Efficacy and Safety of Bimagrumab/BYM338 at 52 Weeks on Physical Function, Muscle Strength, Mobility in sIBM Patients (RESILIENT)

August 7, 2017 updated by: Novartis Pharmaceuticals

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

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
251

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • St. Leonards, New South Wales, Australia, 2065
        • Novartis Investigative Site
    • Victoria
      • Cauldfield, Victoria, Australia, 3162
        • Novartis Investigative Site
    • Western Australia
      • Nedlands, Western Australia, Australia, 6009
        • Novartis Investigative Site
  • Belgium
      • Bruxelles, Belgium, 1200
        • Novartis Investigative Site
      • Edegem, Belgium, 2650
        • Novartis Investigative Site
      • Gent, Belgium, 9000
        • Novartis Investigative Site
  • Denmark
      • Copenhagen, Denmark, 2100
        • Novartis Investigative Site
  • France
      • Paris, France, 75013
        • Novartis Investigative Site
  • Italy
    • BS
      • Brescia, BS, Italy, 25123
        • Novartis Investigative Site
    • Lazio
      • Roma, Lazio, Italy, 00168
        • Novartis Investigative Site
    • ME
      • Messina, ME, Italy, 98125
        • Novartis Investigative Site
    • MI
      • Milano, MI, Italy, 20133
        • Novartis Investigative Site
    • PD
      • Padova, PD, Italy, 35128
        • Novartis Investigative Site
  • Japan
    • Aichi
      • Nagoya-city, Aichi, Japan, 466-8560
        • Novartis Investigative Site
    • Kumamoto
      • Kumamoto City, Kumamoto, Japan, 860-8556
        • Novartis Investigative Site
    • Miyagi
      • Sendai-city, Miyagi, Japan, 980-8574
        • Novartis Investigative Site
    • Osaka
      • Osaka-city, Osaka, Japan, 534-0021
        • Novartis Investigative Site
    • Tokushima
      • Tokushima-city, Tokushima, Japan, 770-8503
        • Novartis Investigative Site
    • Tokyo
      • Kodaira-city, Tokyo, Japan, 187-8551
        • Novartis Investigative Site
    • Wakayama
      • Wakayama-city, Wakayama, Japan, 641-8510
        • Novartis Investigative Site
  • Netherlands
      • Amsterdam, Netherlands
        • Novartis Investigative Site
      • Leiden, Netherlands, 2333 ZA
        • Novartis Investigative Site
  • Switzerland
      • Zuerich, Switzerland, 8091
        • Novartis Investigative Site
  • United Kingdom
      • London, United Kingdom, NW1 2BU
        • Novartis Investigative Site
      • Newcastle upon Tyne, United Kingdom, NE4 5PL
        • Novartis Investigative Site
    • Manchester
      • Salford, Manchester, United Kingdom, M6 8HD
        • Novartis Investigative Site
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85028
        • Novartis Investigative Site
    • California
      • Orange, California, United States, 92868
        • Novartis Investigative Site
      • Sacramento, California, United States, 95817
        • Novartis Investigative Site
    • Florida
      • Miami, Florida, United States, 33101
        • Novartis Investigative Site
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Novartis Investigative Site
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Novartis Investigative Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Novartis Investigative Site
      • Boston, Massachusetts, United States, 02115
        • Novartis Investigative Site
    • Ohio
      • Columbus, Ohio, United States, 43221
        • Novartis Investigative Site
    • Oregon
      • Portland, Oregon, United States, 97239
        • Novartis Investigative Site
    • Texas
      • Dallas, Texas, United States, 75235
        • Novartis Investigative Site
      • Houston, Texas, United States, 77030
        • Novartis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

36 years to 85 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.);

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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.
Drug: 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.
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.
Drug: 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.
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.
Drug: 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.
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.
Drug: Placebo
Matching placebo to BYM338 was provided in colorless glass vials with a rubber stopper and aluminum flip-off caps.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Change From Baseline in 6 Minute Walking Distance (6MWD) Test at Week 52
Time Frame: Baseline, Week 52
The 6MWD test measured the distance (in meters) that a participant walked in a 6 minute timeframe. A positive change from baseline indicates improvement.
Baseline, Week 52

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Estimated Within Treatment Group Lean Body Mass (LBM) Ratio at Week 52
Time Frame: Baseline, Week 52
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.
Baseline, Week 52
Change From Baseline in Quadriceps Quantitative Muscle Testing (QMT) on the Right Side at Week 52
Time Frame: Baseline, Week 52
Quadriceps muscle strength was measured by portable fixed dynamometry (PFD) on the right side. A negative change from baseline indicates deterioration.
Baseline, Week 52
Change From Baseline in Sporadic Inclusion Body Myositis (sIBM) Functional Assessment (sIFA) Score at Week 52
Time Frame: Baseline, Week 52
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.
Baseline, Week 52
Estimated Annual Number of Falls Per Patient Within Treatment Group
Time Frame: Week 52
Participants documented any fall occurrences in a paper diary during the study.
Week 52
Change From Baseline in Short Physical Performance Battery (SPPB) Score at Week 52
Time Frame: Baseline, Week 52
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.
Baseline, Week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Novartis Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 26, 2013

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 6, 2016

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 6, 2016

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 15, 2013

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 15, 2013

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 19, 2013

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 11, 2017

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 7, 2017

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CBYM338B2203

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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