NCT00312247

Brief Summary

The purpose of this research study is to understand the walking patterns, strength and function changes of boys with Duchenne muscular dystrophy on/off corticosteroids to determine the best timing and treatment options to maintain walking for as long as possible.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
85

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Apr 2006

Longer than P75 for all trials

Geographic Reach
1 country

3 active sites

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

April 1, 2006

Completed
4 days until next milestone

First Submitted

Initial submission to the registry

April 5, 2006

Completed
2 days until next milestone

First Posted

Study publicly available on registry

April 7, 2006

Completed
8.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2014

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2015

Completed
Last Updated

May 19, 2015

Status Verified

May 1, 2015

Enrollment Period

8.7 years

First QC Date

April 5, 2006

Last Update Submit

May 18, 2015

Conditions

Keywords

WalkingMuscle StrengthEnergy CostQuality of Life

Outcome Measures

Primary Outcomes (1)

  • Gait pattern

    computerized assessment of walking

    every six months (2x/year)

Secondary Outcomes (4)

  • muscle strength

    every six months (2x/year)

  • energy cost of walking

    every six months (2x/year)

  • gross motor functional skills

    every six months (2x/year)

  • Step activity Monitor-participation

    one week every six months

Study Arms (2)

Boys taking steroids

Boys who are taking prednisone or deflazacort

Boys who are steroid naive

Boys who are not taking steroids for a variety of reasons

Eligibility Criteria

Age4 Years - 21 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-Probability Sample
Study Population

Boys with DMD who are ambulatory starting at the age of 4 until ambulation ceases

You may qualify if:

  • Confirmed diagnosis of DMD
  • Male.
  • Four years of age or older.
  • Ability to walk independently for five minutes to 10 minutes at self-selected speed.
  • Ability to cognitively understand directions for testing procedures.

You may not qualify if:

  • Female
  • Nonambulatory

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

UCLA Department of Orthopaedic Surgery

Los Angeles, California, 90095, United States

Location

Shriners Hospitals for Children

Sacramento, California, 95817, United States

Location

Shriners Hospitals for Children

Portland, Oregon, 97239, United States

Location

Related Publications (2)

  • Sienko Thomas S, Buckon CE, Nicorici A, Bagley A, McDonald CM, Sussman MD. Classification of the gait patterns of boys with Duchenne muscular dystrophy and their relationship to function. J Child Neurol. 2010 Sep;25(9):1103-9. doi: 10.1177/0883073810371002. Epub 2010 Jun 29.

  • McDonald CM, McDonald DA, Bagley A, Sienko Thomas S, Buckon CE, Henricson E, Nicorici A, Sussman MD. Relationship between clinical outcome measures and parent proxy reports of health-related quality of life in ambulatory children with Duchenne muscular dystrophy. J Child Neurol. 2010 Sep;25(9):1130-44. doi: 10.1177/0883073810371509. Epub 2010 Jun 17.

Related Links

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

  • Michael D Sussman, MD

    Shriners Hospitals for Children

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

April 5, 2006

First Posted

April 7, 2006

Study Start

April 1, 2006

Primary Completion

December 1, 2014

Study Completion

March 1, 2015

Last Updated

May 19, 2015

Record last verified: 2015-05

Locations