Parental Coping With Challenging Behavior in Mucopolysacc... | NCT03161171

Studies
Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

NCT03161171

Sponsor

University Hospital Heidelberg

Status

Completed

Last Update Posted

Dec 13, 2017Actual

Enrollment

34Actual

Phase

Not provided

Conditions

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

Interventions

This is an observational study

Countries

Germany

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Dec 2017

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 6, 2017Actual

Primary Completion Date

Nov 13, 2017Actual

Completion Date

Nov 13, 2017Actual

First Submitted Date

Apr 24, 2017

First Submission Date that Met QC Criteria

May 18, 2017

First Posted Date

May 19, 2017Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 11, 2017

Last Update Posted Date

Dec 13, 2017Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Markus Ries, MD PhD MHSc FCP, Professor, University Hospital HeidelbergPrincipal Investigator

Lead Sponsor

University Hospital HeidelbergOTHER

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

34Actual

Arms/Interventions Module

Arm Groups

Not provided

Interventions

Name	Type	Description	Arm Group Labels	Other Names
This is an observational study	Other	This is an observational study

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

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

Other Outcomes

Not provided

Eligibility Module

Eligibility 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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

18 Years

Standard Ages

ChildAdult

Study Population

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

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Markus Ries, MD PhD MHSc FCP	markus.ries@med.uni-heidelberg.de	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
University of Heidelberg		Heidelberg		69120	Germany

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
33008435	Derived	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.

ID	Term
D008059	Mucopolysaccharidosis I
D016532	Mucopolysaccharidosis II
D009084	Mucopolysaccharidosis III
D001523	Mental Disorders

ID	Term
D009083	Mucopolysaccharidoses
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn