Skip to main content
OpenTrials
Completed

NCT Number: NCT03161171

Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

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

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Mucopolysaccharidosis Type I Behavior Disorders Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Coping Behavior Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Intellectual Disability Lysosomal Storage Diseases Mental Disorders Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis I Mucopolysaccharidosis II Mucopolysaccharidosis III Mucopolysaccharidosis Type II Mucopolysaccharidosis Type III Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases X-Linked Intellectual Disability

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Observational

Primary location

University of Heidelberg

Heidelberg, 69120, Germany

About this study

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

  • challenging behavior in children with MPS, an orphan, progressive, neurodegenerative metabolic disorder, and
  • 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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

This is an observational study

Other

This is an observational study

Primary outcomes

  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 outcomes

  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

Sponsors and collaborators

Lead sponsor

University Hospital Heidelberg

Other

Registry information

Official study title

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

Important dates

Study start
2017
Primary completion
2017
Study completion
2017
First posted
May 19, 2017
Registry last updated
Dec 13, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.