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 the three years prior to the date of enrollment.

Exclusion Criteria

1. Currently participating, or participated in the last six months, in

1. a clinical trial of a medicinal product or medical device or
2. other non-clinical or low interventional studies
2. Currently participating or has participated in any BioMarin study at any time

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