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

Fragile-X Syndrome Clinical Trial

— EXPLAIN  

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01711606
• Other study ID #: CAFQ056BDE-EPI-01
• Status: Terminated
• Phase: N/A
• First received: October 18, 2012
• Last updated: March 2, 2016
• Start date: March 2013
• Est. completion date: January 2016

Study information

• Verified date: March 2016
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Health authority: Germany: Ethics CommissionGermany: Federal Institute for Drugs and Medical Devices
• Study type: Observational

Clinical Trial 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

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 75
• Est. completion date: January 2016
• Est. primary completion date: January 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Patients with FXS as confirmed by genetic testing

Exclusion Criteria:

• Other protocol-defined inclusion/exclusion criteria may apply

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Fragile X Syndrome
• Fragile-X Syndrome

Locations

Country	Name	City	State
Germany	Novartis Investigative site	Nuremberg

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Patient characteristics (demographics, family history, comorbidities, education, working situation, care situation, insurance status)		3 years	No
Secondary	Epidemiology data on Fragile X Syndrome (FXS)		3 years	No
Secondary	Health-economic parameters and consumption of resources		3 years	No
Secondary	Therapeutic interventions and their application rationale		3 Years	No
Secondary	Psychosocial parameters (poss. also inclusion of family members, caregivers)		3 years	No
Secondary	Quality of life of the caregiver and possibly the patient		3 years	No