Cardiomyopathy, DilatedPhase 3EARLY-GENENCT05321875
Early Treatment With Candesartan vs Placebo in Genetic Carriers of Dilated Cardiomyopathy (EARLY-GENE Trial)
A Phase 3 study testing Candesartan in people with Cardiomyopathy, Dilated, run by Cristina Avendaño Solá at 1 site in Spain.
Trial summary
Prospective, multicenter, randomized, placebo-controlled, double-blind clinical trial to evaluate safety and efficacy of candesartan in the prevention of the development of Dilated Cardiomyopathy (DCM) in genetic carriers of a DCM-causing variant without disease expression (asymptomatic) Read more about this study
- Who is it for?
- Ages 18 to 64 years
- Conditions the study is for
- Cardiomyopathy, Dilated
- What is being tested?
- Candesartan
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 320 participants
- Where
- 1 siteSpain
- Study dates
- 2 Jun 2022to 2 Jun 2026
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 18 to 64 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age: 18-64 (both included), both sexes
- Carrier of a pathogenic or likely pathogenic DCM genetic variant1 according to modified American College of Medical Genetics (ACMG) criteria.
- Baseline LVEF ≥ 50% measured by MRI1 and evaluated by the eligibility study committee. Carriers with myocardial fibrosis, detected by late gadolinium enhancement in magnetic resonance imaging, are valid.
- Baseline creatinine ≤1.3 mg/dL, potassium ≤ 5.3 mEq/L and an estimated Glomerular Filtration Rate (eGFR)≥ 60 ml/min/1.73 m2 calculated by CKD-EPI formula.
- Able to understand and accept the study constraints and to provide informed consent.
Who cannot join
- Hypotension (systolic arterial pressure <100 mmHg (measured following a standardized methodology).
- Prior ventricular dysfunction (LVEF ≤ 50% at any time prior to study inclusion)
- Candidates who are expected or highly likely to receive an implantable cardioverter defibrillator (ICD) in the following 12 months after inclusion in the trial
- Preexisting hypertension requiring pharmacological treatment.
- Uncontrolled arterial hypertension (i.e., repeatedly systolic arterial pressure > 140 mmHg).
- Carriers of TTN-truncating variants (TTNtv) who are < 35 years old.
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- Known clinically significant coronary artery disease (e.g., ≥70% stenosis in any epicardial artery or ≥50% of left main coronary artery), valvular disease (≥ moderate in severity) or ventricular arrhythmias.
- Ongoing treatment with ACEI, ARB, ARNI or MRA.
- Prior intolerance to ACE inhibitors or ARB.
- Presence of any contraindications to receive candesartan treatment, including severe liver failure and/or cholestasis
- Known bilateral renal artery stenosis.
- Uncontrolled concomitant severe disease (e.g., with expected survival inferior to the duration of the study follow-up)
- Participation in any other clinical trial using an investigational medicinal product or device in the 30 days previous to the inclusion in the study.
- Current pregnancy, breastfeeding or women of childbearing age who are not willing to practice an adequate birth control during the entire duration of the study (a negative pregnancy test result must be confirmed at the time of enrolment)*.
- Drug or alcohol abuse (current).
- Inability to comply with study procedures and treatments.
- Carriers of MRI incompatible internal devices (ICD, pacemakers, aneurysm clips, etc.), with known intolerance to MRI studies or presenting any contraindications to perform cardiac MRI studies.
- Any circumstances that in the investigator's opinion compromise the participant's ability to participate in the clinical trial.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Spain1 site, 1 recruiting
- Recruiting
Hospital Universitario Puerta de Hierro-Majadahonda
Majadahonda, Madrid, 28222
Cristina Avendaño, MD, cavendano@salud.madrid.org
About this study
Official title: Early Treatment With Candesartan vs Placebo in Asymptomatic Genetic Carriers of Dilated Cardiomyopathy (EARLY-GENE Trial)"
Prospective, multicenter, randomized, placebo-controlled, double-blind clinical trial to evaluate safety and efficacy of candesartan in the prevention of the development of Dilated Cardiomyopathy (DCM) in genetic carriers of a DCM-causing variant without disease expression (asymptomatic)
Read the detailed description
Prospective, multicenter, randomized, placebo-controlled, double-blind clinical trial to evaluate safety and efficacy of early administration of candesartan in the prevention of the development of Dilated Cardiomyopathy (DCM) in genetic carriers of a DCM-causing variant without disease expression (asymptomatic).
