Clinical Trial: 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

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

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 Myosi

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.

Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome:

• 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
• 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.

Original Primary Outcome: Same as current

Current Secondary Outcome:

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

Original Secondary Outcome: Same as current

Information By: Novartis

Dates:
Date Received: July 9, 2015
Date Started: November 2015
Date Completion: February 2017
Last Updated: August 15, 2016
Last Verified: August 2016