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

NCT ID: NCT01711606

Last Updated: 2016-03-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Total Enrollment

75 participants

Study Classification

OBSERVATIONAL

Study Start Date

2013-03-31

Study Completion Date

2016-01-31

Brief Summary

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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 Description

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Conditions

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Fragile-X Syndrome

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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unselected Fragile-X patients

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

* Patients with FXS as confirmed by genetic testing

Exclusion Criteria

* none
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Frank Hässler, MD

Role: PRINCIPAL_INVESTIGATOR

Klinik für Psychiatrie, Neurologie, Psychosomatik und Psychotherapie im Kindes- und Jugendalter, Gehlsheimer Straße 20, D- 18147 Rostock

Locations

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Novartis Investigative site

Nuremberg, , Germany

Site Status

Countries

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Germany

References

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Haessler F, Gaese F, Huss M, Kretschmar C, Brinkman M, Peters H, Elstner S, Colla M, Pittrow D. Characterization, treatment patterns, and patient-related outcomes of patients with Fragile X syndrome in Germany: final results of the observational EXPLAIN-FXS study. BMC Psychiatry. 2016 Sep 10;16(1):318. doi: 10.1186/s12888-016-1020-5.

Reference Type DERIVED
PMID: 27612457 (View on PubMed)

Haessler F, Gaese F, Colla M, Huss M, Kretschmar C, Brinkman M, Schieb H, Peters H, Elstner S, Pittrow D. EXPLAIN Fragile-X: an explorative, longitudinal study on the characterization, treatment pathways, and patient-related outcomes of Fragile X Syndrome. BMC Psychiatry. 2013 Dec 19;13:339. doi: 10.1186/1471-244X-13-339.

Reference Type DERIVED
PMID: 24354947 (View on PubMed)

Other Identifiers

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CAFQ056BDE-EPI-01

Identifier Type: -

Identifier Source: org_study_id

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