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

What Was Tested

The trial (phase 3) is built around Bimagrumab.

The extension study was planned to consist of a Screening epoch (to assess patient eligibility), followed by a Treatment Period 1 epoch (double-blind and placebo-controlled), and a Treatment Period 2 epoch (open-label).

A Post-treatment Follow-up (FUP) epoch was also planned for patients who discontinued prematurely.

It had four arms (groups): one (experimental), one (experimental), one (experimental) and Placebo (placebo comparator).

The registry classes the interventions as drug.

Who Took Part

The registry lists 211 actual participants took part.

It was open to people of any sex aged from 36 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: 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.

The full record lists 4 inclusion and 7 exclusion criteria; among the exclusions: women who are pregnant; current use of prohibited treatments; history of severe hypersensitivity reaction in the core study.

How the Study Was Run

The set-up was non-randomized, parallel-group and quadruple-blind.

The Bimagrumab group and the comparison group ran side by side rather than switching.

As a double-blind Bimagrumab study, the assignments were hidden from both participants and investigators until the end.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2015-11-02 with completion listed for 2016-08-17.

It ran at 38 sites in United States (12), Japan (7), Italy (5), Australia (3), Belgium (3) and United Kingdom (3).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Change From Core Study Baseline in 6 Minute Walking Distance Test (6MWD) (time frame: Core study baseline, weeks 52, 78, 104, and >=117).

It also tracked 6 secondary outcomes, for example Change From Core Study Baseline in Quadriceps Quantitative Muscle Testing (QMT) on the Right Side, Change From Core Study Baseline in Sporadic Inclusion Body Myositis (sIBM) Functional Assessment (sIFA) Score, Estimated Annual Number of Falls Per Participant Within Treatment Group, Change From Core Study Baseline in Short Physical Performance Battery (SPPB) Score, Change in Muscles of the Thigh and Number of Patients With Anti-BYM338 Antibodies.

What This Means in Plain Language

Put simply, this is a phase 3, non-randomized trial of Bimagrumab in 211 people.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the Bimagrumab compound page, all human trials.