Hypertrophic CardiomyopathyOPTIM-HCMNCT03846297
Optimisation of Decision Making for Defibrillator Implantation in Hypertrophic Cardiomyopathy
An observational study of Hypertrophic Cardiomyopathy, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
The main objective of the study is to improve implantable cardioverter defibrillator (ICD) implantation decision-making processing relevance by developing a new prediction model of sudden cardiac death (SCD) in hypertrophic cardiomyopathy (HCM), including newly identified potential biomarkers by magnetic resonance… Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Hypertrophic Cardiomyopathy
- How many people
- 2,000 participants
- Where
- 1 siteFrance
- Study dates
- 23 Sep 2020to Mar 2027
- Phase
- Observational
- 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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: * Inclusion of adults with HCM, as well as teenagers 16 or older since SCD occurs frequently in this age group * Necessary not to include specific etiologies such as amyloidosis because of a known different prognosis and natural history * Necessary not to include patients with ICD as secondary prevention because of a different prognosis and a worldwide consensus on the need for ICD implantation in this case * Necessary to include all cases whatever their date of diagnosis as the predictive modelling (primary objective) targets the actual population being followed up in real life, and will ultimately be applied on this population
Who may be able to join
- Patient with a diagnosis of HCM based on conventional criteria (left ventricle wall thickness ≥ 15 mm in adult index or ≥ 13 mm in adult relatives) in the absence of abnormal loading conditions
- Aged ≥ 16 years
- Patient without or with a defibrillator (in this latter case it should have been implanted for primary prevention, not for secondary prevention)
- Affiliation to a social security insurance
Who cannot join
- Specific etiologies such as amyloidosis
- Patients with ICD as secondary prevention (after aborted SCD or sustained ventricular arrhythmia)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site, 1 recruiting
- Recruiting
Centre de référence pour les cardiomyopathies et les troubles du rythme héréditaires ou rares - UF de Génétique, Hôpital Ambroise Paré,
Boulogne-Billancourt, Hauts-de-Seine, 92100
Philippe Charron, MD, PhD, +33 1 49 09 55 43, philippe.charron@aphp.fr
About this study
The main objective of the study is to improve implantable cardioverter defibrillator (ICD) implantation decision-making processing relevance by developing a new prediction model of sudden cardiac death (SCD) in hypertrophic cardiomyopathy (HCM), including newly identified potential biomarkers by magnetic resonance imaging (MRI) and genetics, through a prospective nationwide study, multivariate analysis and modelling of an absolute risk.
The secondary objective is to perform a medico-economic analysis of ICD implantation in order to define an optimal rule for ICD implantation in patients with HCM, taking into account the benefits of ICD, adverse effects of ICD and associated costs (cost of quality adjusted life years saved).
Read the detailed description
HCM is a genetic disease, usually autosomal dominant, with a prevalence of about 1/500 in the general population. HCM represents a major cause of SCD in the young, and is the first cause of SCD in athletes less than 35 years of age. The only efficient prevention of SCD, apart from exercise restriction, is ICD.
Given this dreadful potential outcome of HCM, the identification of patients that should receive an ICD is of paramount importance. However, the indications for ICD are controversial and very different guidelines are currently available, coming from the American society of Cardiology, the French Haute Autorité de Santé and from the European society of Cardiology. In addition, these guidelines are all based on retrospective studies and have major limitations. The situation faces a critical need of improvement. Thus, this study has been designed with the objective to propose a revision of international recommendations for defibrillator implantation in HCM patients.
This prospective multicenter non-interventional single-arm study is planned to be conducted in 40 sites with a collection of clinical and biological data, carried out as required for the usual management and care of patients. No investigation will be performed specifically for this study except for an additional blood sample collection at baseline during routine outpatient care and annual interview during follow-up.
- Conditions
- Hypertrophic Cardiomyopathy
- Keywords
- hypertrophic cardiomyopathy, implantable cardioverter defibrillator
- Medical subject headings
- Cardiomyopathy, Hypertrophic
What is being measured
The primary outcome is the main question the study is designed to answer.
Improvement of the ability to predict major clinical events: C-index comparisons
Comparisons of the C-index obtained between with the new score and with the different existing scores or algorithms of actual processing used for ICD decision-making.
C-index is a discrimination index, which will be determined after multivariate analyses and score modeling, in the case of the new score.
This evaluate of the improvement of the ability to predict major clinical events during follow-up will toward to development of a new prediction model in patients with HCM in responding the main objective of the study.
Data will be collected are: (composite end point): SCD, aborted sudden death, appropriate therapy of ICD that is a physiological equivalent of SCD.
Measured: throughout of the study, an average of 1 year
Secondary outcomes (4)
Cost-effectiveness study
To determine incremental cost-effectiveness ratio (ICER) of ICD decision, in order to assess the economic impact of defibrillator implantation as a function of the chosen rule for device implantation.
Data will be collected are: direct costs (health insurance tariffs) of defibrillators, cost of their maintenance and for management of side effects, for the treatment of sudden death recovered, the medical transport, hospitalizations and for cardiology acts.
Measured: throughout of the study, an average of 1 year
Cost-utility analysis
To determine incremental cost-utility ratio of ICD decision, in order to assess the economic impact of defibrillator implantation as a function of the chosen rule for device implantation.
Data will be collected are: direct costs (health insurance tariffs) of defibrillators, cost of their maintenance and for management of side effects, for the treatment of sudden death recovered, the medical transport, hospitalizations and for cardiology acts.
Measured: throughout of the study, an average of 1 year
Quality Adjusted Life Years
QALYs calculated using EQ-5d Index:
"EQ5D-5L QoL questionnaire", a questionnaire established by EuroQol Group in 5 dimensions with 5 levers.
