NCT04384354

Brief Summary

Relation between clinical and genetic features and acute digestive events in Duchenne muscular dystrophy patients

Trial Health

87
On Track

Trial Health Score

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

Enrollment
200

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Jan 2007

Longer than P75 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

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2007

Completed
11.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2018

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2019

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

July 19, 2019

Completed
10 months until next milestone

First Posted

Study publicly available on registry

May 12, 2020

Completed
Last Updated

May 12, 2020

Status Verified

May 1, 2020

Enrollment Period

11.9 years

First QC Date

July 19, 2019

Last Update Submit

May 11, 2020

Conditions

Duchenne Muscular Dystrophy

Keywords

pseudo occlusiongastrostomy

Outcome Measures

Primary Outcomes (7)

  • Incidence of digestive events

    Clinical parameters (yes or no): gastrostomy, bowel occlusion, acute gastric paresia, biliary complication

    11 years

  • Prevalence of digestive events

    Clinical parameters (yes or no) : gastrostomy, bowel occlusion, acute gastric paresia, biliary complication

    11 years

  • Risk factors for digestive events (1)

    age (years)

    11 years

  • Risk factors for digestive events (2)

    pulmonary functional tests (vital capacity in ml/kg)

    11 years

  • Risk factors for digestive events (3)

    echocardiography parameters (LVEF: left ventricular ejection fraction in %)

    11 years

  • Risk factors for digestive events (4)

    residual dystrophin level (Western blot and/or immunohistochemistry in muscle)

    11 years

  • Risks factors for digestive events (5)

    genetic parameters (type of mutation, functional domain of altered dystrophin)

    11 years

Secondary Outcomes (2)

  • Nutritional status (1)

    11 years

  • Nutritional status (2)

    11 years

Eligibility Criteria

Age15 Years+
Sexmale(Gender-based eligibility)
Gender Eligibility DetailsOnly male patients, due to the specific disease (Duchenne muscular dystrophy patients)
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Duchenne muscular dystrophy patients

You may qualify if:

  • all Duchenne muscular dystrophy patients in our center

You may not qualify if:

  • none

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

CICIT805

Garches, 92380, France

Location

Biospecimen

Retention: SAMPLES WITH DNA

blood samples

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • CICIT805 Garches

    APHP/UVSQ

    STUDY CHAIR

Study Design

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

Study Record Dates

First Submitted

July 19, 2019

First Posted

May 12, 2020

Study Start

January 1, 2007

Primary Completion

December 1, 2018

Study Completion

April 1, 2019

Last Updated

May 12, 2020

Record last verified: 2020-05

Locations

FR(1)