Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

December 11, 2017 updated by: Markus Ries, MD PhD MHSc FCP, University Hospital Heidelberg

Behavioral Challenges in Children With Mucopolysaccharidosis Type I-III and Parental Coping Strategies

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Observational

Enrollment (Actual)

34

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
      • Heidelberg, Germany, 69120
        • University of Heidelberg

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

No older than 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with mucopolysaccharidosis type I-III below 18 years of age registered at the German Society for Mucopolysaccharidosis

Description

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.

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: Case-Only
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Coping strategies for challenging behavior and for family burden in mucopolysaccharidosis type I-III
Time Frame: 10 minutes
assessed by visual analogue scale
10 minutes

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Quantity of behavioral abnormalities in children with mucopolysaccharidosis type I-III
Time Frame: 10 minutes
assessed by visual analogue scale
10 minutes
Intensity of behavioral abnormalities in children with mucopolysaccharidosis type I-III
Time Frame: 10 minutes
assessed by visual analogue scale
10 minutes
Psychosocial burden in families with mucopolysaccharidosis type I-III
Time Frame: 10 minutes
assessed by visual analogue scale
10 minutes
Psychosocial burden in families with mucopolysaccharidosis type I-III
Time Frame: 10 minutes
assessed by Likert scale
10 minutes

Collaborators and Investigators

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

Sponsor

University Hospital Heidelberg

Investigators

  • Principal Investigator: Markus Ries, MD PhD MHSc FCP, markus.ries@med.uni-heidelberg.de

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 (Actual)

July 6, 2017

Primary Completion (Actual)

November 13, 2017

Study Completion (Actual)

November 13, 2017

Study Registration Dates

First Submitted

April 24, 2017

First Submitted That Met QC Criteria

May 18, 2017

First Posted (Actual)

May 19, 2017

Study Record Updates

Last Update Posted (Actual)

December 13, 2017

Last Update Submitted That Met QC Criteria

December 11, 2017

Last Verified

December 1, 2017

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 01 (Miami VAHS)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

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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