Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

NCT03161171 | Completed

• 1 other identifier: 01
• Study Type: observational
• Target: 34
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

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

Outcome Measures

Primary Outcomes (1)

• Coping strategies for challenging behavior and for family burden in mucopolysaccharidosis type I-III

  assessed by visual analogue scale

  10 minutes

Secondary Outcomes (4)

• Quantity of behavioral abnormalities in children with mucopolysaccharidosis type I-III

  10 minutes

• Intensity of behavioral abnormalities in children with mucopolysaccharidosis type I-III

  10 minutes

• Psychosocial burden in families with mucopolysaccharidosis type I-III

  10 minutes

• Psychosocial burden in families with mucopolysaccharidosis type I-III

  10 minutes

Interventions

This is an observational study OTHER

This is an observational study

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• 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

You may qualify if:

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

You may not qualify if:

• Patient / Family does not give written informed consent.

Sponsors & Collaborators

• University Hospital Heidelberg: lead

Study Sites (1)

University of Heidelberg

Heidelberg, 69120, Germany

Location

Related Publications (1)

• 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.

  PMID: 33008435 DERIVED

MeSH Terms

Conditions

Mucopolysaccharidosis I; Mucopolysaccharidosis II; Mucopolysaccharidosis III; Mental Disorders

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System

Study Officials

• Markus Ries, MD PhD MHSc FCP

  markus.ries@med.uni-heidelberg.de

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: April 24, 2017
• First Posted: May 19, 2017
• Study Start: July 6, 2017
• Primary Completion: November 13, 2017
• Study Completion: November 13, 2017
• Last Updated: December 13, 2017

Record last verified: 2017-12

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)