Study of efficacy and safety of BYM338 in sporadic inclusion body myositis patients

A randomized, double-blind, placebo-controlled, multicenter, parallel group, dose-finding, pivotal, phase IIb/III study to evaluate the efficacy, safety and tolerability of intravenous BYM338 at 52 weeks on physical function, muscle strength, and mobility and additional long-term safety up to 2 years in patients with sporadic inclusion body myositis - RESILIENT

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-000705-23-DE

Enrollment

240

Registered

2014-01-16

Start date

2014-07-10

Completion date

Unknown

Last updated

2019-06-30

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

Conditions

Sporadic Inclusion Body Myositis MedDRA version: 16.1 Level: PT Classification code 10066407 Term: Inclusion body myositis System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders

Interventions

Product Code: BYM338 Pharmaceutical Form: Solution for injection/infusion CAS Number: 1356922-05-8 Current Sponsor code: BYM338 Concentr

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: •Diagnosed with sporadic inclusion body myositis; •Must be able to walk (assistive aids allowed, including intermittent use of wheelchair) Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 70 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 170

Exclusion criteria

Exclusion criteria: •No other conditions that significantly limit ability to move around; •Must not be using corticosteroids. Must not have used systemic corticosteroid (at daily dose >=10mg prednisone) for the past 3 months; •Must meet cardiovascular requirements; •Must not be pregnant or nursing; •Must not have a chronic active infection (e.g., HIV, hepatitis B or C, tuberculosis, etc); Other protocol-defined inclusion/exclusion criteria may apply

Design outcomes

Primary

Measure	Time frame
Main Objective: To demonstrate that at least one dose regimen of BYM338 will increase the 6 minute walking distance test relative to placebo at week 52.; Secondary Objective: •Change from Baseline in lean body mass (LBM) at Week 52 •Change from Baseline in quadriceps Quantitative Muscle Testing (QMT) at Week 52 •Change from Baseline in Patient-Reported Physical Function at Week 52 •Rate of Fall Events •Change from Baseline in Short Physical Performance Battery score at Week 52 •Dose-response relationship in the change from Baseline in 6MWD meters to Week 52 ;Primary end point(s): Change from Baseline in 6 Minute Walking Distance Test (6MWD) meters to Week 52 ;Timepoint(s) of evaluation of this end point: 52 weeks	—

Measure

Time frame

Main Objective: To demonstrate that at least one dose regimen of BYM338 will increase the 6 minute walking distance test relative to placebo at week 52.; Secondary Objective: •Change from Baseline in lean body mass (LBM) at Week 52 •Change from Baseline in quadriceps Quantitative Muscle Testing (QMT) at Week 52 •Change from Baseline in Patient-Reported Physical Function at Week 52 •Rate of Fall Events •Change from Baseline in Short Physical Performance Battery score at Week 52 •Dose-response relationship in the change from Baseline in 6MWD meters to Week 52 ;Primary end point(s): Change from Baseline in 6 Minute Walking Distance Test (6MWD) meters to Week 52 ;Timepoint(s) of evaluation of this end point: 52 weeks

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Secondary

Measure	Time frame
Secondary end point(s): •Change from Baseline in lean body mass (LBM) at Week 52 •Change from Baseline in quadriceps Quantitative Muscle Testing (QMT) at Week 52 •Change from Baseline in Patient-Reported Physical Function at Week 52 •Rate of Fall Events •Change from Baseline in Short Physical Performance Battery score at Week 52 •Dose-response relationship in the change from Baseline in 6MWD meters to Week 52 ;Timepoint(s) of evaluation of this end point: Week 52 for LBM, QMT, patient-reporter physical function, short physical performance battery and dose-response relationship for 6MWD and measured during entire study for falls , safety and tolerability	—

Measure

Time frame

Secondary end point(s): •Change from Baseline in lean body mass (LBM) at Week 52 •Change from Baseline in quadriceps Quantitative Muscle Testing (QMT) at Week 52 •Change from Baseline in Patient-Reported Physical Function at Week 52 •Rate of Fall Events •Change from Baseline in Short Physical Performance Battery score at Week 52 •Dose-response relationship in the change from Baseline in 6MWD meters to Week 52 ;Timepoint(s) of evaluation of this end point: Week 52 for LBM, QMT, patient-reporter physical function, short physical performance battery and dose-response relationship for 6MWD and measured during entire study for falls , safety and tolerability

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Countries

Australia, Belgium, Denmark, France, Germany, Italy, Japan, Netherlands, Poland, Switzerland, United Kingdom, United States

Contacts

Public ContactMedizinischer Infoservice (MCC)

Novartis Pharma GmbH

infoservice.novartis@novartis.com+491802232300

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026