Pilot study of the treatment of patients with sporadic Inclusion Body Myositis with the Anaplerotic medication Triheptanoi

Phase 1

• Conditions: Sporadic Inclusion body Myositis; Musculoskeletal - Other muscular and skeletal disorders; Inflammatory and Immune System - Other inflammatory or immune system disorders

• Registration Number: ACTRN12614000082606
• Lead Sponsor: ltragenyx

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-01-22
• Last Update Posted: 13 January 2020

Recruitment & Eligibility

• Status: ot yet recruiting
• Sex: All
• Target Recruitment: 3

Inclusion Criteria

Biopsy proven sporadic Inclusion Body Myositis

Exclusion Criteria

Inability to walk

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Muscle strength - 6 minute walk test. Manual muscle testing of distal and proximal upper and lower limb muscles by standard protocol[6 months]

Secondary Outcome Measures

Name	Time	Method
Blood Creatine kinase level[6 months];Muscle MRI of thighs and legs will be assessed qualitatively for signal change and fat content[6 months];Weight change on digital scale[6 months];Assessment of Swallow function using items from the ASSIST tool[6 months]