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 |
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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 | 20.1 |
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 |
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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 effect of 24-weeks of bimagrumab treatment on patient physical function assessed by a change in the Short Physical Performance Battery (SPPB) total score from baseline to week 25 relative to placebo in older adults with sarcopenia. |
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E.2.2 | Secondary objectives of the trial |
- assess safety and tolerability of bimagrumab variables,
- assess the effect of bimagrumab compared to placebo on the change from baseline to week 25 in the 6-Minute Walk Test (6MWT) distance
- assess the effect of bimagrumab compared to placebo on the change from baseline at week 25 in usual gait speed
- assess the effect of bimagrumab on total lean body mass and appendicular skeletal muscle index (ASMI) as assessed by change from baseline to week 25 compared to placebo |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
•Low muscle mass as confirmed by DXA;
•Low gait speed;
•Weigh at least 35 kg;
•Adequate dietary intake;
• Total SPPB score of less than or equal to 9 at screening and baseline.
Other protocol-defined inclusion criteria may apply. |
|
E.4 | Principal exclusion criteria |
•A lower limb fracture in the past 6 months or any impairment or disease severely affecting gait (e.g. stroke with hemiparesis, myasthenia gravis, Parkinson's disease, peripheral polyneuropathy, intermittent claudication in advanced peripheral vascular disease, spinal stenosis, or severe osteoarthritis of the knee or hip with ineffective pain management);
•Confirmed diagnosis of heart failure classified as New York Heart Association Class III and IV (e.g. dilated cardiomyopathy);
•Type I diabetes
•Requires regular assistance from another person for general activities of daily living (e.g. bathing, dressing).
Other protocol-defined exclusion criteria may apply.
|
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E.5 End points |
E.5.1 | Primary end point(s) |
Short Physical Performance Battery (SPPB) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
- Safety and tolerability
- 6MWT
- Gait speed
- Total lean body mass and appendicular skeletal muscle index measured by DXA |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
- Safety and tolerability: baseline, weeks 1, 5, 9, 13, 17, 21 and 25
6MWT; Gait speed; and Total lean body mass and appendicular skeletal muscle index measured by DXA: week 25 |
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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 | Yes |
E.6.10 | Pharmacogenetic | Yes |
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 |
tolerability and biomarker assessments |
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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 | No |
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 | 4 |
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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 15 |
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 |
Australia |
European Union |
Japan |
Korea, Republic of |
Russian Federation |
Switzerland |
Taiwan |
United States |
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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
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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 | 5 |
E.8.9.1 | In the Member State concerned days | 13 |
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 |