E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
Low muscle mass and gait speed in elderly subjects |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10063024 |
E.1.2 | Term | Sarcopenia |
E.1.2 | System Organ Class | 10028395 - Musculoskeletal and connective tissue disorders |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the durability of effect of bimagrumab (BYM338) as measured by the short physical performance battery (SPPB) total score at week 49. |
|
E.2.2 | Secondary objectives of the trial |
- To evaluate the safety and tolerability of bimagrumab following treatment withdrawal as assessed by measures such as vital signs, clinical laboratory variables, electrocardiogram (ECG), and adverse
events (AE).
-To evaluate the durability of effect of bimagrumab at week 49 as measured by:
o 6 minute walk test (6MWT)
o mobility as measured in gait speed (GS) over 4 meters
o total lean body mass (LBM) and appendicular skeletal muscle index (ASMI) measured by dual energy X-ray absorptiometry (DXA) |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Men and postmenopausal women aged 70 years or older that have participated in, and have completed the full study treatment period per protocol (24 weeks/EOT visit) in the preceding core study (CBYM338E2202).
Other protocol-defined inclusion criteria may apply. |
|
E.4 | Principal exclusion criteria |
This is an extension study and has the same exclusions as the core study. Because only participants who complete the core study are eligible for inclusion in this study, patients who no longer qualify or
should be discontinued from the core study are excluded from participating in this study. Patients fulfilling the following criterion are not eligible for inclusion in this study:
1. Any condition which should have led to treatment discontinuation per protocol in the core study
No additional exclusions may be applied by the investigator, in order to ensure that the study population will be representative of all eligible patients, within and across study sites. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
short physical performance battery (SPPB) total score |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
- Safety and tolerability
- 6 minute walk test
- Gait speed
- Total lean body mass and appendicular skeletal muscle index measured by DXA |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
-Safety and tolerability: Week 25, 37, 49
-6 minute walk test; Gait speed; Total lean body mass and appendicular skeletal muscle index measured by DXA: Week 49 |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Durability of benefit of bimagrumab |
|
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Relevant for pts enrolled prior protocol v1. Off drug parallel group study for those enrolled after |
|
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 8 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 11 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
European Union |
Japan |
Switzerland |
Taiwan |
United States |
|
E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 1 |