MucopolysaccharidosesNCT04491747
Assessment of Factors That Affected Respiratory Parameters in Mucopolysaccharidoses Patients
An observational study of Mucopolysaccharidoses, run by Hacettepe University at 1 site in Turkey (Türkiye).
Trial summary
Respiratory system affects in mucopolysaccharidosis (MPS) disease. Respiratory system symptoms are seen in almost every MPS patients and respiratory failure is one of the most common causes of death in this population. Read more about this study
- Who is it for?
- Ages 7 to 30 years
- Conditions the study is for
- Mucopolysaccharidoses
- What is being tested?
- functional capacity
- How many people
- 24 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 1 Feb 2019to 1 Feb 2020
- 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 7 to 30 years
- All sexes
- Child, Adult
Study population: People who diagnosed Mucopolysaccharidoses disease in Hacettepe University were recruited the study.
Who may be able to join
- People who diagnosed Mucopolysaccharidoses disease in Hacettepe University.
- Cooperation
Who cannot join
- Patients who were not walking ability.
Trial locations and contacts
1 site in 1 country.
About this study
Respiratory system affects in mucopolysaccharidosis (MPS) disease. Respiratory system symptoms are seen in almost every MPS patients and respiratory failure is one of the most common causes of death in this population. The aim of the study was to evaluate respiratory muscle strength and endurance, cough effectiveness and functional capacity in MPS patients and to defined the factors that affected the respiratory problem in this.
Read the detailed description
This study was participated MPS patients. Patients' pulmonary function test, functional capacity, respiratory muscle strength and endurance, peak cough flow was assessed and recorded.
Pulmonary function test was assessed with spirometry, functional capacity was evaluated with 3-minute step test and six minute walk test (MWT). Respiratory muscle strength was evaluated mouth pressure measurement (MIP- maximal inspiratory pressure and MEP- maximal expiratory pressure) and Sniff nasal inspiratory pressure (SNIP). Respiratory muscle endurance was assessed Powerbreathe. And cough effectiveness was evaluated peak cough flow.
- Conditions
- Mucopolysaccharidoses
- Being tested
- functional capacity
- Keywords
- Respiratory muscles, exercise test
- Medical subject headings
- Mucopolysaccharidoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
functional capacityDiagnostic test
Also called Exercise capacity
Six minute walk test used assessment of functional capacity.
What is being measured
The primary outcome is the main question the study is designed to answer.
functional capacity
Six minute walk test: Distance walked in six minute was recorded
Measured: 15 minutes
3 minute step test
number of completed step in 3 minute was recorded
Measured: 5 minutes
Respiratory muscle strength assessment
Respiratory muscle strength was evaluated by measuring maximal inspiratory pressure (MIP) and maximal expiratory pressure (MEP) and sniff nasal inspiratory pressure(SNIP). Also dynamic inspiratory muscle strength was evaluated Powerbreathe K5 device.
Measured: 15 minutes
Respiratory muscle endurance
Pressure time unit: A constant load test was used to evaluate respiratory muscle endurance. Using a PowerBreathe device (HaB International Ltd., Southam, UK), the participants were asked to breathe maintaining a constant threshold (60% of MIP) until task failure, and the test duration was recorded. Results were obtained by multiplying the set pressure by the test duration
Measured: 10 minutes
Pulmonary functions
FVC (Forced vital capacity) assessment via spirometry
Measured: 15 minutes
Pulmonary functions
FEV 1(forced expiratory volume in first second) assessment via spirometry
Measured: 15 minutes
Pulmonary functions
PEF (peak expiratory flow) assessment via spirometry
Measured: 15 minutes
MVV (Maximal ventilatory assesssment)
Maximize ventilation by inhaling and exhaling as quickly and deeply as possible for 15 s via spirometry
Measured: 5 minutes
cough effectiveness
cough effectiveness assessment was evaluated peak cough flow meter.
Measured: 5 minutes
Secondary outcomes (2)
heart rate
difference pre and post test heart beat number
Measured: 6 minutes
oxygen saturation
difference pre and post test percentage of oxygen saturation
Measured: 6 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2020
- Study start1 Feb 2019
- Primary completion1 Feb 2020
- Study completion1 Feb 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MucopolysaccharidosesStudies of the same condition, open and under way.
