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Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

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ClinicalTrials.gov Identifier: NCT03161171
Recruitment Status : Completed
First Posted : May 19, 2017
Last Update Posted : December 13, 2017
Sponsor:
Information provided by (Responsible Party):
Markus Ries, MD PhD MHSc FCP, University Hospital Heidelberg

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

Condition or disease Intervention/treatment
Mucopolysaccharidosis Type I Mucopolysaccharidosis Type II Mucopolysaccharidosis Type III Behavior Disorders Coping Behavior Other: This is an observational study

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
Actual Enrollment : 34 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Behavioral Challenges in Children With Mucopolysaccharidosis Type I-III and Parental Coping Strategies
Actual Study Start Date : July 6, 2017
Actual Primary Completion Date : November 13, 2017
Actual Study Completion Date : November 13, 2017



Intervention Details:
  • Other: This is an observational study
    This is an observational study


Primary Outcome Measures :
  1. Coping strategies for challenging behavior and for family burden in mucopolysaccharidosis type I-III [ Time Frame: 10 minutes ]
    assessed by visual analogue scale


Secondary Outcome Measures :
  1. Quantity of behavioral abnormalities in children with mucopolysaccharidosis type I-III [ Time Frame: 10 minutes ]
    assessed by visual analogue scale

  2. Intensity of behavioral abnormalities in children with mucopolysaccharidosis type I-III [ Time Frame: 10 minutes ]
    assessed by visual analogue scale

  3. Psychosocial burden in families with mucopolysaccharidosis type I-III [ Time Frame: 10 minutes ]
    assessed by visual analogue scale

  4. Psychosocial burden in families with mucopolysaccharidosis type I-III [ Time Frame: 10 minutes ]
    assessed by Likert scale



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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03161171


Locations
Germany
University of Heidelberg
Heidelberg, Germany, 69120
Sponsors and Collaborators
University Hospital Heidelberg
Investigators
Principal Investigator: Markus Ries, MD PhD MHSc FCP markus.ries@med.uni-heidelberg.de

Responsible Party: Markus Ries, MD PhD MHSc FCP, Professor, University Hospital Heidelberg
ClinicalTrials.gov Identifier: NCT03161171     History of Changes
Other Study ID Numbers: 01
First Posted: May 19, 2017    Key Record Dates
Last Update Posted: December 13, 2017
Last Verified: December 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Mucopolysaccharidosis III
Mucopolysaccharidoses
Mental Disorders
Mucopolysaccharidosis I
Mucopolysaccharidosis II
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System