Study of Long-term Safety, Efficacy Tolerability of BYM338 in Patients With Sporadic Inclusion Body Myositis

What Was Tested

In this phase 2, phase 3 trial, researchers set out to test Bimagrumab.

It had 1 arm (groups): BYM338 (experimental).

BYM338 Group.

The registry classes the interventions as drug.

More on the Plan

This is a non-confirmatory, multicenter, open-label, non-randomized trial which will extend active treatment to those patients that participated in the preceding proof-of-concept study (CBYM338X2205) in order to collect long-term safety and tolerability data.

Up to 14 patients with sIBM will be invited to enroll into the study to receive BYM338 open-label for a period of approximately 3 years or until BYM338 is commercially available, whichever comes first.

All patients will be administered a medium level i.v.

Who Took Part

10 actual participants took part.

It was open to people of any sex aged from 40 years up to 75 years.

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

Examples of who could join: Written informed consent must be obtained before any assessment is performed. Patients who participated in the CBYM338X2205 study. A patient is defined as participating in the study if they were enrolled and received study medication. Able to communicate well with the investigator, to understand and comply with the requirements of the study.

The full record lists 3 inclusion and 21 exclusion criteria; among the exclusions: history of hypersensitivity to any of the study drugs or to drugs of similar chemical classes; use of a combination of any two of the following (a+b or a+c or b+c):; use of oral, injected or implanted hormonal methods of contraception.

How the Study Was Run

It was run as a single-group and open-label study.

Nothing was hidden in this open-label Bimagrumab study: the participants and staff both knew the treatment.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2014-03-11 with completion listed for 2016-08-23.

It ran at 2 sites in United States (2).

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Number of Participants With Adverse Events as a Measure of Safety and Tolerability (time frame: Up to 29 month).

It also tracked 9 secondary outcomes, for example Changes From Baseline in Lean Body Mass (LBM) by Dual-Energy X-ray Absorptiometery (DXA), Changes From Baseline in Physical Function Reported by Patients, Changes From Baseline in Muscle Strength, Changes From Baseline in Muscle Function (Hand-grip and Pinch-grip Dynamometry), Changes From Baseline in Muscle Function 6 Minute Walking Distance and Change From Baseline of Thigh Muscle Volume (TMV) by MRI Scan.

What This Means in Plain Language

Put simply, this is a phase 2, phase 3 trial of Bimagrumab in 10 people.

Its size is small, so on its own it can only give an early look.

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.