Randomization will be 1:1 and patients are allocated to candesartan or matching placebo.
Patients will be followed for a 3 years period and efficacy will be demonstrated if candesartan (compared to placebo) prevents either a significant Left ventricular ejection fraction (LVEF) decline of ≥10%, or a ventricular dilatation (left ventricular end-diastolic volume, LVEDV) increase of ≥10% within a 3-years period of follow-up
- Conditions
- Cardiomyopathy, Dilated
- Being tested
- Candesartan
- Keywords
- Dilated Cardiomyopathy, Genetic Mutation Carrier, Randomized Clinical Trial, Genetic Dilated Cardiomyopathy
- Medical subject headings
- Cardiomyopathy, Dilated
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CandesartanDrug
3 years treatment with candesartan target dose: 32 mg or maximum tolerated dose after dose escalation from 16 mg
| Study group | Role | What they receive |
|---|---|---|
| Candesartan | Experimental | Candesartan, 16 mg oral tablets. Target dose 32 mg or maximum tolerated dose after dose escalation from 16 mg Receives: Drug: Candesartan |
| Placebo | Placebo comparator | Matching placebo. Target dose 2 tablets or maximum tolerated dose after dose escalation from 1 tablet Receives: Drug: Candesartan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of participants that progress to either a LVEF or LVEDV deterioration of ≥10% with respect to the baseline value at the end of follow-up as measured by MRI
Measured: 3 years
Secondary outcomes (7)
Proportion of participants that progress to a LVEF deterioration of ≥10% compared to baseline value at the end of follow-up as measured by MRI.
Measured: 3 years
Proportion of participants that progress to a LVEDV deterioration of ≥10% compared to baseline value at the end of follow-up as measured by MRI.
Measured: 3 years
Changes in LVEF measured by MRI (vs baseline)
Measured: 3 years
Changes in LVEDV measured by MRI (vs baseline)
Measured: 3 years
Proportion of individuals who develop DCM (LVEF<50%).
Measured: 3 years
Proportion of participants in each treatment group developing Serious Adverse Events (SAEs), Grade 3-4 adverse events (AEs), Adverse Reactions, or AEs of Special Interest (AESIs).
Measured: 3 years
Proportion of treatment discontinuations in the candesartan and placebo groups.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2022
- Study start2 Jun 2022
- Primary completion2 Jun 2026 (estimated)
- Study completion2 Jun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cardiomyopathy, DilatedStudies 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 3
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 320 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cristina Avendaño Solá
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Cristina Avendaño Solá, Puerta de Hierro University Hospital
- Investigators
- Pablo García-Pavía, MD, PhD, Study chair, Hospital Universitario Puerta de Hierro Majadahonda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05321875
- Sponsor's protocol ID
- EARLY-GENE
- Other IDs
- 2021-004577-30
- Acronym
- EARLY-GENE
- Status last verified
- Nov 2024
- First submitted
- 1 Apr 2022
- Last updated
- 7 Nov 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Under agreement, individual or aggregated patient data could be shared with other scientific groups for new scientific projects. Data can be shared only for scientific purposes and in full compliance with Personal Data Protection requirements in the EU
Study at a glance
From the registry record, last updated by the sponsor on 7 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Early Treatment With Candesartan vs Placebo in Asymptomatic Genetic Carriers of Dilated Cardiomyopathy (EARLY-GENE Trial)"
- ClinicalTrials.gov ID
- NCT05321875
- Acronym / sponsor ID
- EARLY-GENE / EARLY-GENE
- Conditions
- Cardiomyopathy, Dilated
- Keywords
- Dilated Cardiomyopathy, Genetic Mutation Carrier, Randomized Clinical Trial, Genetic Dilated Cardiomyopathy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 320 participants (estimated)
- Lead sponsor
- Cristina Avendaño SoláUniversity, hospital or other
- Study start
- 2 Jun 2022 (actual)
- Primary completion
- 2 Jun 2026 (estimated)
- Study completion
- 2 Jun 2026 (estimated)
- First posted
- 11 Apr 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.
Prospective, multicenter, randomized, placebo-controlled, double-blind clinical trial to evaluate safety and efficacy of candesartan in the prevention of the development of Dilated Cardiomyopathy (DCM) in genetic carriers of a DCM-causing variant without disease expression (asymptomatic)
Detailed description
Prospective, multicenter, randomized, placebo-controlled, double-blind clinical trial to evaluate safety and efficacy of early administration of candesartan in the prevention of the development of Dilated Cardiomyopathy (DCM) in genetic carriers of a DCM-causing variant without disease expression (asymptomatic).
