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Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

NCT ID: NCT03161171

Last Updated: 2017-12-13

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

COMPLETED

Total Enrollment

34 participants

Study Classification

OBSERVATIONAL

Study Start Date

2017-07-06

Study Completion Date

2017-11-13

Brief Summary

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The study quantitates behavioral challenges in mucopolysaccharidosis type I-III and parental coping strategies

Detailed Description

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This is a prospective, cross-sectional, observational study quantitatively assessing

1. challenging behavior in children with MPS, an orphan, progressive, neurodegenerative metabolic disorder, and
2. parental coping strategies for this challenging behavior.

Patients registered at the German Society for Mucopolysacchardoses giving written informed consent are eligible. The study is based on questionnaires. IRB approval was obtained.

Conditions

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Mucopolysaccharidosis Type I Mucopolysaccharidosis Type II Mucopolysaccharidosis Type III Behavior Disorders Coping Behavior

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Interventions

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This is an observational study

This is an observational study

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of mucopolysaccharidosis type I, II or III
* Written informed consent
* Patient is registered at the German Society for Mucopolysaccharidosis

Exclusion Criteria

* Patient / Family does not give written informed consent.
Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University Hospital Heidelberg

OTHER

Sponsor Role lead

Responsible Party

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Markus Ries, MD PhD MHSc FCP

Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Markus Ries, MD PhD MHSc FCP

Role: PRINCIPAL_INVESTIGATOR

[email protected]

Locations

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University of Heidelberg

Heidelberg, , Germany

Site Status

Countries

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Germany

References

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Hoffmann F, Hoffmann S, Kunzmann K, Ries M. Challenging behavior in mucopolysaccharidoses types I-III and day-to-day coping strategies: a cross sectional explorative study. Orphanet J Rare Dis. 2020 Oct 2;15(1):275. doi: 10.1186/s13023-020-01548-9.

Reference Type DERIVED
PMID: 33008435 (View on PubMed)

Other Identifiers

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01

Identifier Type: -

Identifier Source: org_study_id