Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12622000480785p
Ethics application status
Submitted, not yet approved
Date submitted
5/03/2022
Date registered
25/03/2022
Date last updated
29/07/2024
Date data sharing statement initially provided
25/03/2022
Type of registration
Prospectively registered

Titles & IDs
Public title
Rapamycin impact on muscle strength and endurance in older adults
Scientific title
A Single-Centre, Double-Blind, Randomized, Placebo-Controlled 2-Arm Study to Evaluate Safety and Efficacy of Intermittent Rapamycin On Muscle Strength and Endurance In Older Adults Following A 13-Week Exercise Program
Secondary ID [1] 306604 0
Nil known
Universal Trial Number (UTN)
U1111-1279-0901
Trial acronym
RAPA-EX
Linked study record

Health condition
Health condition(s) or problem(s) studied:
sarcopenia 325513 0
Condition category
Condition code
Musculoskeletal 322889 322889 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
The study evaluates the change in muscle performance, as measured by the 30-Second Chair-Stand Test (30CST), following a 13-week exercise regimen combined with weekly dosing of Rapamycin, compared to placebo and exercise.
The assessment will establish whether weekly administration of Rapamycin, which inhibits the mechanistic target of the Rapamycin (mTORC1) pathway, impacts the beneficial effects of exercise on muscle performance.
Intervention code [1] 323031 0
Prevention
Intervention code [2] 323032 0
Treatment: Drugs
Intervention code [3] 323033 0
Lifestyle
Comparator / control treatment
The 20 participants in the control group will also complete the 13-week, thrice-weekly, at-home exercise program, but will take a matching placebo instead of the Sirolimus (Rapamycin) once a week. The matching placebo will be manufactured by CompoundLabs
Control group
Placebo

Outcomes
Primary outcome [1] 330668 0
General muscle performance as measured by the 30-Second Chair-Stand Test (30CST)
Timepoint [1] 330668 0
The 30-Second Chair-Stand Test (30CST) will be measured at baseline and after 13 weeks post commencing treatment.
Secondary outcome [1] 407129 0
To investigate the safety and tolerability of low dose Rapamycin in exercising older adults.
• Incidence, relatedness, seriousness and severity of adverse events:
- Clinical laboratory tests (Full Blood Count, U&Es, LFTs, HbA1c, lipids, serum IGF-1) at baseline and EOS
- Vital sign measurements
- Adverse events that impact exercise ability or dosing or serious adverse events
Timepoint [1] 407129 0
Measured at baseline and after 13 weeks post commencing treatment.
Secondary outcome [2] 407130 0
To evaluate change of muscle strength and muscle endurance from baseline to EOS.
The muscle strength and endurance measures are:
• Change in the 30-Second Chair-Stand Test
• Change in the 6-minute walk test
• Change from baseline in handgrip strength
Timepoint [2] 407130 0
Measured at baseline and after 13 weeks post commencing treatment.
Secondary outcome [3] 407131 0

Participant reported outcomes

• Observed change from baseline in self-reported measures of health as determined by the 36-Item Short Form Survey (SF-36) through EOS
• Participant reported physical activity from baseline to EOS.
Timepoint [3] 407131 0
Measured at baseline and after 13 weeks post commencing treatment.
Secondary outcome [4] 407132 0
Change in DNA methylation from baseline to week 13, determined using blood samples
Timepoint [4] 407132 0
Measured at baseline and after 13 weeks post commencing treatment.

