Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

Completed

• Conditions: Mucopolysaccharidosis Type I; Mucopolysaccharidosis Type II; Coping Behavior; Behavior Disorders; Mucopolysaccharidosis Type III

• Registration Number: NCT03161171
• Lead Sponsor: University Hospital Heidelberg

Brief Summary

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

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 Timeline

• Study First Submitted: 2017-04-24
• Study First Submitted QC: 2017-05-18
• Study First Posted: 2017-05-19
• Study Start: 2017-07-06
• Primary Completion: 2017-11-13
• Study Completion: 2017-11-13
• Status Verified: 2017-12
• Last Update Submitted: 2017-12-11
• Last Update Posted: 2017-12-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 34

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Coping strategies for challenging behavior and for family burden in mucopolysaccharidosis type I-III	10 minutes	assessed by visual analogue scale

Secondary Outcome Measures

Name	Time	Method
Quantity of behavioral abnormalities in children with mucopolysaccharidosis type I-III	10 minutes	assessed by visual analogue scale
Intensity of behavioral abnormalities in children with mucopolysaccharidosis type I-III	10 minutes	assessed by visual analogue scale
Psychosocial burden in families with mucopolysaccharidosis type I-III	10 minutes	assessed by Likert scale

Trial Locations

Locations (1)

University of Heidelberg; 🇩🇪 Heidelberg, Germany