NCT02501083

Brief 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 .

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
350

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Jul 2016

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
unknown

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

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 26, 2015

Completed
21 days until next milestone

First Posted

Study publicly available on registry

July 17, 2015

Completed
12 months until next milestone

Study Start

First participant enrolled

July 1, 2016

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2018

Completed
Last Updated

August 17, 2018

Status Verified

July 1, 2018

Enrollment Period

2.3 years

First QC Date

June 26, 2015

Last Update Submit

August 16, 2018

Conditions

Heart, Respiratory, Disease, Functional

Keywords

cardiomyopathyechocardiographyprognosisrespiratoryhome mechanical ventilationvital capacitymaximal inspiratory pressuremaximal expiratory pressure

Outcome Measures

Primary Outcomes (1)

  • predictive factors for long term survival

    10 years

Secondary Outcomes (8)

  • predictuve factors for cardiac events

    10 years

  • predictive factors for respiratory events

    10 years

  • cumulative cardiac events incidence

    10 years

  • cumulative respiratory events incidence

    10 years

  • annual rate of left ventricular ejection fraction decline

    10 years

  • +3 more secondary outcomes

Interventions

no interventionOTHER

The study will not include any intervention.

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Adult patients with muscular dystrophies

You may qualify if:

  • Age higher or equal to 18 years
  • Duchenne muscular dystrophy
  • Becker muscular dystrophy
  • Sarcoglycanopathy
  • limb girdle muscular dystrophy
  • Pompe disease
  • Steinert disease
  • FSH disease
  • Others myopathies

You may not qualify if:

  • Minor patient
  • Sepsis condition

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hopital Raymond Poincare

Garches, 92380, France

Location

Related Publications (3)

  • 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.

    PMID: 37581390DERIVED

  • Fayssoil 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.

    PMID: 27526372DERIVED

  • Fayssoil 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.

    PMID: 27120200DERIVED

MeSH Terms

Conditions

DiseaseCardiomyopathies

Condition Hierarchy (Ancestors)

Pathologic ProcessesPathological Conditions, Signs and SymptomsHeart DiseasesCardiovascular Diseases

Study Officials

  • Abdallah FAYSSOIL, MDPhD

    HOPITAL RAYMOND POINCARE

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 26, 2015

First Posted

July 17, 2015

Study Start

July 1, 2016

Primary Completion

November 1, 2018

Study Completion

November 1, 2018

Last Updated

August 17, 2018

Record last verified: 2018-07

Locations

FR(1)