Trials Sponsors Conditions Drugs Pricing

Conditions

Myotonic Dystrophy, Steinert

Keywords

Sleep Apnea SyndromeMuscular Diseasesnon invasive ventilationDiaphragmatic Disorder

Outcome Measures

Primary Outcomes (7)

Change of measurement of spirometry
composite predictive factors of alveolar hypoventilation
every year during 5 years
Change respiratory force
composite predictive factors of alveolar hypoventilation to change Inspiratory and expiratory force
every year during 5 years
Change CO2 response (Read test)
composite predictive factors of alveolar hypoventilation
every year during 5 years
Change subjective sleepiness (Epworth)
composite predictive factors of alveolar hypoventilation
every year during 5 years
change Krupp's Fatigue Severity Scale (KFSS)
composite predictive factors of alveolar hypoventilation This is a short questionnaire of 9 questions to which the patient responds on a Likert scale ranging from 1 to 7
every year during 5 years

Study Arms (2)

PaCO2> 45 mmHg

Diurnal hypercapnia defined by PaCO2\> 45 mmHg

PaCO2 <45 mmHg

PaCO2 diurnal within the limits of normal (PaCO2 \<45 mmHg)

Eligibility Criteria

Age18 Years+

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

Sampling MethodNon-Probability Sample

Study Population

patient with a myotonic dystrophy of Steinert who consults in pneumology and who will have Respiratory functional exploration

You may qualify if:

Myotonic dystrophy of Steinert genetically confirmed (number of known CTG repeats), in its classical form of adult

Sponsors & Collaborators

University Hospital, Lille
Otherlead

Study Sites (6)

Centre Hospitalier Regional D' Angers

Angers, France

Ap-Hp Hu Paris Site Raymond Poincare

Garches, France

Hôpital Calmette, CHRU

Lille, France

Chu Montpellier

Montpellier, France

Chu de Nantes :Site Hôtel-Dieu- Hme - Nantes 1

Nantes, France

Ap-Hp Hu Pitie Salpetriere

Paris, France

MeSH Terms

Conditions

Myotonic DystrophySleep Apnea SyndromesMuscular Diseases

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMusculoskeletal DiseasesMyotonic DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesNervous System DiseasesNeuromuscular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesApnea

Study Officials

Thierry Perez, MD
University Hospital, Lille
PRINCIPAL INVESTIGATOR

Enrollment

113 participants (actual enrollment reported by the registry).

Study Design

Study Type: observational
Observational Model: COHORT
Time Perspective: PROSPECTIVE
Sponsor Type: OTHER
Responsible Party: SPONSOR

Study Record Dates

First Submitted

November 19, 2018

First Posted

December 4, 2018

Study Start

June 30, 2010

Actual (reported)

Factors Associated With Hypoventilation in the Myotonic Dystrophy, Progressive Profile Over 5 Years

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (7)

Study Arms (2)

PaCO2> 45 mmHg

PaCO2 <45 mmHg

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (6)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (7)

Study Arms (2)

PaCO2> 45 mmHg

PaCO2 <45 mmHg

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (6)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations