The Impact of Achondroplasia on Quality of Life, Healthcare Resource Use, Clinical, Socio-economic and Psychosocial state of the Individual (LIAISE)

Study identification

• EU PAS number:
  EUPAS17347
• Study ID:
  39022
• Official title and acronym:
  The Impact of Achondroplasia on Quality of Life, Healthcare Resource Use, Clinical, Socio-economic and Psychosocial state of the Individual (LIAISE)
• DARWIN EU® study:
  No
• Study countries:
  Austria

  Denmark

  Germany

  Italy

  Spain

  Sweden
• Study description:
  Observational studylooking at the burden of illness in achondroplasia patients aged 5-70. The study will include a 5 year review of historical clinical data as well as a 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 13 sites in Germany, Austria, Spain, Italy, Denmark and Sweden
• Study status:
  Finalised

Research institutions and networks

Institutions

BioMarin Pharmaceuticals

Multiple centres: 13 centres are involved in the study

Study timelines

Date when funding contract was signed

Planned: 03/11/2016

Actual: 03/11/2016

Study start date

Planned: 30/10/2017

Actual: 22/12/2017

Data analysis start date

Planned: 28/04/2018

Actual: 31/08/2018

Date of interim report, if expected

Planned: 29/06/2018

Actual: 19/10/2018

Date of final study report

Planned: 14/08/2020

Actual: 19/11/2020

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

BioMarin Pharmaceutical Inc

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable