Transthyretin Amyloid CardiopathyPhase 4CACTuS - TMZNCT05633563
The Effect of Trimetazidine on Mitochondrial Function, Myocardial Performance, and Invasive Hemodynamics in Patients Diagnosed With Wild-Type Transthyretin Cardiac Amyloidosis
A Phase 4 study testing Trimetazidine in people with Transthyretin Amyloid Cardiopathy and Mitochondrial Pathology, run by Steen Hvitfeldt Poulsen at 1 site in Denmark.
Trial summary
Wild-type transthyretin cardiac amyloidosis (ATTRwt) is a deposition disorder in which one of the proteins of the body misfolds and accumulates at various places in the body, including the heart, leading to both mechanical and cellular damage. Read more about this study
- Who is it for?
- Ages 60 years and older
- Conditions the study is for
- Transthyretin Amyloid Cardiopathy, Mitochondrial Pathology
- What is being tested?
- Trimetazidine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 1 siteDenmark
- Study dates
- 1 Oct 2021to 1 Jun 2023
- Phase
- Phase 4
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Wild-type transthyretin cardiac amyloidosis
- NAC stage I
- NYHA class of I or II
- Informed consent
Who cannot join
- Other, similar diagnoses
- Hereditary transthyretin cardiac amyloidosis
- Light chain amyloidosis
- Morbus Waldenstrøm
- Myelomatosis
- Medical treatment with loop diuretics in standard doses (40 mgx1 daily)
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- Contraindications to trimetazidine
- Significant comorbidity assessed by the investigators
- Unable to provide informed consent
Trial locations and contacts
1 site in 1 country.
Denmark1 site
Aarhus University Hospital, Department of Cardiology
Aarhus N, Danmark, 8200
About this study
Wild-type transthyretin cardiac amyloidosis (ATTRwt) is a deposition disorder in which one of the proteins of the body misfolds and accumulates at various places in the body, including the heart, leading to both mechanical and cellular damage. The gradual development of the disease will ultimately lead to heart failure and death
The protein which deposits in the heart of patients, damages both the heart mechanically as the myocardium becomes rigid and hypertrophic over time but also at the cellular level. Cell damage can be observed by elevated blood tests for cell damage (Troponin) and during exercise tests that show patients' hearts burning oxygen inefficiently when exposed to physical stress compared with the hearts of healthy individuals . No one has, however, intimately studied this cellular damage.
Read more
Vastarel® (Trimetazidine, TMZ) is an already known drug for the treatment of chest pain. The mechanism of action indicates that it may have an effect on patients with cardiac amyloidosis.
The study aims to investigate the effects of TMZ on the mitochondrial function, myocardial performance, and invasive hemodynamics in patients with ATTRwt with a randomized, double-blinded, crossover-trial.
- Being tested
- Trimetazidine, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TrimetazidineDrug
Oral intake of capsules
PlaceboDrug
Oral intake of capsules
| Study group | Role | What they receive |
|---|---|---|
| Active Drug | Active comparator | Study participants receiving Trimetazidine Receives: Drug: Trimetazidine |
| Placebo | Placebo comparator | Study participants receiving placebo (calcium) Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change: pulmonary capillary wedge pressure (PCWP)
We hypothesize a change in PCWP of 5 mmHg between the active drug and placebo using right heart catheterization.
Measured: Four weeks of treatment
Secondary outcomes (1)
Change: cardiac index (CI)
We hypothesize a change in CI of 0.5 L/min between the active drug and placebo using right heart catheterization.
Measured: Four weeks of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Dec 2022
- Study start1 Oct 2021
- Primary completion1 Jun 2023
- Study completion1 Jun 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin Amyloid CardiopathyStudies of the same condition, open and under way.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Steen Hvitfeldt Poulsen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Steen Hvitfeldt Poulsen, Aarhus University Hospital Skejby
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05633563
- Sponsor's protocol ID
- 72864
- Acronym
- CACTuS - TMZ
- Status last verified
- Apr 2024
- First submitted
- 15 Oct 2021
- Last updated
- 25 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 25 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Trimetazidine on Mitochondrial Function, Myocardial Performance, and Invasive Hemodynamics in Patients Diagnosed With Wild-Type Transthyretin Cardiac Amyloidosis
- ClinicalTrials.gov ID
- NCT05633563
- Acronym / sponsor ID
- CACTuS - TMZ / 72864
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Steen Hvitfeldt PoulsenUniversity, hospital or other
- Study start
- 1 Oct 2021 (actual)
- Primary completion
- 1 Jun 2023 (actual)
- Study completion
- 1 Jun 2023 (actual)
- First posted
- 1 Dec 2022 (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.
Wild-type transthyretin cardiac amyloidosis (ATTRwt) is a deposition disorder in which one of the proteins of the body misfolds and accumulates at various places in the body, including the heart, leading to both mechanical and cellular damage. The gradual development of the disease will ultimately lead to heart failure and death
The protein which deposits in the heart of patients, damages both the heart mechanically as the myocardium becomes rigid and hypertrophic over time but also at the cellular level. Cell damage can be observed by elevated blood tests for cell damage (Troponin) and during exercise tests that show patients' hearts burning oxygen inefficiently when exposed to physical stress compared with the hearts of healthy individuals . No one has, however, intimately studied this cellular damage.
Vastarel® (Trimetazidine, TMZ) is an already known drug for the treatment of chest pain. The mechanism of action indicates that it may have an effect on patients with cardiac amyloidosis.
The study aims to investigate the effects of TMZ on the mitochondrial function, myocardial performance, and invasive hemodynamics in patients with ATTRwt with a randomized, double-blinded, crossover-trial.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Active Drug | Active comparator | Drug: TrimetazidineStudy participants receiving Trimetazidine |
| Placebo | Placebo comparator | Drug: PlaceboStudy participants receiving placebo (calcium) |
Who may qualify
- Ages
- 60 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Wild-type transthyretin cardiac amyloidosis
- NAC stage I
- NYHA class of I or II
- Informed consent
Key exclusion criteria (9 listed)
- Other, similar diagnoses
- Hereditary transthyretin cardiac amyloidosis
- Light chain amyloidosis
- Morbus Waldenstrøm
- Myelomatosis
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 |
|---|---|---|
| TrimetazidineOral intake of capsules | Drug | Active Drug |
| PlaceboOral intake of capsules | Drug | Placebo |
Primary outcome
Change: pulmonary capillary wedge pressure (PCWP)
Time frame: Four weeks of treatment
We hypothesize a change in PCWP of 5 mmHg between the active drug and placebo using right heart catheterization.
1 secondary outcome
Change: cardiac index (CI)
Time frame: Four weeks of treatment
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 25 Apr 2024.
- Individual participant dataSharing planned: Undecided
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HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Trimetazidine in people with Transthyretin Amyloid Cardiopathy and Mitochondrial Pathology, run by Steen Hvitfeldt Poulsen at 1 site in Denmark.
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 4 inclusion and 9 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 25 Apr 2024. 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 Denmark. 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. Some participants in this study may receive a placebo. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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: pulmonary capillary wedge pressure (PCWP) (time frame: Four weeks of treatment). 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 25 Apr 2024.