Clinical trial NCT01925209: Efficacy and Safety of Bimagrumab/BYM338 at 52 Weeks on Physical Function, Muscle Strength, Mobility in sIBM Patients
Official title: A Randomized, Double-blind, Placebo-controlled, Multicenter, Parallel Group, Dose-finding, Pivotal, Phase 2b/3 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Sporadic Inclusion Body Myositis
Keywords: sporadic inclusion body myositis,
myositis,
muscle wasting,
controlled clinical trial,
randomized,
body mass,
muscle function,
strength,
performance,
physical function
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2013-08-19
Enrollment: 251
Sex: ALL
Minimum age (years): 36
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study evaluated the efficacy, safety and tolerability of multiple doses of bimagrumab/BYM338 vs placebo, when administered intravenously (i.v.), on physical function, muscle strength, and mobility in patients with sporadic inclusion body myositis (sIBM).


Eligibility criteria: Key Inclusion Criteria:

* Diagnosed with sporadic inclusion body myositis;
* Must be able to walk (assistive aids allowed, including intermittent use of wheelchair);

Key Exclusion Criteria:

* Must not have 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.);

Primary outcomes:
measure: Change From Baseline in 6 Minute Walking Distance (6MWD) Test at Week 52
timeFrame: Baseline, Week 52
Secondary outcomes:
measure: Estimated Within Treatment Group Lean Body Mass (LBM) Ratio at Week 52
timeFrame: Baseline, Week 52
measure: Change From Baseline in Quadriceps Quantitative Muscle Testing (QMT) on the Right Side at Week 52
timeFrame: Baseline, Week 52
measure: Change From Baseline in Sporadic Inclusion Body Myositis (sIBM) Functional Assessment (sIFA) Score at Week 52
timeFrame: Baseline, Week 52
measure: Estimated Annual Number of Falls Per Patient Within Treatment Group
timeFrame: Week 52
measure: Change From Baseline in Short Physical Performance Battery (SPPB) Score at Week 52
timeFrame: Baseline, Week 52
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Novartis Investigative Site
state: Arizona
city: Orange
country: United States
facility: Novartis Investigative Site
state: California
city: Sacramento
country: United States
facility: Novartis Investigative Site
state: California
city: Miami
country: United States
facility: Novartis Investigative Site
state: Florida
city: Kansas City
country: United States
facility: Novartis Investigative Site
state: Kansas
city: Baltimore
country: United States
facility: Novartis Investigative Site
state: Maryland
city: Boston
country: United States
facility: Novartis Investigative Site
state: Massachusetts
city: Boston
country: United States
facility: Novartis Investigative Site
state: Massachusetts
city: Columbus
country: United States
facility: Novartis Investigative Site
state: Ohio
city: Portland
country: United States
facility: Novartis Investigative Site
state: Oregon
city: Dallas
country: United States
facility: Novartis Investigative Site
state: Texas
city: Houston
country: United States
facility: Novartis Investigative Site
state: Texas
city: St. Leonards
country: Australia
facility: Novartis Investigative Site
state: New South Wales
city: Cauldfield
country: Australia
facility: Novartis Investigative Site
state: Victoria
city: Nedlands
country: Australia
facility: Novartis Investigative Site
state: Western Australia
city: Bruxelles
country: Belgium
facility: Novartis Investigative Site
city: Edegem
country: Belgium
facility: Novartis Investigative Site
city: Gent
country: Belgium
facility: Novartis Investigative Site
city: Copenhagen
country: Denmark
facility: Novartis Investigative Site
city: Paris
country: France
facility: Novartis Investigative Site
city: Brescia
country: Italy
facility: Novartis Investigative Site
state: BS
city: Roma
country: Italy
facility: Novartis Investigative Site
state: Lazio
city: Messina
country: Italy
facility: Novartis Investigative Site
state: ME
city: Milano
country: Italy
facility: Novartis Investigative Site
state: MI
city: Padova
country: Italy
facility: Novartis Investigative Site
state: PD
city: Nagoya-city
country: Japan
facility: Novartis Investigative Site
state: Aichi
city: Kumamoto City
country: Japan
facility: Novartis Investigative Site
state: Kumamoto
city: Sendai-city
country: Japan
facility: Novartis Investigative Site
state: Miyagi
city: Osaka-city
country: Japan
facility: Novartis Investigative Site
state: Osaka
city: Tokushima-city
country: Japan
facility: Novartis Investigative Site
state: Tokushima
city: Kodaira-city
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Wakayama-city
country: Japan
facility: Novartis Investigative Site
state: Wakayama
city: Amsterdam
country: Netherlands
facility: Novartis Investigative Site
city: Leiden
country: Netherlands
facility: Novartis Investigative Site
city: Zuerich
country: Switzerland
facility: Novartis Investigative Site
city: Salford
country: United Kingdom
facility: Novartis Investigative Site
state: Manchester
city: London
country: United Kingdom
facility: Novartis Investigative Site
city: Newcastle upon Tyne
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Hanna MG, Badrising UA, Benveniste O, Lloyd TE, Needham M, Chinoy H, Aoki M, Machado PM, Liang C, Reardon KA, de Visser M, Ascherman DP, Barohn RJ, Dimachkie MM, Miller JAL, Kissel JT, Oskarsson B, Joyce NC, Van den Bergh P, Baets J, De Bleecker JL, Karam C, David WS, Mirabella M, Nations SP, Jung HH, Pegoraro E, Maggi L, Rodolico C, Filosto M, Shaibani AI, Sivakumar K, Goyal NA, Mori-Yoshimura M, Yamashita S, Suzuki N, Katsuno M, Murata K, Nodera H, Nishino I, Romano CD, Williams VSL, Vissing J, Auberson LZ, Wu M, de Vera A, Papanicolaou DA, Amato AA; RESILIENT Study Group. Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial. Lancet Neurol. 2019 Sep;18(9):834-844. doi: 10.1016/S1474-4422(19)30200-5.