E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Sarcopenia including sarcopenic obesity |
|
E.1.1.1 | Medical condition in easily understood language |
Sarcopenia including sarcopenic obesity |
|
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 |
|
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 |
1. To evaluate the safety and efficacy of two doses of BIO101 (175
mg b.i.d. and 350 mg b.i.d.) orally administered for 26 weeks
versus placebo in a population of community dwelling older men
and women (aged ≥ 65 years) living at home and at risk of
mobility disability.
2. To estimate treatment effect on improvement of physical
function after a six-month treatment versus placebo in the target
population.
3. To estimate treatment effect on reduced risk of mobility
disability after a six-month treatment versus placebo in the
target population. |
|
E.2.2 | Secondary objectives of the trial |
a. To compare the change from baseline of a standardized patient
reported outcome (PRO): the PF-10 sub-score of the SF-36. A
minimum clinically significant benefit is set at 2-point difference
of the change at M6 from baseline versus placebo in the mean
difference between groups.
b. To compare the change from baseline to month 6 of the muscle
strength using the handgrip strength test. A minimum clinically
significant benefit is set at 2 kg difference of the change at M6
from baseline versus placebo in the mean difference between
groups |
|
E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
A population PK sub-study (SARA-POP-PK), with the objective of assessing pharmacokinetic values after 1 month, 3 months or 6 months in a subset of participants in the European centers. |
|
E.3 | Principal inclusion criteria |
1. Provision of signed and dated informed consent form (ICF)
2. Stated willingness to comply with all study procedures and
availability for the duration of the study
3. Male or female aged ≥ 65 years, living in the community
(living at home, able to walk outside from time to time. Homes
can include any living community that may or may not offer
optional services for the convenience of the residents. Older
adults living in nursing homes or in medicalized residences
where caretaker services are mandatory are not eligible) and
reporting a loss of physical function over the last 6-12
months
4. SPPB score ≤ 8
5. ALM/BMI < 0.789 in men and <0.512 in women, or ALM <
19.75kg in men and < 15.02kg in women, as measured by
DEXA scan
6. Ability to take oral medication and be willing to adhere to the
study intervention regimen (see section 6)
7. Agreement to adhere to the outlined Lifestyle Considerations
(see section 5.4) throughout the study duration
8. In the US, women and members of minority groups should not
be excluded, in accordance with the NIH Policy on Inclusion of
Women and Minorities as Participants In Research Involving
Human Subjects. |
|
E.4 | Principal exclusion criteria |
1. Current use of anabolic drugs (e.g. testosterone); current use
of Erythropoietin; current use of corticosteroid agents (except ocal administration route, like eye drops or dermatologic formulations)
2. Non-menopaused women (however, ongoing hormonal
replacement hormonal treatment is not an exclusion criterion)
3. Known allergic reactions to sourcing components of the investigational drug (i.e. Cyanotis species; e.g. Cyanotis arachnoidea C.B. Clarke or Cyanotis vaga Lour. (Shultes)
leaves and roots)
4. Treatment with another investigational drug or other interventions within three months
5. Unable to understand and perform the functional tests, as judged by the Investigator
6. Inability to perform the 400MW test within 15 minutes
7. Clinical conditions:
a. Current diagnosis of major psychiatric disorders.
b. Alcohol abuse or dependence
c. Severe arthritis
d. Cancer requiring active treatment (cancer previously
treated with chemotherapy and/or radiotherapy and
participants currently on remission is not an exclusion
criterion)
e. Lung disease requiring regular use of supplemental
oxygen
f. Inflammatory conditions requiring regular use of oral
or parenteral corticosteroid agents
g. Severe cardiovascular disease (including New York
Heart Association [NYHA] class III or IV congestive
heart failure, clinically significant valvular disease,
history of cardiac arrest, presence of an implantable
defibrillator, or uncontrolled angina)
h. Parkinson’s disease or other progressive neurological
disorder
i. Renal disease requiring dialysis, or known renal
insufficiency (moderate or severe reduction of
eGFR≤30 ml/min/1.73 m2, based on Cockroft & Gault
formula)
j. Chest pain, severe shortness of breath, or occurrence
of other safety concerns during the baseline
functional tests such as the 400MW test
k. History or active signs or symptoms of
gallbladder/biliary disease (e.g. previous episodes of
cholestasis/biliary tract obstruction, cholelithiasis,
cholecystitis, etc.). Of note, history of
cholecystectomy and no active biliary signs or
symptoms, is not an exclusion criterion.
8. Current physical/rehabilitation therapy (except for passive
physical therapy. However, this should not be initiated the
week before an evaluation visit and once started, it should be
maintained over the study duration). |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Gait speed measured during the 400MW test, the change from baseline to month 6 will be compared between groups of treatment (each dose versus placebo). |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
PF-10 subscore of the SF-36: the change from baseline to month 6 will be compared between groups of treatment (each dose versus placebo).
Muscle strength as measured by the handgrip test: the change from
baseline to month 6 will be compared between groups of treatment
(each dose versus placebo) |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
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 | No |
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 | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
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 | No |
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 | 3 |
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 | No |
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 |
|
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 | 2 |
E.8.9.1 | In the Member State concerned months | 11 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 11 |