Heart, Respiratory, Disease, FunctionalNCT02501083
Natural History of Cardiac and Respiration Function in Patients With Muscular Dystrophies on Home Mechanical Ventilation
An observational study of Heart, Respiratory, Disease, Functional, run by Centre d'Investigation Clinique et Technologique 805 at 1 site in France.
Trial summary
Muscular dystrophies are inherited disorders that affect skeletal muscle. Cardiac and respiratory function may be affected in this group of diseases. The investigators sought to analyze the long term cardiac and respiratory function in patients with muscular dystrophies recquiring home mechnaical ventilation . Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Heart, Respiratory, Disease, Functional
- How many people
- 350 participants
- Where
- 1 siteFrance
- Study dates
- Jul 2016to Nov 2018
- 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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adult patients with muscular dystrophies
Who may be able to join
- Age higher or equal to 18 years
- Duchenne muscular dystrophy
- Becker muscular dystrophy
- Sarcoglycanopathy
- limb girdle muscular dystrophy
- Pompe disease
Show all 9Show fewer
- Steinert disease
- FSH disease
- Others myopathies
Who cannot join
- Minor patient
- Sepsis condition
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Natural History of Cardiac and Respiratory Function in Patients With Muscular Dystrophies on Home Mechanical Ventilation
Muscular dystrophies are inherited disorders that affect skeletal muscle. Cardiac and respiratory function may be affected in this group of diseases. The investigators sought to analyze the long term cardiac and respiratory function in patients with muscular dystrophies recquiring home mechnaical ventilation .
Read the detailed description
Muscular dystrophies are neuromuscular disorders with disability and can affect respiratory muscle function and left ventricular function. Medical management of patients with cardiomyopathy relies mainly on angiotensin-converting-enzyme inhibitors and beta blockers. Respiratory management relies on home mechanical ventilation and couph assist.
The investigators aim
- to analyze cardiac and respiratory function over time in patients with muscular dystrophies followed at the home mechanical ventilation unit.
- to determine the long term impact of the home mechanical ventilation on cardiac and respiratory events and to define the predictive factors associated.
Patients will be included retrospectively. Tissue Doppler imaging and Tricuspid annular plane systolic excursion were used for the assessment of the right ventricle. Left ventricular ejection fraction was used for the analysis of the left ventricle. Respiratory function was assessed using the vital capacity, the maximal inspiratory pressure, the maximal expiratory pressure, the peak couph flow rate.
- Conditions
- Heart, Respiratory, Disease, Functional
- Being tested
- no intervention
- Keywords
- cardiomyopathy, echocardiography, prognosis, respiratory, home mechanical ventilation, vital capacity, maximal inspiratory pressure, maximal expiratory pressure
- Medical subject headings
- Disease, Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
no interventionOther
The study will not include any intervention.
What is being measured
The primary outcome is the main question the study is designed to answer.
predictive factors for long term survival
Measured: 10 years
Secondary outcomes (8)
predictuve factors for cardiac events
Measured: 10 years
predictive factors for respiratory events
Measured: 10 years
cumulative cardiac events incidence
Measured: 10 years
cumulative respiratory events incidence
Measured: 10 years
annual rate of left ventricular ejection fraction decline
Measured: 10 years
annual rate of respiratory vital capacity decline
Measured: 10 years
annual rate of maximal inspiratory pressure decline
Measured: 10 years
annual rate of maximal expiratory pressure decline
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jul 2015 (estimated)
- Study startJul 2016
- Primary completionNov 2018 (estimated)
- Study completionNov 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Heart, Respiratory, Disease, FunctionalStudies of the same condition, open and under way.
- 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
- Retrospective
- Enrolment
- 350 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre d'Investigation Clinique et Technologique 805
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Abdallah FAYSSOIL, MDPhD, Principal investigator, HOPITAL RAYMOND POINCARE
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02501083
- Sponsor's protocol ID
- CRHF-MD
- Status last verified
- Jul 2018
- First submitted
- 26 Jun 2015
- Last updated
- 17 Aug 2018
- Results posted
- No
- Data monitoring committee
- No
Publications
Fayssoil A, Mansencal N, Nguyen LS, Nardi O, Yaou RB, Leturcq F, Amthor H, Wahbi K, Becane HM, Lofaso F, Prigent H, Bassez G, Behin A, Stojkovic T, Fontaine B, Duboc D, Dubourg O, Clair B, Laforet P, Annane D, Orlikowski D. Prognosis of Right Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy. J Am Heart Assoc. 2023 Aug 15;12(16):e027231. doi: 10.1161/JAHA.122.027231. Epub 2023 Aug 10.
PubMed 37581390Fayssoil A, Lazarus A, Wahbi K, Ogna A, Nardi O, Lofaso F, Clair B, Orlikowski D, Annane D. Cardiac implantable electronic devices in tracheotomized muscular dystrophy patients: Safety and risks. Int J Cardiol. 2016 Nov 1;222:975-977. doi: 10.1016/j.ijcard.2016.08.040. Epub 2016 Aug 6.
PubMed 27526372Fayssoil A, Ogna A, Chaffaut C, Chevret S, Guimaraes-Costa R, Leturcq F, Wahbi K, Prigent H, Lofaso F, Nardi O, Clair B, Behin A, Stojkovic T, Laforet P, Orlikowski D, Annane D. Natural History of Cardiac and Respiratory Involvement, Prognosis and Predictive Factors for Long-Term Survival in Adult Patients with Limb Girdle Muscular Dystrophies Type 2C and 2D. PLoS One. 2016 Apr 27;11(4):e0153095. doi: 10.1371/journal.pone.0153095. eCollection 2016.