Randomization will be 1:1 and patients are allocated to candesartan or matching placebo.
Patients will be followed for a 3 years period and efficacy will be demonstrated if candesartan (compared to placebo) prevents either a significant Left ventricular ejection fraction (LVEF) decline of ≥10%, or a ventricular dilatation (left ventricular end-diastolic volume, LVEDV) increase of ≥10% within a 3-years period of follow-up
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Candesartan | Experimental | Drug: CandesartanCandesartan, 16 mg oral tablets. Target dose 32 mg or maximum tolerated dose after dose escalation from 16 mg |
| Placebo | Placebo comparator | Drug: CandesartanMatching placebo. Target dose 2 tablets or maximum tolerated dose after dose escalation from 1 tablet |
Who may qualify
- Ages
- 18 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Age: 18-64 (both included), both sexes
- Carrier of a pathogenic or likely pathogenic DCM genetic variant1 according to modified American College of Medical Genetics (ACMG) criteria.
- Baseline LVEF ≥ 50% measured by MRI1 and evaluated by the eligibility study committee. Carriers with myocardial fibrosis, detected by late gadolinium enhancement in magnetic resonance imaging, are valid.
- Baseline creatinine ≤1.3 mg/dL, potassium ≤ 5.3 mEq/L and an estimated Glomerular Filtration Rate (eGFR)≥ 60 ml/min/1.73 m2 calculated by CKD-EPI formula.
- Able to understand and accept the study constraints and to provide informed consent.
Key exclusion criteria (18 listed)
- Hypotension (systolic arterial pressure <100 mmHg (measured following a standardized methodology).
- Prior ventricular dysfunction (LVEF ≤ 50% at any time prior to study inclusion)
- Candidates who are expected or highly likely to receive an implantable cardioverter defibrillator (ICD) in the following 12 months after inclusion in the trial
- Preexisting hypertension requiring pharmacological treatment.
- Uncontrolled arterial hypertension (i.e., repeatedly systolic arterial pressure > 140 mmHg).
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 |
|---|---|---|
| Candesartan3 years treatment with candesartan target dose: 32 mg or maximum tolerated dose after dose escalation from 16 mg | Drug | Candesartan, Placebo |
Primary outcome
Proportion of participants that progress to either a LVEF or LVEDV deterioration of ≥10% with respect to the baseline value at the end of follow-up as measured by MRI
Time frame: 3 years
7 secondary outcomes
Proportion of participants that progress to a LVEF deterioration of ≥10% compared to baseline value at the end of follow-up as measured by MRI.
Time frame: 3 years
Proportion of participants that progress to a LVEDV deterioration of ≥10% compared to baseline value at the end of follow-up as measured by MRI.
Time frame: 3 years
Changes in LVEF measured by MRI (vs baseline)
Time frame: 3 years
Changes in LVEDV measured by MRI (vs baseline)
Time frame: 3 years
Proportion of individuals who develop DCM (LVEF<50%).
Time frame: 3 years
Proportion of participants in each treatment group developing Serious Adverse Events (SAEs), Grade 3-4 adverse events (AEs), Adverse Reactions, or AEs of Special Interest (AESIs).
Time frame: 3 years
Proportion of treatment discontinuations in the candesartan and placebo groups.
Time frame: 3 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Nov 2024.
- Individual participant dataSharing planned: Yes. Under agreement, individual or aggregated patient data could be shared with other scientific groups for new scientific projects. Data can be shared only for scientific purposes and in full compliance with Personal Data Protection requirements in the EU
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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.
Pablo García-Pavía, MD, PhD study chair
Hospital Universitario Puerta de Hierro Majadahonda
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Candesartan in people with Cardiomyopathy, Dilated, run by Cristina Avendaño Solá at 1 site in Spain.
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 5 inclusion and 18 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: looking for participants now, as of the sponsor's last update on 7 Nov 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 Spain. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Cristina Avendaño-Solá, MD, PhD (+34 91 1916479, cavendano@salud.madrid.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of participants that progress to either a LVEF or LVEDV deterioration of ≥10% with respect to the baseline value at the end of follow-up as measured by MRI (time frame: 3 years). 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 7 Nov 2024.