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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01925209
First received: August 15, 2013
Last updated: April 5, 2017
Last verified: April 2017
  Purpose
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).

Condition Intervention Phase
Sporadic Inclusion Body Myositis
Drug: BYM338/bimagrumab
Drug: Placebo
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant, Investigator
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Novartis ( Novartis Pharmaceuticals ):

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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.

  • 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.

  • 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.

  • 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.

  • 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.


Enrollment: 251
Actual Study Start Date: September 26, 2013
Study Completion Date: January 6, 2016
Primary Completion Date: January 6, 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

  Eligibility

Ages Eligible for Study:   36 Years to 85 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.);
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01925209

  Show 38 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01925209     History of Changes
Other Study ID Numbers: CBYM338B2203
Study First Received: August 15, 2013
Results First Received: January 5, 2017
Last Updated: April 5, 2017

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
sporadic inclusion body myositis,
myositis,
muscle wasting,
controlled clinical trial,
randomized,
body mass,
muscle function,
strength,
performance,
physical function

Additional relevant MeSH terms:
Myositis, Inclusion Body
Myositis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Antibodies, Monoclonal
Antibodies, Blocking
Immunologic Factors
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on May 25, 2017