PubMed 27120200
Study at a glance
From the registry record, last updated by the sponsor on 17 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Natural History of Cardiac and Respiratory Function in Patients With Muscular Dystrophies on Home Mechanical Ventilation
- ClinicalTrials.gov ID
- NCT02501083
- Acronym / sponsor ID
- CRHF-MD
- Conditions
- Heart, Respiratory, Disease, Functional
- Keywords
- cardiomyopathy, echocardiography, prognosis, respiratory, home mechanical ventilation, vital capacity, maximal inspiratory pressure, maximal expiratory pressure
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 350 participants (estimated)
- Lead sponsor
- Centre d'Investigation Clinique et Technologique 805University, hospital or other
- Study start
- Jul 2016
- Primary completion
- Nov 2018 (estimated)
- Study completion
- Nov 2018 (estimated)
- First posted
- 17 Jul 2015 (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.
Muscular dystrophies are inherited disorders that affect skeletal muscle. Cardiac and respiratory function may be affected in this group of diseases. The investigators sought to analyze the long term cardiac and respiratory function in patients with muscular dystrophies recquiring home mechnaical ventilation .
Detailed description
Muscular dystrophies are neuromuscular disorders with disability and can affect respiratory muscle function and left ventricular function. Medical management of patients with cardiomyopathy relies mainly on angiotensin-converting-enzyme inhibitors and beta blockers. Respiratory management relies on home mechanical ventilation and couph assist.
The investigators aim
- to analyze cardiac and respiratory function over time in patients with muscular dystrophies followed at the home mechanical ventilation unit.
- to determine the long term impact of the home mechanical ventilation on cardiac and respiratory events and to define the predictive factors associated.
Patients will be included retrospectively. Tissue Doppler imaging and Tricuspid annular plane systolic excursion were used for the assessment of the right ventricle. Left ventricular ejection fraction was used for the analysis of the left ventricle. Respiratory function was assessed using the vital capacity, the maximal inspiratory pressure, the maximal expiratory pressure, the peak couph flow rate.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult patients with muscular dystrophies
Key inclusion criteria (9 listed)
- Age higher or equal to 18 years
- Duchenne muscular dystrophy
- Becker muscular dystrophy
- Sarcoglycanopathy
- limb girdle muscular dystrophy
Key exclusion criteria (2 listed)
- Minor patient
- Sepsis condition
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 |
|---|---|---|
| no interventionThe study will not include any intervention. | Other | – |
Primary outcome
predictive factors for long term survival
Time frame: 10 years
8 secondary outcomes
predictuve factors for cardiac events
Time frame: 10 years
predictive factors for respiratory events
Time frame: 10 years
cumulative cardiac events incidence
Time frame: 10 years
cumulative respiratory events incidence
Time frame: 10 years
annual rate of left ventricular ejection fraction decline
Time frame: 10 years
annual rate of respiratory vital capacity decline
Time frame: 10 years
annual rate of maximal inspiratory pressure decline
Time frame: 10 years
annual rate of maximal expiratory pressure decline
Time frame: 10 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Aug 2018.
- Derived from this study: PubMed 37581390Fayssoil A, Mansencal N, Nguyen LS, Nardi O, Yaou RB, Leturcq F, Amthor H, Wahbi K, Becane HM, Lofaso F, Prigent H, Bassez G, Behin A, Stojkovic T, Fontaine B, Duboc D, Dubourg O, Clair B, Laforet P, Annane D, Orlikowski D. Prognosis of Right Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy. J Am Heart Assoc. 2023 Aug 15;12(16):e027231. doi: 10.1161/JAHA.122.027231. Epub 2023 Aug 10.
- Derived from this study: PubMed 27526372Fayssoil A, Lazarus A, Wahbi K, Ogna A, Nardi O, Lofaso F, Clair B, Orlikowski D, Annane D. Cardiac implantable electronic devices in tracheotomized muscular dystrophy patients: Safety and risks. Int J Cardiol. 2016 Nov 1;222:975-977. doi: 10.1016/j.ijcard.2016.08.040. Epub 2016 Aug 6.
- Derived from this study: PubMed 27120200Fayssoil A, Ogna A, Chaffaut C, Chevret S, Guimaraes-Costa R, Leturcq F, Wahbi K, Prigent H, Lofaso F, Nardi O, Clair B, Behin A, Stojkovic T, Laforet P, Orlikowski D, Annane D. Natural History of Cardiac and Respiratory Involvement, Prognosis and Predictive Factors for Long-Term Survival in Adult Patients with Limb Girdle Muscular Dystrophies Type 2C and 2D. PLoS One. 2016 Apr 27;11(4):e0153095. doi: 10.1371/journal.pone.0153095. eCollection 2016.
Related trials for Heart, Respiratory, Disease, Functional
RecruitingObservational study
Physical Impairments in Premature Ventricular Complex
Premature Ventricular Complexes
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Not yet recruiting
Bedside Assessment of Right Ventricular Output Lying in Prone Or Supine
Right Ventricular Function, Prone Position
RecruitingPhase 4
Central Apnea in Heart Failure: Physiological Mechanisms to Inform Treatment
Sleep Disordered Breathing, Able Bodied
RecruitingObservational study
PH-DyPred: A Multimodal Dynamic Risk Prediction Study in Pulmonary Hypertension
Pulmonary Hypertension
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.
Abdallah FAYSSOIL, MDPhD principal investigator
HOPITAL RAYMOND POINCARE
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 Heart, Respiratory, Disease, Functional, run by Centre d'Investigation Clinique et Technologique 805 at 1 site in France.
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 9 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: status not confirmed, as of the sponsor's last update on 17 Aug 2018. 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 France. 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?
Results are not available in the source record.
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?
predictive factors for long term survival (time frame: 10 years). 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 NCT02501083, which also lists 3 linked publications.
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 17 Aug 2018.