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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT01925209
Registration number
NCT01925209
Ethics application status
Date submitted
15/08/2013
Date registered
19/08/2013
Date last updated
11/08/2017
Titles & IDs
Public title
Efficacy and Safety of Bimagrumab/BYM338 at 52 Weeks on Physical Function, Muscle Strength, Mobility in sIBM Patients
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Scientific 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
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Secondary ID [1]
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CBYM338B2203
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Universal Trial Number (UTN)
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Trial acronym
RESILIENT
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Sporadic Inclusion Body Myositis
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Condition category
Condition code
Musculoskeletal
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Other muscular and skeletal disorders
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Inflammatory and Immune System
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Other inflammatory or immune system disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - BYM338/bimagrumab
Treatment: Drugs - Placebo
Experimental: BYM338/bimagrumab 10 mg/kg - Participants received study medication with BYM338 at 10 mg/kg from Day 1 to Week 52 and up to Week 104, administered by intravenous (i.v.) infusion every 4 weeks.
Experimental: BYM338/bimagrumab 3 mg/kg - Participants received study medication with BYM338 at 3 mg/kg from Day 1 to Week 52 and up to Week 104, administered by i.v. infusion every 4 weeks.
Experimental: BYM338/bimagrumab 1 mg/kg - Participants received study medication with BYM338 at 1 mg/kg from Day 1 to Week 52 and up to Week 104, administered by i.v. infusion every 4 weeks.
Placebo comparator: Placebo - Participants received matching placebo to BYM338 from Day 1 to Week 52 and up to Week 104, administered by i.v. infusion every 4 weeks.
Treatment: Drugs: BYM338/bimagrumab
BYM338, a 150 mg/mL concentrate for solution for i.v. infusion, was provided in colorless glass vials with a rubber stopper and aluminum flip-off caps.
Treatment: Drugs: Placebo
Matching placebo to BYM338 was provided in colorless glass vials with a rubber stopper and aluminum flip-off caps.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change From Baseline in 6 Minute Walking Distance (6MWD) Test at Week 52
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Assessment method [1]
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The 6MWD test measured the distance (in meters) that a participant walked in a 6 minute timeframe. A positive change from baseline indicates improvement.
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Timepoint [1]
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Baseline, Week 52
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Secondary outcome [1]
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Estimated Within Treatment Group Lean Body Mass (LBM) Ratio at Week 52
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Assessment method [1]
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LBM was measured via dual energy x-ray absorptiometry (DXA) and calculated as (LBM at Week 52/LBM at baseline)\*100 . A positive change from baseline indicates improvement.
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Timepoint [1]
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Baseline, Week 52
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Secondary outcome [2]
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Change From Baseline in Quadriceps Quantitative Muscle Testing (QMT) on the Right Side at Week 52
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Assessment method [2]
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Quadriceps muscle strength was measured by portable fixed dynamometry (PFD) on the right side. A negative change from baseline indicates deterioration.
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Timepoint [2]
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Baseline, Week 52
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Secondary outcome [3]
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Change From Baseline in Sporadic Inclusion Body Myositis (sIBM) Functional Assessment (sIFA) Score at Week 52
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Assessment method [3]
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Self-reported physical function was assessed by a newly developed patient reported outcome named sporadic inclusion body myositis (sIBM) functional assessment (sIFA). The sIFA consists of 11 items scored on an 11 point numerical rating scale from 0 (no difficulty) to 10 (unable to do) across 3 domains: upper body functioning, lower body functioning and general functioning. Participants completed the assessment where the recall period was the past week prior to completing the patient reported outcome (PRO). The total score on the sIFA scale ranges from 0 (minimum) to 110 (maximum). Higher values represent a worse outcome. A positive change from baseline indicates deterioration.
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Timepoint [3]
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Baseline, Week 52
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Secondary outcome [4]
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Estimated Annual Number of Falls Per Patient Within Treatment Group
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Assessment method [4]
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Participants documented any fall occurrences in a paper diary during the study.
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Timepoint [4]
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Week 52
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Secondary outcome [5]
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Change From Baseline in Short Physical Performance Battery (SPPB) Score at Week 52
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Assessment method [5]
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The SPPB evaluated lower extremities function by testing gait speed, ability to keep standing balance and time to rise from a chair five times. The sub-score for each test ranged from 0 to 4. The summary score, which was a summation of scores from the 3 tests, ranged from 0 to 12. An increase in score indicates improvement in physical performance. A negative change from baseline indicates deterioration.
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Timepoint [5]
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Baseline, Week 52
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Eligibility
Key inclusion criteria
Key
* Diagnosed with sporadic inclusion body myositis;
* Must be able to walk (assistive aids allowed, including intermittent use of wheelchair);
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Minimum age
36
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Maximum age
85
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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.);
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
26/09/2013
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
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Actual
6/01/2016
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Sample size
Target
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Accrual to date
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Final
251
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Recruitment in Australia
Recruitment state(s)
NSW,VIC,WA
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Recruitment hospital [1]
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Novartis Investigative Site - St. Leonards
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Novartis Investigative Site - Cauldfield
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Recruitment hospital [3]
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Novartis Investigative Site - Nedlands
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Recruitment postcode(s) [1]
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2065 - St. Leonards
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Recruitment postcode(s) [2]
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3162 - Cauldfield
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Recruitment postcode(s) [3]
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6009 - Nedlands
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Recruitment outside Australia
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United States of America
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Arizona
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California
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Florida
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Massachusetts
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Texas
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Belgium
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Bruxelles
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Belgium
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Edegem
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Belgium
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Gent
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Copenhagen
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France
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Paris
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Italy
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BS
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Italy
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Lazio
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Italy
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ME
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Italy
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MI
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Italy
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PD
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Aichi
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Kumamoto
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Miyagi
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Osaka
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Tokushima
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Tokyo
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Wakayama
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Amsterdam
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Leiden
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Zuerich
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Manchester
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London
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United Kingdom
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Newcastle upon Tyne
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Novartis Pharmaceuticals
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Address
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Ethics approval
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Summary
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).
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Trial website
https://clinicaltrials.gov/study/NCT01925209
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Trial related presentations / publications
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.
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Public notes
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Contacts
Principal investigator
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Novartis Pharmaceuticals
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Novartis Pharmaceuticals
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT01925209
Download to PDF