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An Extension Study of the Efficacy, Safety and Tolerability of BYM338 (Bimagrumab) in Patients With Sporadic Inclusion Body Myositis Who Previously Participated in the Core Study CBYM338B2203

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ClinicalTrials.gov Identifier: NCT02573467
Recruitment Status : Completed
First Posted : October 9, 2015
Last Update Posted : November 13, 2017
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:
This extension study will provide data to further evaluate the efficacy, safety, and tolerability of three doses of BYM338 and to assess the long-term effects of BYM338 in patients with sporadic inclusion body myositis. Patients who complete the core study and qualify for this extension study will enter Treatment Period 1, during which they will continue on the study drug which they were receiving in the core study (either one of the three BYM338 doses or placebo). Treatment Period 1 will continue until the dose of BYM338 with the best benefit-risk profile is determined and selected. The duration of Treatment Period 1 could be up to one year (but estimated to be between 6 and 8 months) and the total duration of Treatment Period 1 will depend upon the timing of selection of the dose for Treatment Period 2. Once the dose with the best benefit-risk profile is selected, all ongoing subjects (including those who have been receiving placebo) will enter Treatment Period 2 and will be switched to open-label treatment with BYM338 at the selected dose. Treatment Period 2 will continue until BYM338 is approved and commercially available in the country of the investigational site.

Condition or disease Intervention/treatment Phase
Sporadic Inclusion Body Myositis Drug: Bimagrumab Drug: Placebo Phase 3

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 211 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: Extension of the CBYM338B2203 Phase IIb/III Study to Evaluate the Long-term Efficacy, Safety and Tolerability of Intravenous BYM338 in Patients With Sporadic Inclusion Body Myositis
Actual Study Start Date : November 2, 2015
Primary Completion Date : August 17, 2016
Study Completion Date : February 13, 2017


Arms and Interventions

Arm Intervention/treatment
Experimental: BYM338/bimagrumab 10 mg/kg
Approximately 60 patients who meet all inclusion criteria and none of the exclusion criteria will be treated in Treatment Period 1 with the BYM338 10 mg/kg dose administered via intravenous infusion every 4 weeks. In Treatment Period 2, patients will be treated with open-label BYM338 at the selected dose, administered via intravenous infusion every 4 weeks.
Drug: Bimagrumab
Other Name: BYM338
Experimental: BYM338/bimagrumab 3 mg/kg
Approximately 60 patients who meet all inclusion criteria and none of the exclusion criteria will be treated in Treatment Period 1 with the BYM338 3 mg/kg dose administered via intravenous infusion every 4 weeks. In Treatment Period 2, patients will be treated with open-label BYM338 at the selected dose, administered via intravenous infusion every 4 weeks.
Drug: Bimagrumab
Other Name: BYM338
Experimental: BYM338/bimagrumab 1 mg/kg
Approximately 60 patients who meet all inclusion criteria and none of the exclusion criteria will be treated in Treatment Period 1 with the BYM338 1 mg/kg dose administered via intravenous infusion every 4 weeks. In Treatment Period 2, patients will be treated with open-label BYM338 at the selected dose, administered via intravenous infusion every 4 weeks.
Drug: Bimagrumab
Other Name: BYM338
Placebo Comparator: Placebo
Approximately 60 patients who meet all inclusion criteria and none of the exclusion criteria will be treated in Treatment Period 1 with Placebo administered via intravenous infusion every 4 weeks. In Treatment Period 2, patients will be treated with open-label BYM338 at the selected dose, administered via intravenous infusion every 4 weeks.
Drug: Placebo


Outcome Measures

Primary Outcome Measures :
  1. Safety Assessment, incidence of Treatment-Emergent Adverse Events [ Time Frame: to end of study (2 years) ]
    Adverse events and serious adverse events collected will be reported

  2. Change from baseline in 6 Minute Walking Distance Test (6MWD) [ Time Frame: Baseline, 1 year ]
    The 6MWD test measures the distance (in meters) that a patient can walk in a 6 minute time frame.


Secondary Outcome Measures :
  1. Change from baseline in quadriceps muscle strength [ Time Frame: Baseline, 1 and 2 years ]
    Quantitative Muscle Testing (QMT) will be used to describe the long-term evolution of quadriceps muscle strength.

  2. Change from baseline in patient-reported physical performance [ Time Frame: Baseline, 1 and 2 years ]
    The sIFA is a self-administered, patient reported questionnaire designed to assess the impact of sIBM on patient reported physical functioning. The sIFA consists of 11 items scored on an 11 point numerical rating scale from 0 (no difficulty) to 10 (unable to do) across three domains: upper body functioning, lower body functioning and general functioning.

  3. Incidence of patients with self-reported falls and self-reported injurious falls [ Time Frame: Baseline, 1 and 2 years ]
    Patients will document in a diary details of all falls that occur during the study. These data will be used to assess the incidence rate of self-reported falls and self-reported injurious falls.

  4. Change from baseline in physical performance [ Time Frame: Baseline, 1 and 2 years ]
    The Short Physical Performance Battery (SPPB) is a physical performance test which evaluates lower extremities function by using tests of gait speed, ability to keep standing balance, and time to rise from a chair five times. A five-level categorical score is used for each test (ranging from 0 = inability to complete the test and 4 = the highest level of performance). A summary performance score is created by summation of the scores for tests of standing balance (side by side stand, semi-tandem stand, and tandem stand), gait speed, and rising from a chair 5 times.

  5. Change in muscles of the thigh [ Time Frame: 1 and 2 years ]
    Magnetic resonance imaging (MRI) will be used to characterize changes in muscles of the thigh in a subset of patients.

  6. Number of patients who develop immunogenicity against BYM338 [ Time Frame: 2 years ]
    To investigate the development of immunogenicity against BYM338.


Eligibility Criteria

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Ages Eligible for Study:   36 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients who completed the core study
  • Written informed consent must be obtained before any extension study assessment is performed.
  • Able to communicate well with the investigator.
  • Willing to participate for the entire duration of the extension study with commitment to follow study requirements and procedures.

Exclusion Criteria:

  • Women who are pregnant
  • Women of child-bearing potential unless they are using highly effective methods of contraception during dosing and for 6 months after the last BYM338 dose.
  • Current use of prohibited treatments
  • History of severe hypersensitivity reaction in the core study
  • History of adverse event(s) (including those from the core study) prior to the start of study drug in the extension study that, in the judgment of the investigator, taking into account the subject's overall status, prevent the subject from entering the extension study
  • Clinically significant abnormal liver function tests
  • Any medical condition or laboratory finding which, in the opinion of the investigator may interfere with participation in the study, might confound the results of the study, or pose an additional safety risk in administering BYM338
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02573467


  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: NCT02573467     History of Changes
Other Study ID Numbers: CBYM338B2203E1
2015-001411-12 ( EudraCT Number )
First Posted: October 9, 2015    Key Record Dates
Last Update Posted: November 13, 2017
Last Verified: November 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
sporadic inclusion body myositis,
muscle wasting,
extension study,
BYM338,
bimagrumab,

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