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

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

Status

Terminated

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT01711606

Acronym

EXPLAIN

Enrollment

Registered

2012-10-22

Start date

2013-03-31

Completion date

2016-01-31

Last updated

2016-03-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Fragile-X Syndrome

Keywords

Fragile-X Syndrome, registry, non-interventional trial, patient-related outcome study, drug utilization, effectiveness, treatment patterns, patient characteristics under real life conditions, quality of life, economics

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

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Healthy volunteers

Inclusion criteria

* Patients with FXS as confirmed by genetic testing

Exclusion criteria

* none * Other protocol-defined inclusion/

Design outcomes

Primary

Measure	Time frame
Patient characteristics (demographics, family history, comorbidities, education, working situation, care situation, insurance status)	3 years

Secondary

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

Countries

Germany

Outcome results