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

March 2, 2016 updated by: Novartis Pharmaceuticals

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

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

Study Overview

Status

Terminated

Conditions

Study Type

Observational

Enrollment (Actual)

75

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
      • Nuremberg, Germany
        • Novartis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with fragile-X Syndrome

Description

Inclusion Criteria:

  • Patients with FXS as confirmed by genetic testing

Exclusion Criteria:

  • none
  • Other protocol-defined inclusion/exclusion criteria may apply

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
unselected Fragile-X patients

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Investigators

  • Principal Investigator: Frank Hässler, MD, Klinik für Psychiatrie, Neurologie, Psychosomatik und Psychotherapie im Kindes- und Jugendalter, Gehlsheimer Straße 20, D- 18147 Rostock

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2013

Primary Completion (Actual)

January 1, 2016

Study Completion (Actual)

January 1, 2016

Study Registration Dates

First Submitted

October 18, 2012

First Submitted That Met QC Criteria

October 19, 2012

First Posted (Estimate)

October 22, 2012

Study Record Updates

Last Update Posted (Estimate)

March 3, 2016

Last Update Submitted That Met QC Criteria

March 2, 2016

Last Verified

March 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CAFQ056BDE-EPI-01

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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