- More trials run by Hacettepe UniversityThe 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hacettepe University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Kübra Kılıç, Hacettepe University
- Investigators
- Deniz Dogru- Ersoz, Professor, Study director, Hacettepe UniversityNaciye Vardar- Yagli, PhD, Study chair, Hacettepe UniversityDilber Ademhan- Tural, MD, Study chair, Hacettepe UniversityNural Kiper, Professor, Study chair, Hacettepe UniversityUgur Ozcelik, Professor, Study chair, Hacettepe UniversityEbru Yalcin, Professor, Study chair, Hacettepe UniversityDeniz Inal-Ince, Professor, Study chair, Hacettepe UniversityMelda Saglam, PhD, Study chair, Hacettepe University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04491747
- Sponsor's protocol ID
- GO 19/97
- Status last verified
- Jul 2020
- First submitted
- 19 Jul 2020
- Last updated
- 29 Jul 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Factors That Affected Respiratory Parameters in Mucopolysaccharidoses Patients
- ClinicalTrials.gov ID
- NCT04491747
- Acronym / sponsor ID
- GO 19/97
- Conditions
- Mucopolysaccharidoses
- Keywords
- Respiratory muscles, exercise test
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Hacettepe UniversityUniversity, hospital or other
- Study start
- 1 Feb 2019 (actual)
- Primary completion
- 1 Feb 2020 (actual)
- Study completion
- 1 Feb 2020 (actual)
- First posted
- 29 Jul 2020 (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.
Respiratory system affects in mucopolysaccharidosis (MPS) disease. Respiratory system symptoms are seen in almost every MPS patients and respiratory failure is one of the most common causes of death in this population. The aim of the study was to evaluate respiratory muscle strength and endurance, cough effectiveness and functional capacity in MPS patients and to defined the factors that affected the respiratory problem in this.
Detailed description
This study was participated MPS patients. Patients' pulmonary function test, functional capacity, respiratory muscle strength and endurance, peak cough flow was assessed and recorded.
Pulmonary function test was assessed with spirometry, functional capacity was evaluated with 3-minute step test and six minute walk test (MWT). Respiratory muscle strength was evaluated mouth pressure measurement (MIP- maximal inspiratory pressure and MEP- maximal expiratory pressure) and Sniff nasal inspiratory pressure (SNIP). Respiratory muscle endurance was assessed Powerbreathe. And cough effectiveness was evaluated peak cough flow.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 7 to 30 years
- Sex
- All
- Age groups
- Child, Adult
- Study population
- People who diagnosed Mucopolysaccharidoses disease in Hacettepe University were recruited the study.
Key inclusion criteria (2 listed)
- People who diagnosed Mucopolysaccharidoses disease in Hacettepe University.
- Cooperation
Key exclusion criteria (1 listed)
- Patients who were not walking ability.
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 |
|---|---|---|
| functional capacityAlso: Exercise capacitySix minute walk test used assessment of functional capacity. | Diagnostic test | – |
Primary outcome
functional capacity
Time frame: 15 minutes
Six minute walk test: Distance walked in six minute was recorded
3 minute step test
Time frame: 5 minutes
number of completed step in 3 minute was recorded
Respiratory muscle strength assessment
Time frame: 15 minutes
Respiratory muscle strength was evaluated by measuring maximal inspiratory pressure (MIP) and maximal expiratory pressure (MEP) and sniff nasal inspiratory pressure(SNIP). Also dynamic inspiratory muscle strength was evaluated Powerbreathe K5 device.
Respiratory muscle endurance
Time frame: 10 minutes
Pressure time unit: A constant load test was used to evaluate respiratory muscle endurance. Using a PowerBreathe device (HaB International Ltd., Southam, UK), the participants were asked to breathe maintaining a constant threshold (60% of MIP) until task failure, and the test duration was recorded. Results were obtained by multiplying the set pressure by the test duration
Pulmonary functions
Time frame: 15 minutes
FVC (Forced vital capacity) assessment via spirometry
Pulmonary functions
Time frame: 15 minutes
FEV 1(forced expiratory volume in first second) assessment via spirometry
Pulmonary functions
Time frame: 15 minutes
PEF (peak expiratory flow) assessment via spirometry
MVV (Maximal ventilatory assesssment)
Time frame: 5 minutes
Maximize ventilation by inhaling and exhaling as quickly and deeply as possible for 15 s via spirometry
cough effectiveness
Time frame: 5 minutes
cough effectiveness assessment was evaluated peak cough flow meter.
2 secondary outcomes
heart rate
Time frame: 6 minutes
oxygen saturation
Time frame: 6 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 29 Jul 2020.
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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.
Deniz Dogru- Ersoz, Professor study director
Hacettepe University
Naciye Vardar- Yagli, PhD study chair
Hacettepe University
Dilber Ademhan- Tural, MD study chair
Hacettepe University
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 Mucopolysaccharidoses, run by Hacettepe University at 1 site in Turkey (Türkiye).
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 2 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 29 Jul 2020. 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 Turkey (Türkiye). 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?
functional capacity (time frame: 15 minutes). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 29 Jul 2020.