Clinical Trial: EXPLAIN -FragilE X Registry: An exPlorative Longitudinal Study for chAracterIzation, Treatment Pathways and patieNt-related Outcomes

Study Status: Terminated
Recruit Status: Terminated
Study Type: Observational

Official Title: EXPLAIN - FragilE X Registry: An exPlorative Longitudinal Study for chAracterIzation, Treatment Pathways and patieNt-related Outcomes

Brief Summary:

This prospective observational study (registry) on Fragile X syndrome (FXS) is designed to evaluate characteristics, management and patient and caregiver-related outcomes the quality of life of Families and patients with FXS and to collect epidemiological and health economic data.

  • EXPLAIN will report current and comprehensive data onEpidemiology data on fragile X syndrome
  • Characterisation of the phenotype of FXS patients
  • Description of patient characteristics (demographics, family history, comorbidity, education, working situations, care situations, insurance status)
  • Documentation of therapeutic interventions
  • Recording and assessment of psychosocial parameters (possibly also inclusion of family members, carers)
  • quality of life of the carer and if possible the patient
  • Health economic parameters and consumption of resources

Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: Patient characteristics (demographics, family history, comorbidities, education, working situation, care situation, insurance status) [ Time Frame: 3 years ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Epidemiology data on Fragile X Syndrome (FXS) [ Time Frame: 3 years ]
  • Health-economic parameters and consumption of resources [ Time Frame: 3 years ]
  • Therapeutic interventions and their application rationale [ Time Frame: 3 Years ]
  • Psychosocial parameters (poss. also inclusion of family members, caregivers) [ Time Frame: 3 years ]
  • Quality of life of the caregiver and possibly the patient [ Time Frame: 3 years ]


Original Secondary Outcome: Same as current

Information By: Novartis

Dates:
Date Received: October 18, 2012
Date Started: March 2013
Date Completion:
Last Updated: March 2, 2016
Last Verified: March 2016