Lifetime Impact of Achondroplasia Study in Europe-LIAISE

Interested in this Trial?

Contact Us

Trial Status Completed

Trial Identifier

NCT03449368

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 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

Eligibility Criteria

Birth Sex

All

Age

5 Years to 70 Years

Healthy Volunteers

No

Study Type:

Observational

Number of Participants:

196

Study Started:

2017-12-17

Study Updated:

2021-04-05

Trial Locations

  • Medizinische Universitat Wien

    Vienna, , Austria

  • Aarhus Universitetshospital

    Aarhus, , Denmark

  • Klinik für Kinder- und Jugendmedizin

    Cologne, , Germany

  • Medizinische Fakultät, Universitätskinderklinik (KPAE)

    Magdeburg, , Germany

  • Universitätsmedizin

    Mainz, , Germany

  • UOC Pediatria

    Como, , Italy

  • Maternal and Child Health - University of Genova

    Genova, , Italy

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma, , Italy

  • Hospital Universitario La Paz

    Madrid, , Spain

  • Hospital Clínico Universitario Virgen de la Arrixaca

    Murcia, , Spain

  • Se ubica en los siguientes centros, Hospital Universitario Virgen de la Victoria

    Málaga, , Spain

  • Unidad de Dismorfología y metabolismo Hospital Universitario Virgen del Rocío

    Sevilla, , Spain

  • Karolinska Hospital

    Solna, , Sweden

Inclusion Criteria

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.

Exclusion Criteria

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

Contact BioMarin Clinical Trials

Please complete and submit the following form to express interest in participating and request more information about one of our clinical trials.

By completing this form and clicking the send button, you understand and hereby consent to storage of your personal information, including within the United States and Europe, which will be accessible by BioMarin for purposes such as responding to your request, quality control, fulfilling compliance obligations, and assisting with products, services, or clinical trials. Depending on where you live, you may have the right to request access to, removal, or correction of your personal information held by BioMarin. You may submit your request via the contact information and/or webform located within the full BioMarin Privacy Policy.

*required fields

"*" indicates required fields

This field is hidden when viewing the form
This field is hidden when viewing the form