Eligibility
Key inclusion criteria
1. Male or female aged 65 years to 85 years at the time of signing informed consent
2. BMI between 18 and 40 and a maximum weight of 120kg at screening.
3. Currently sedentary lifestyle or performing moderate exercise for less than 15 mins, 3 times a week
4. Capable of providing written informed consent
5. Willing to swallow a #000 sized capsule
6. Willing and able to adhere to and comply with all study requirements, and attend all study visits
7. Able to complete the 30 second Chair Stand Test utilising correct technique
8. Is able to accommodate and use an exercycle at home for the duration of study participation
Minimum age
65 Years
Maximum age
85 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1. Anaemia - Hg < 9.0 g/dl, Leukopenia - white blood cells (WBC) < 3,500/mm3, Neutropenia - absolute neutrophil count < 2,000/mm3 , or Platelet count - platelet count < 125,000/mm3
2. Planned surgery during the period of the study that is likely to impact on ability to perform required study exercises
3. Any medical or psychological condition which in the opinion of the investigator, may interfere with the participants ability to and comply with the study requirements and/or put the participant at significant risk.
4. Impaired wound healing or history of a chronic open wound.
5. Active infection at the time of signing consent.
6. Malignancy (except non-melanoma skin cancers, cervical carcinoma in-situ) within the last 5 years.
7. Known hypersensitivity, allergy, or any contraindication to Rapamycin or placebo (cellulose powder) or its excipients
8. Fibromyalgia or Chronic Fatigue Syndrome/Myalgic Encephalomyelitis, Breast Implant Illness, or other conditions that impact the participants ability to perform the exercise program
9. Known congestive heart failure with New York Heart Association (NYHA) classification III or IV
10. COPD Global Initiative for Chronic Obstructive Lung Disease (GOLD) classification III or IV
11. Impaired renal function, as defined as glomerular filtration rate eGFR < 30
12. Type 1 diabetes or uncontrolled Type 2 Diabetes (defined as HbA1c at least 60 mmol/mol)
13. Metformin, Rapamycin, or rapalogs use within 6 months prior to baseline.
14. Impaired hepatic function, measured by alkaline Phosphatase (ALP), alanine aminotransferase (ALT), Albumin, or T. Bili, whereby the levels are 1.5x greater than the normal upper limit.
15. Any form of clinically relevant primary or secondary immune dysfunction or deficiency (e.g. X-linked agammaglobulinemia (XLA), common variable immunodeficiency (CVID))
16. Chronic oral corticosteroid or immunosuppressive medication use (e.g. Enbrel, Humira, methotrexate).
17. Participation in any other study (for 30 days) prior to or during this study.

Study design
Purpose of the study
Prevention
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Withdrawn
Reason for early stopping/withdrawal
Other reasons/comments
Other reasons
There were too many changes required. A new Study was registered.
This was done on advice from ANZCTR staff.
No participants were enrolled in this study.
Date of first participant enrolment
Anticipated
5/02/2024
Actual
Date of last participant enrolment
Anticipated
5/08/2024
Actual
Date of last data collection
Anticipated
7/10/2024
Actual
Sample size
Target
40
Accrual to date
Final
Recruitment outside Australia
Country [1] 24639 0
New Zealand
State/province [1] 24639 0

Funding & Sponsors
Funding source category [1] 310941 0
Other Collaborative groups
Name [1] 310941 0
Vitasang Ltd
Country [1] 310941 0
United States of America
Funding source category [2] 310942 0
Other Collaborative groups
Name [2] 310942 0
none
Country [2] 310942 0
New Zealand
Primary sponsor type
Other Collaborative groups
Name
Dr Brad Stanfield Ltd
Address
3456 Paul Anka Drive. Ottawa, ON K1V 9K6
Country
New Zealand
Secondary sponsor category [1] 312234 0
None
Name [1] 312234 0
Address [1] 312234 0
Country [1] 312234 0

Ethics approval
Ethics application status
Submitted, not yet approved
Ethics committee name [1] 310494 0
Health and Disability Ethics Committee
Ethics committee address [1] 310494 0
Ethics committee country [1] 310494 0
New Zealand
Date submitted for ethics approval [1] 310494 0
11/04/2024
Approval date [1] 310494 0
Ethics approval number [1] 310494 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 117870 0
Dr Joanna Wojciechowska
Address 117870 0
Aotearoa Clinical Trials Trust
Esme Green Building
Middlemore Hospital
100 Hospital Rd
Papatoetoe, 2025
Auckland, New Zealand
Country 117870 0
New Zealand
Phone 117870 0
+64 9 270 9758
Fax 117870 0
Email 117870 0
joanna.wojciechowska@middlemore.co.nz
Contact person for public queries
Name 117871 0
Dr Brad Stanfield
Address 117871 0
Tuakau Health Centre
55 George Street, Tuakau, Auckland, 2121
Country 117871 0
New Zealand
Phone 117871 0
+64210426045
Fax 117871 0
Email 117871 0
brad@drstanfield.com
Contact person for scientific queries
Name 117872 0
Dr Brad Stanfield
Address 117872 0
Tuakau Health Centre
55 George Street, Tuakau, Auckland, 2121
Country 117872 0
New Zealand
Phone 117872 0
+64210426045
Fax 117872 0
Email 117872 0
brad@drstanfield.com

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Anonymised patient result data, demographic data, exercise diary data, adherence to placebo/Sirolimus data
When will data be available (start and end dates)?
The data will be available after the trial is submitted to a clinical journal, and there will be no end date.
Available to whom?
The data will be available upon reasonable request
Available for what types of analyses?
Meta-analysis and auditing
How or where can data be obtained?
Contact the trial Sponsor at brad@drstanfield.com


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.