Duchenne Muscular DystrophyNCT02034305
Peak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy
An observational study of Duchenne Muscular Dystrophy, run by University of Pittsburgh at 1 site in United States.
Trial summary
This study is to determine whether physiologic measures (peak cough flow, measures of respiratory muscle strength including MIP, MEP ,SNIP, and spirometry) can predict spontaneous cough clearance (as measured by a nuclear medicine study) in children with neuromuscular disease. Read more about this study
- Who is it for?
- Ages 6 to 21 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2014to 1 Jan 2021
- 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 6 to 21 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: Boys 6-21 years of age with Duchenne Muscular Dystrophy
Who may be able to join
- Age 6-21; able to cooperate with study procedures
- Duchenne Muscular Dystrophy OR Becker Muscular Dystrophy
Who cannot join
- Need for mechanical ventilation during the day
- Recent (within 2 weeks) lower respiratory tract infection
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
About this study
Official title: Pilot Study of Cough Peak Flow And Airway Clearance in Pediatric Patients With Neuromuscular Disease
This study is to determine whether physiologic measures (peak cough flow, measures of respiratory muscle strength including MIP, MEP ,SNIP, and spirometry) can predict spontaneous cough clearance (as measured by a nuclear medicine study) in children with neuromuscular disease. It will also determine whether airway clearance is augmented by high frequency chest wall oscillation.
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Airway Clearance, Duchenne Muscular Dystrophy, Peak Cough Flow
- Medical subject headings
- Muscular Dystrophy, Duchenne
What is being measured
The primary outcome is the main question the study is designed to answer.
Spontaneous cough clearance
Measured: Assessed at multiple time points over 30 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2014 (estimated)
- Study startJan 2014
- Primary completion1 Jan 2021
- Study completion1 Jan 2021
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 Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by University of PittsburghThe 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
- Cross-sectional
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Pittsburgh
- Sponsor type
- University, hospital or other
- Collaborators
- Respirtech, Inc.
- Responsible party
- Principal investigator, Daniel J. Weiner, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02034305
- Sponsor's protocol ID
- PRO11100704
- Status last verified
- Jan 2021
- First submitted
- 9 Jan 2014
- Last updated
- 5 Feb 2024
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Weiner DJ, Abdel-Hamid H, Corcoran TE. Pilot study of nuclear scintigraphy to assess cough clearance in DMD. Pediatr Pulmonol. 2022 Jul;57(7):1776-1778. doi: 10.1002/ppul.25894. Epub 2022 May 5. No abstract available.
PubMed 35293696
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Cough Peak Flow And Airway Clearance in Pediatric Patients With Neuromuscular Disease
- ClinicalTrials.gov ID
- NCT02034305
- Acronym / sponsor ID
- PRO11100704
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Airway Clearance, Duchenne Muscular Dystrophy, Peak Cough Flow
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- University of PittsburghUniversity, hospital or other
- Collaborators
- Respirtech, Inc.
- Study start
- Jan 2014
- Primary completion
- 1 Jan 2021 (actual)
- Study completion
- 1 Jan 2021 (actual)
- First posted
- 13 Jan 2014 (estimated)
- 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.
This study is to determine whether physiologic measures (peak cough flow, measures of respiratory muscle strength including MIP, MEP ,SNIP, and spirometry) can predict spontaneous cough clearance (as measured by a nuclear medicine study) in children with neuromuscular disease. It will also determine whether airway clearance is augmented by high frequency chest wall oscillation.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 6 to 21 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Boys 6-21 years of age with Duchenne Muscular Dystrophy
Key inclusion criteria (2 listed)
- Age 6-21; able to cooperate with study procedures
- Duchenne Muscular Dystrophy OR Becker Muscular Dystrophy
Key exclusion criteria (2 listed)
- Need for mechanical ventilation during the day
- Recent (within 2 weeks) lower respiratory tract infection
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Spontaneous cough clearance
Time frame: Assessed at multiple time points over 30 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 5 Feb 2024.
- Derived from this study: PubMed 35293696Weiner DJ, Abdel-Hamid H, Corcoran TE. Pilot study of nuclear scintigraphy to assess cough clearance in DMD. Pediatr Pulmonol. 2022 Jul;57(7):1776-1778. doi: 10.1002/ppul.25894. Epub 2022 May 5. No abstract available.
Related trials for Duchenne Muscular Dystrophy
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy
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.
The record lists no contact. Use the official record to reach the sponsor.
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 Duchenne Muscular Dystrophy, run by University of Pittsburgh at 1 site in United States.
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 2 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 5 Feb 2024. 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 United States. 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?
Spontaneous cough clearance (time frame: Assessed at multiple time points over 30 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 NCT02034305, 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 5 Feb 2024.