Mucopolysaccharidosis Type INCT03161171
Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III
An observational study of Mucopolysaccharidosis Type I and Mucopolysaccharidosis Type II, run by University Hospital Heidelberg at 1 site in Germany.
Trial summary
The study quantitates behavioral challenges in mucopolysaccharidosis type I-III and parental coping strategies Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type III
- What is being tested?
- This is an observational study
- How many people
- 34 participants
- Where
- 1 siteGermany
- Study dates
- 6 Jul 2017to 13 Nov 2017
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patients with mucopolysaccharidosis type I-III below 18 years of age registered at the German Society for Mucopolysaccharidosis
Who may be able to join
- Diagnosis of mucopolysaccharidosis type I, II or III
- Written informed consent
- Patient is registered at the German Society for Mucopolysaccharidosis
Who cannot join
- Patient / Family does not give written informed consent.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Behavioral Challenges in Children With Mucopolysaccharidosis Type I-III and Parental Coping Strategies
The study quantitates behavioral challenges in mucopolysaccharidosis type I-III and parental coping strategies
Read the 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.
- Conditions
- Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type III, Behavior Disorders, Coping Behavior
- Being tested
- This is an observational study
- Medical subject headings
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis III, Mental Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
This is an observational studyOther
This is an observational study
What is being measured
The primary outcome is the main question the study is designed to answer.
Coping strategies for challenging behavior and for family burden in mucopolysaccharidosis type I-III
assessed by visual analogue scale
Measured: 10 minutes
Secondary outcomes (4)
Quantity of behavioral abnormalities in children with mucopolysaccharidosis type I-III
assessed by visual analogue scale
Measured: 10 minutes
Intensity of behavioral abnormalities in children with mucopolysaccharidosis type I-III
assessed by visual analogue scale
Measured: 10 minutes
Psychosocial burden in families with mucopolysaccharidosis type I-III
assessed by visual analogue scale
Measured: 10 minutes
Psychosocial burden in families with mucopolysaccharidosis type I-III
assessed by Likert scale
Measured: 10 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 May 2017
- Study start6 Jul 2017
- Primary completion13 Nov 2017
- Study completion13 Nov 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Mucopolysaccharidosis Type IStudies of the same condition, open and under way.
- More trials run by University Hospital HeidelbergThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital Heidelberg
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Markus Ries, MD PhD MHSc FCP, University Hospital Heidelberg
- Investigators
- Markus Ries, MD PhD MHSc FCP, Principal investigator, markus.ries@med.uni-heidelberg.de
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03161171
- Sponsor's protocol ID
- 01
- Status last verified
- Dec 2017
- First submitted
- 24 Apr 2017
- Last updated
- 13 Dec 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 33008435
Study at a glance
From the registry record, last updated by the sponsor on 13 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Behavioral Challenges in Children With Mucopolysaccharidosis Type I-III and Parental Coping Strategies
- ClinicalTrials.gov ID
- NCT03161171
- Acronym / sponsor ID
- 01
- Conditions
- Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type III, Behavior Disorders, Coping Behavior
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- University Hospital HeidelbergUniversity, hospital or other
- Study start
- 6 Jul 2017 (actual)
- Primary completion
- 13 Nov 2017 (actual)
- Study completion
- 13 Nov 2017 (actual)
- First posted
- 19 May 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients with mucopolysaccharidosis type I-III below 18 years of age registered at the German Society for Mucopolysaccharidosis
Key inclusion criteria (3 listed)
- Diagnosis of mucopolysaccharidosis type I, II or III
- Written informed consent
- Patient is registered at the German Society for Mucopolysaccharidosis
Key exclusion criteria (1 listed)
- Patient / Family does not give written informed consent.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| This is an observational studyThis is an observational study | Other | – |
Primary outcome
Coping strategies for challenging behavior and for family burden in mucopolysaccharidosis type I-III
Time frame: 10 minutes
assessed by visual analogue scale
4 secondary outcomes
Quantity of behavioral abnormalities in children with mucopolysaccharidosis type I-III
Time frame: 10 minutes
Intensity of behavioral abnormalities in children with mucopolysaccharidosis type I-III
Time frame: 10 minutes
Psychosocial burden in families with mucopolysaccharidosis type I-III
Time frame: 10 minutes
Psychosocial burden in families with mucopolysaccharidosis type I-III
Time frame: 10 minutes
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Dec 2017.
- Derived from this study: PubMed 33008435Hoffmann 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Markus Ries, MD PhD MHSc FCP principal investigator
markus.ries@med.uni-heidelberg.de
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Mucopolysaccharidosis Type I and Mucopolysaccharidosis Type II, run by University Hospital Heidelberg at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Dec 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Coping strategies for challenging behavior and for family burden in mucopolysaccharidosis type I-III (time frame: 10 minutes). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03161171, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Dec 2017.