Heart FailurePhase 1NCT04996719
Effect of Rapamycin to Improve Cardiac Function in Frail Older Adults
A Phase 1 study testing Rapamune in people with Heart Failure, run by Mayo Clinic at 1 site in United States.
Trial summary
The purpose of this research is to see if the drug rapamycin will improve the heart's ability to pump by improving your oxygen consumption. Read more about this study
- Who is it for?
- Ages 60 to 99 years
- Conditions the study is for
- Heart Failure
- What is being tested?
- Rapamune
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 24 May 2022to 25 Aug 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 60 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥60 years
- Diagnosis of HFpEF by either H2FEPF score of 6-9 or HFA-PEFF score of 5-6, or invasively confirmed diagnosis of HFpEF (resting pulmonary capillary wedge pressure ≥15mmHg or ≥25mmHg with exercise).
- Diagnosis of computable phenotype of frailty rests on screening questions about ability to climb two flights of stairs, activities of daily living, living environment, wearing of dentures/hearing aids, and whether the patient uses assistive devices.
Who cannot join
- Reduced ejection fraction (≤50%) with or without prior history of myocardial infarction
- Acute coronary syndrome <3 months
- Uncontrolled diabetes mellitus (HbA1C>8)
- Uncontrolled hypertension (>160/100mmHg, despite 3 antihypertensive medications)
- Nephrotic syndrome or eGFR <30mL/min/1.73m2
- Cirrhosis
Show all 13Show fewer
- Hepatitis B/C positive
- Elevated lived enzymes (AST/ALT>3ULN
- Prior malignancy other than basal cell carcinoma
- History of ongoing, chronic or recurrent infectious disease
- Suspected/proven immunocompromised state
- Uncontrolled hyperlipidemia (fasting TG >400 mg/dL (>4.5 mmol/L) or total cholesterol >300 mg/dL (>7.8 mmol/L) despite maximum lipid lowering therapy
- Class IV HF symptoms
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Effect of Rapamycin to Improve Cardiac Function in Frail Older Adults With Heart Failure With Preserved Ejection Fraction: A Randomized Pilot and Proof-of-Concept Study
The purpose of this research is to see if the drug rapamycin will improve the heart's ability to pump by improving your oxygen consumption.
Read the detailed description
This will be a pilot, proof-of-concept study. Patients 60 years or older with invasively proven HFpEF or have definite HFpEF as determined by the 2 established scores and frailty (positive screen for computable phenotype), will be approached for enrollment. Subjects meeting screening criteria following written informed consent will be randomized to receive rapamycin or matching placebo (study drug). Efficacy outcomes will be measured at baseline and at 6 months. Safety will be assessed by in person or remote video monitoring weekly for 1 month, then monthly for 5 months till the end of the study. We will also measure trough rapamycin levels (≤4ng/mL) twice-a-month for the first month and then monthly for 5 months.
- Conditions
- Heart Failure
- Being tested
- Rapamune
- Medical subject headings
- Heart Failure
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RapamuneDrug
Also called Rapamycin
We will start the dose at 0.5mg capsule every other day for 2 weeks. If participants tolerate this dose and trough blood levels are (≤4ng/mL), the dose of rapamycin will be increased to 0.5mg daily for the duration of the study. If necessary, dose can remain at 0.5 mg every other day, depending on the trough level. To avoid immunosuppression, we will target lower dose of rapamycin, maintain lower trough serum levels (≤4ng/mL).
| Study group | Role | What they receive |
|---|---|---|
| Rapamycin | Experimental | find safe doses for patients who have heart failure with preserved ejection fraction Receives: Drug: Rapamune |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change cardiopulmonary reserve capacity by improving peak VO2 with rapamycin
In this pilot, proof-of-concept study, we hypothesize that in older adults (≥60 years) with HFpEF who are frail, chronic rapamycin administration will change their cardiopulmonary reserve capacity by improving peak VO2.Primary safety objective is the safety of rapamycin.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2021
- Study start24 May 2022
- Primary completion25 Aug 2023
- Study completion25 Aug 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Heart FailureStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mandeep Singh, Mayo Clinic
- Investigators
- Mandeep Singh, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04996719
- Sponsor's protocol ID
- 21-003837
- Status last verified
- Sep 2023
- First submitted
- 3 Aug 2021
- Last updated
- 28 Sep 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Rapamycin to Improve Cardiac Function in Frail Older Adults With Heart Failure With Preserved Ejection Fraction: A Randomized Pilot and Proof-of-Concept Study
- ClinicalTrials.gov ID
- NCT04996719
- Acronym / sponsor ID
- 21-003837
- Conditions
- Heart Failure
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- 24 May 2022 (actual)
- Primary completion
- 25 Aug 2023 (actual)
- Study completion
- 25 Aug 2023 (actual)
- First posted
- 9 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this research is to see if the drug rapamycin will improve the heart's ability to pump by improving your oxygen consumption.
Detailed description
This will be a pilot, proof-of-concept study. Patients 60 years or older with invasively proven HFpEF or have definite HFpEF as determined by the 2 established scores and frailty (positive screen for computable phenotype), will be approached for enrollment. Subjects meeting screening criteria following written informed consent will be randomized to receive rapamycin or matching placebo (study drug). Efficacy outcomes will be measured at baseline and at 6 months. Safety will be assessed by in person or remote video monitoring weekly for 1 month, then monthly for 5 months till the end of the study. We will also measure trough rapamycin levels (≤4ng/mL) twice-a-month for the first month and then monthly for 5 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Rapamycin | Experimental | Drug: Rapamunefind safe doses for patients who have heart failure with preserved ejection fraction |
Who may qualify
- Ages
- 60 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Age ≥60 years
- Diagnosis of HFpEF by either H2FEPF score of 6-9 or HFA-PEFF score of 5-6, or invasively confirmed diagnosis of HFpEF (resting pulmonary capillary wedge pressure ≥15mmHg or ≥25mmHg with exercise).
- Diagnosis of computable phenotype of frailty rests on screening questions about ability to climb two flights of stairs, activities of daily living, living environment, wearing of dentures/hearing aids, and whether the patient uses assistive devices.
Key exclusion criteria (13 listed)
- Reduced ejection fraction (≤50%) with or without prior history of myocardial infarction
- Acute coronary syndrome <3 months
- Uncontrolled diabetes mellitus (HbA1C>8)
- Uncontrolled hypertension (>160/100mmHg, despite 3 antihypertensive medications)
- Nephrotic syndrome or eGFR <30mL/min/1.73m2
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RapamuneAlso: RapamycinWe will start the dose at 0.5mg capsule every other day for 2 weeks. If participants tolerate this dose and trough blood levels are (≤4ng/mL), the dose of rapamycin will be increased to 0.5mg daily for the duration of the study. If necessary, dose can remain at 0.5 mg every other day, depending on the trough level. To avoid immunosuppression, we will target lower dose of rapamycin, maintain lower trough serum levels (≤4ng/mL). | Drug | Rapamycin |
Primary outcome
Change cardiopulmonary reserve capacity by improving peak VO2 with rapamycin
Time frame: 6 months
In this pilot, proof-of-concept study, we hypothesize that in older adults (≥60 years) with HFpEF who are frail, chronic rapamycin administration will change their cardiopulmonary reserve capacity by improving peak VO2.Primary safety objective is the safety of rapamycin.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2023.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mandeep Singh, MD principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Rapamune in people with Heart Failure, run by Mayo Clinic at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change cardiopulmonary reserve capacity by improving peak VO2 with rapamycin (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Sep 2023.