Measured: at baseline and at 6-year follow-up
Improvement of the ability to predict major clinical events: C-index comparisons
Comparison of the C-index obtained between the new score developed for the primary objective and the C-index obtained when the following additional covariates are added to the multivariate predictive model: gender, maximum left ventricle wall thickness obtained by MRI, left atrial volume on echocardiography, left ventricle strain on echocardiography.
Measured: at baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Feb 2019
- Study start23 Sep 2020
- Primary completionMar 2027 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hypertrophic CardiomyopathyStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 2,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Philippe CHARRON, MD, PhD, Principal investigator, Centre de référence maladies cardiaques héréditaires, Hôpital Ambroise Paré & Hôpital Pitié-Salpêtrière
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03846297
- Sponsor's protocol ID
- AOR17412
- Acronym
- OPTIM-HCM
- Status last verified
- Mar 2023
- First submitted
- 4 Feb 2019
- Last updated
- 16 Mar 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Optimisation of Decision Making for Defibrillator Implantation in Hypertrophic Cardiomyopathy
- ClinicalTrials.gov ID
- NCT03846297
- Acronym / sponsor ID
- OPTIM-HCM / AOR17412
- Conditions
- Hypertrophic Cardiomyopathy
- Keywords
- hypertrophic cardiomyopathy, implantable cardioverter defibrillator
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 2,000 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 23 Sep 2020 (actual)
- Primary completion
- Mar 2027 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 19 Feb 2019 (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 main objective of the study is to improve implantable cardioverter defibrillator (ICD) implantation decision-making processing relevance by developing a new prediction model of sudden cardiac death (SCD) in hypertrophic cardiomyopathy (HCM), including newly identified potential biomarkers by magnetic resonance imaging (MRI) and genetics, through a prospective nationwide study, multivariate analysis and modelling of an absolute risk.
The secondary objective is to perform a medico-economic analysis of ICD implantation in order to define an optimal rule for ICD implantation in patients with HCM, taking into account the benefits of ICD, adverse effects of ICD and associated costs (cost of quality adjusted life years saved).
Detailed description
HCM is a genetic disease, usually autosomal dominant, with a prevalence of about 1/500 in the general population. HCM represents a major cause of SCD in the young, and is the first cause of SCD in athletes less than 35 years of age. The only efficient prevention of SCD, apart from exercise restriction, is ICD.
Given this dreadful potential outcome of HCM, the identification of patients that should receive an ICD is of paramount importance. However, the indications for ICD are controversial and very different guidelines are currently available, coming from the American society of Cardiology, the French Haute Autorité de Santé and from the European society of Cardiology. In addition, these guidelines are all based on retrospective studies and have major limitations. The situation faces a critical need of improvement. Thus, this study has been designed with the objective to propose a revision of international recommendations for defibrillator implantation in HCM patients.
This prospective multicenter non-interventional single-arm study is planned to be conducted in 40 sites with a collection of clinical and biological data, carried out as required for the usual management and care of patients. No investigation will be performed specifically for this study except for an additional blood sample collection at baseline during routine outpatient care and annual interview during follow-up.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- * Inclusion of adults with HCM, as well as teenagers 16 or older since SCD occurs frequently in this age group * Necessary not to include specific etiologies such as amyloidosis because of a known different prognosis and natural history * Necessary not to include patients with ICD as secondary prevention because of a different prognosis and a worldwide consensus on the need for ICD implantation in this case * Necessary to include all cases whatever their date of diagnosis as the predictive modelling (primary objective) targets the actual population being followed up in real life, and will ultimately be applied on this population
Key inclusion criteria (4 listed)
- Patient with a diagnosis of HCM based on conventional criteria (left ventricle wall thickness ≥ 15 mm in adult index or ≥ 13 mm in adult relatives) in the absence of abnormal loading conditions
- Aged ≥ 16 years
- Patient without or with a defibrillator (in this latter case it should have been implanted for primary prevention, not for secondary prevention)
- Affiliation to a social security insurance
Key exclusion criteria (2 listed)
- Specific etiologies such as amyloidosis
- Patients with ICD as secondary prevention (after aborted SCD or sustained ventricular arrhythmia)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Improvement of the ability to predict major clinical events: C-index comparisons
Time frame: throughout of the study, an average of 1 year
Comparisons of the C-index obtained between with the new score and with the different existing scores or algorithms of actual processing used for ICD decision-making. C-index is a discrimination index, which will be determined after multivariate analyses and score modeling, in the case of the new score. This evaluate of the improvement of the ability to predict major clinical events during follow-up will toward to development of a new prediction model in patients with HCM in responding the main objective of the study. Data will be collected are: (composite end point): SCD, aborted sudden death, appropriate therapy of ICD that is a physiological equivalent of SCD.
4 secondary outcomes
Cost-effectiveness study
Time frame: throughout of the study, an average of 1 year
Cost-utility analysis
Time frame: throughout of the study, an average of 1 year
Quality Adjusted Life Years
Time frame: at baseline and at 6-year follow-up
Improvement of the ability to predict major clinical events: C-index comparisons
Time frame: at baseline
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 16 Mar 2023.
- 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.
Philippe CHARRON, MD, PhD principal investigator
Centre de référence maladies cardiaques héréditaires, Hôpital Ambroise Paré & Hôpital Pitié-Salpêtrière
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hypertrophic Cardiomyopathy, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 2 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 16 Mar 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 France. 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.
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 Philippe CHARRON, MD, PhD (+ 33 1 49 09 55 43, philippe.charron@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Improvement of the ability to predict major clinical events: C-index comparisons (time frame: throughout of the study, an average of 1 year). 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 16 Mar 2023.