Study of outcome measures in muscular dystrophy clinical trials

Study of outcome measures in muscular dystrophy clinical trials - MDCTN-OM

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

JPRN

Registry ID

JPRN-UMIN000018235

Enrollment

Registered

2015-07-07

Start date

2015-07-08

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne muscular dystrophy

Interventions

None listed

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: a) Patients with Duchenne muscular dystrophy confirmed by dystrophin gene analysis and/or muscle pathology. b) Sex; males. c) Age; 6 to <12 years old. d) Patients who are able to stand up from the floor and complete 6-minute walk test with minimal distance of at least 75m (allowing wheelchair user in his daily life). e) Patients receiving steroid for a minimum of 6 months immediately prior to baseline evaluation, with no significant change in dosing regimen or total daily dosage for a minimum of 3 months immediately prior to baseline evaluation when used. Patients must meet all of the inclusion criteria a)-e) and able to give informed assent (where appropriate) and/or consent in writing signed by the patients and/or legally authorized representative for this study.

Exclusion criteria

Exclusion criteria: a) Patients with obvious heart failure and respiratory failure in the judgement of principal investigator or sub-investigator. b) Patients with persistent artificial ventilation (excepting the use of Noninvasive Positive Pressure Ventilation (NPPV) at night). c) Patients with behavioral problems interfered with motor function test such as intellectual disability, autism, attention-deficit hyperactivity disorder in the judgement of principal investigator or sub-investigator. d) Patients who were considered inappropriate for this study by principal investigator or sub-investigator for other reason. e) Patients who are part of other clinical trials now.

Design outcomes

Primary

Measure	Time frame
Test-retest reliability Concurrent validity Minimal clinically important differences; MCIDs	—

Secondary

Measure	Time frame
Relationship between subjective estimate and motor function test.	—

Countries

Japan

Contacts

Public ContactMikiko Shigemori

Muscular Dystrophy Clinical Trial Network Secretariat Office

shigemori@ncnp.go.jp042-341-2711

Outcome results