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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/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
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
Secondary ID [1] 0 0
CBYM338B2203
Universal Trial Number (UTN)
Trial acronym
RESILIENT
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Sporadic Inclusion Body Myositis 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in 6 Minute Walking Distance (6MWD) Test at Week 52
Timepoint [1] 0 0
Baseline, Week 52
Secondary outcome [1] 0 0
Estimated Within Treatment Group Lean Body Mass (LBM) Ratio at Week 52
Timepoint [1] 0 0
Baseline, Week 52
Secondary outcome [2] 0 0
Change From Baseline in Quadriceps Quantitative Muscle Testing (QMT) on the Right Side at Week 52
Timepoint [2] 0 0
Baseline, Week 52
Secondary outcome [3] 0 0
Change From Baseline in Sporadic Inclusion Body Myositis (sIBM) Functional Assessment (sIFA) Score at Week 52
Timepoint [3] 0 0
Baseline, Week 52
Secondary outcome [4] 0 0
Estimated Annual Number of Falls Per Patient Within Treatment Group
Timepoint [4] 0 0
Week 52
Secondary outcome [5] 0 0
Change From Baseline in Short Physical Performance Battery (SPPB) Score at Week 52
Timepoint [5] 0 0
Baseline, Week 52

Eligibility
Key inclusion criteria
Key

- Diagnosed with sporadic inclusion body myositis;

- Must be able to walk (assistive aids allowed, including intermittent use of
wheelchair);

Key
Minimum age
36 Years
Maximum age
85 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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.);

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2/Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
26/09/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
6/01/2016
Sample size
Target
Accrual to date
Final
251
Recruitment in Australia
Recruitment state(s)
NSW,VIC,WA
Recruitment hospital [1] 0 0
Novartis Investigative Site - St. Leonards
Recruitment hospital [2] 0 0
Novartis Investigative Site - Cauldfield
Recruitment hospital [3] 0 0
Novartis Investigative Site - Nedlands
Recruitment postcode(s) [1] 0 0
2065 - St. Leonards
Recruitment postcode(s) [2] 0 0
3162 - Cauldfield
Recruitment postcode(s) [3] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Kansas
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Oregon
Country [9] 0 0
United States of America
State/province [9] 0 0
Texas
Country [10] 0 0
Belgium
State/province [10] 0 0
Bruxelles
Country [11] 0 0
Belgium
State/province [11] 0 0
Edegem
Country [12] 0 0
Belgium
State/province [12] 0 0
Gent
Country [13] 0 0
Denmark
State/province [13] 0 0
Copenhagen
Country [14] 0 0
France
State/province [14] 0 0
Paris
Country [15] 0 0
Italy
State/province [15] 0 0
BS
Country [16] 0 0
Italy
State/province [16] 0 0
Lazio
Country [17] 0 0
Italy
State/province [17] 0 0
ME
Country [18] 0 0
Italy
State/province [18] 0 0
MI
Country [19] 0 0
Italy
State/province [19] 0 0
PD
Country [20] 0 0
Japan
State/province [20] 0 0
Aichi
Country [21] 0 0
Japan
State/province [21] 0 0
Kumamoto
Country [22] 0 0
Japan
State/province [22] 0 0
Miyagi
Country [23] 0 0
Japan
State/province [23] 0 0
Osaka
Country [24] 0 0
Japan
State/province [24] 0 0
Tokushima
Country [25] 0 0
Japan
State/province [25] 0 0
Tokyo
Country [26] 0 0
Japan
State/province [26] 0 0
Wakayama
Country [27] 0 0
Netherlands
State/province [27] 0 0
Amsterdam
Country [28] 0 0
Netherlands
State/province [28] 0 0
Leiden
Country [29] 0 0
Switzerland
State/province [29] 0 0
Zuerich
Country [30] 0 0
United Kingdom
State/province [30] 0 0
Manchester
Country [31] 0 0
United Kingdom
State/province [31] 0 0
London
Country [32] 0 0
United Kingdom
State/province [32] 0 0
Newcastle upon Tyne

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

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).
Trial website
https://clinicaltrials.gov/ct2/show/NCT01925209
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries