NCT04491747

Brief 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. 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
24

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Feb 2019

Shorter than P25 for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

February 1, 2019

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2020

Completed
6 months until next milestone

First Submitted

Initial submission to the registry

July 19, 2020

Completed
10 days until next milestone

First Posted

Study publicly available on registry

July 29, 2020

Completed
Last Updated

July 29, 2020

Status Verified

July 1, 2020

Enrollment Period

1 year

First QC Date

July 19, 2020

Last Update Submit

July 27, 2020

Conditions

Mucopolysaccharidoses

Keywords

Respiratory musclesexercise test

Outcome Measures

Primary Outcomes (9)

  • functional capacity

    Six minute walk test: Distance walked in six minute was recorded

    15 minutes

  • 3 minute step test

    number of completed step in 3 minute was recorded

    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.

    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

    10 minutes

  • Pulmonary functions

    FVC (Forced vital capacity) assessment via spirometry

    15 minutes

  • Pulmonary functions

    FEV 1(forced expiratory volume in first second) assessment via spirometry

    15 minutes

  • Pulmonary functions

    PEF (peak expiratory flow) assessment via spirometry

    15 minutes

  • MVV (Maximal ventilatory assesssment)

    Maximize ventilation by inhaling and exhaling as quickly and deeply as possible for 15 s via spirometry

    5 minutes

  • cough effectiveness

    cough effectiveness assessment was evaluated peak cough flow meter.

    5 minutes

Secondary Outcomes (2)

  • heart rate

    6 minutes

  • oxygen saturation

    6 minutes

Interventions

functional capacityDIAGNOSTIC_TEST

Six minute walk test used assessment of functional capacity.

Also known as: Exercise capacity

Eligibility Criteria

Age7 Years - 30 Years
Sexall
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodProbability Sample
Study Population

People who diagnosed Mucopolysaccharidoses disease in Hacettepe University were recruited the study.

You may qualify if:

  • People who diagnosed Mucopolysaccharidoses disease in Hacettepe University.
  • Cooperation

You may not qualify if:

  • Patients who were not walking ability.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hacettepe University

Ankara, Turkey (Türkiye)

Location

MeSH Terms

Conditions

Mucopolysaccharidoses

Interventions

Functional Residual Capacity

Condition Hierarchy (Ancestors)

Carbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Total Lung CapacityLung Volume MeasurementsRespiratory Function TestsDiagnostic Techniques, Respiratory SystemDiagnostic Techniques and ProceduresDiagnosisRespiratory Physiological PhenomenaCirculatory and Respiratory Physiological Phenomena

Study Officials

  • Deniz Dogru- Ersoz, Professor

    Hacettepe University

    STUDY DIRECTOR

  • Naciye Vardar- Yagli, PhD

    Hacettepe University

    STUDY CHAIR

  • Dilber Ademhan- Tural, MD

    Hacettepe University

    STUDY CHAIR

  • Nural Kiper, Professor

    Hacettepe University

    STUDY CHAIR

  • Ugur Ozcelik, Professor

    Hacettepe University

    STUDY CHAIR

  • Ebru Yalcin, Professor

    Hacettepe University

    STUDY CHAIR

  • Deniz Inal-Ince, Professor

    Hacettepe University

    STUDY CHAIR

  • Melda Saglam, PhD

    Hacettepe University

    STUDY CHAIR

  • Serap Sivri, Professor

    Hacettepe University

    STUDY CHAIR

  • Aysegul Tokatlı, Professor

    Hacettepe University

    STUDY CHAIR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Physiotherapist

Study Record Dates

First Submitted

July 19, 2020

First Posted

July 29, 2020

Study Start

February 1, 2019

Primary Completion

February 1, 2020

Study Completion

February 1, 2020

Last Updated

July 29, 2020

Record last verified: 2020-07

Locations

TU(1)