A Prospective Registry Study to Assess Real-world Patient Characteristics, Treatment Patterns, and Longitudinal Outcomes in Patients Receiving Mavacamten and Other Treatments for Symptomatic Obstructive Hypertrophic Cardiomyopathy (Obstructive-HCM)

Официальное название

Deliver Insights in Hypertrophic Cardiomyopathy and Observational Outcomes in Real World: United States and European Prospective Registry Study

Обзор

This registry evaluates patient characteristics, real-world treatment patterns, and short- and long-term outcomes in a population of patients in the United States and Europe with symptomatic obstructive hypertrophic cardiomyopathy (HCM) who are receiving mavacamten, receiving other treatment for obstructive HCM, or not receiving treatment for obstructive HCM due to intolerance or failure of prior treatment. United States Sub-Study: The purpose of this study is to evaluate the safety of mavacamten in patients with symptomatic obstructive HCM in the real-world setting. Europe Sub-Study: The purpose of this study is to evaluate the effectiveness and safety of mavacamten in patients with symptomatic obstructive HCM in the real-world setting.

Вмешательства

• Препарат Mavacamten
  As per product label
• Препарат Non-mavacamten symptomatic oHCM therapy
  Including Beta-blocker (BB), non-dihydropyridine (non-DHP) calcium channel blocker (CCB) and/or disopyramide as per product label

Первичные конечные точки

• Incidence rate of new or worsening heart failure due to systolic dysfunction among participants with symptomatic obstructive HCM during periods of exposure to mavacamten and other oHCM treatment [Срок оценки: Up to 5 Years]
• Change in resting left ventricular outflow tract (LVOT) gradient from baseline [Срок оценки: Up to 18 months]

Вторичные конечные точки (12)

• Occurrence of arrhythmia [Срок оценки: Baseline, and up to 18 months (European participants), or 5 Years (United States participants)]
• New or worsening heart failure due to systolic dysfunction [Срок оценки: Baseline and up to 18 months]
• Proportion of participants with a resting or provoked peak left ventricular outflow tract (LVOT) gradient below 50 mmHG [Срок оценки: Baseline and up to 18 months]
• Proportion of participants with a resting or provoked peak left ventricular outflow tract (LVOT) gradient below 30 mmHG [Срок оценки: Baseline and up to 18 months]
• Occurrence of Major Adverse Cardiovascular Events (MACE) [Срок оценки: Up to 5 Years]
• Occurrence of non-fatal acute Myocardial Infarction (MI) [Срок оценки: Up to 5 Years]
• Occurrence of Stroke [Срок оценки: Up to 5 Years]
• Occurrence of hospitalization due to heart failure [Срок оценки: Baseline, and up to 18 months (European participants), or 5 Years (United States participants)]
• Occurrence of cardiovascular mortality [Срок оценки: Up to 5 Years]
• Occurrence of all-cause mortality [Срок оценки: Baseline, and up to 18 months (European participants), or 5 Years (United States participants)]
• Evaluation of functional responses: New York Heart Association (NYHA) function class [Срок оценки: Baseline, and up to 18 months (European participants), or 5 Years (United States participants)]
• Evaluation of functional responses: Left Ventricular Outflow Tract (LVOT) gradient [Срок оценки: Baseline, and up to 18 months (European participants), or 5 Years (United States participants)]

Критерии участия

Критерии включения

• ≥ 18 years of age at the time of informed consent.
• Willing and able to provide written informed consent form (ICF) and any required privacy authorization prior to the initiation of study procedures (or in those situations where consent cannot be given by participants, consent provided by their legally acceptable representatives)

United States Sub-Study

• Diagnosis of obstructive HCM consistent with 2020 American Heart Association/American College of Cardiology (AHA/ACC) guidelines.

• Obstructive HCM is defined clinically by the presence of increased LV wall thickness ≥ 15 mm (or ≥ 13 mm with positive family history of HCM) in a nondilated ventricular chamber that is not solely explained by abnormal loading conditions (eg, another cardiac or systemic disease) and peak LVOT gradient of ≥ 30 mmHg at rest or with provocation.
• Has documented LVEF of ≥ 55% recorded by echocardiography within the last 6 months.
• Symptoms consistent with NYHA functional class II-IV.
• Receiving beta blocker (BB)s, non-dihydropyridine calcium. channel blockers (nonDHP CCBs), disopyramide, and/or mavacamten (once available) as part of routine clinical care; or currently receiving no treatment due to intolerance or failure of prior treatment (eg, BBs, non-DHP CCBs, or disopyramide) for obstructive HCM.

European Sub-study

• Diagnosis of obstructive HCM consistent with the most recent European Society of Cardiology (ESC) and American Heart Association/American College of Cardiology (AHA/ACC) guidelines
• Documented LVEF of ≥55% recorded by TTE
• Documented symptoms consistent with NYHA functional class II-III at enrollment or within 6 months prior to enrollment (if not available at enrollment).
• As part of routine clinical care for obstructive HCM: receiving BBs, non-DHP CCBs, disopyramide; initiating mavacamten at enrollment; or currently receiving no treatment due to intolerance or failure of prior treatment (e.g., BBs, non-DHP CCBs, or disopyramide).

Критерии исключения

• Known phenocopy disease (e.g., Fabry disease, amyloidosis) or LV hypertrophy associated with hypertension.
• Documentation of any fixed obstruction of the outflow tract such as aortic valve stenosis or replacement.
• Prior treatment of obstructive HCM with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation \[ASA\]) within 6 months prior to enrollment; participants with an unsuccessful myectomy or percutaneous ASA performed > 6 months prior to enrollment may be enrolled.
• Naïve to treatment for obstructive HCM (ie, never treated with BBs, nonDHP CCBs, or disopyramide).

United States Sub-Study

• Receiving an investigational therapeutic agent for obstructive HCM (eg, myosin-inhibitors other than mavacamten) in an interventional clinical trial at participant enrollment.
• Previously or currently enrolled in a long-term safety extension study of mavacamten (eg, EXPLORER-HCM \[ClinicalTrials.gov, NCT03470545\], MAVA-LTE \[NCT03723655\], PIONEER-OLE \[NCT03496168\], VALORHCM \[NCT04349072\], or MAVERICK \[NCT03442764\])

European Sub-study

• Receiving an investigational therapeutic agent or any cardiac myosin inhibitor and/or modulators for obstructive HCM at patient enrolment
• Previously or currently enrolled in other HCM registry studies (e.g., TORCH, REMY, EU-PASS)
• Previously or currently enrolled in a study of mavacamten (e.g., EXPLORER-HCM \[ClinicalTrials.gov, NCT03470545\], MAVA-LTE \[NCT03723655\], PIONEER-OLE \[NCT03496168\], VALOR-HCM \[NCT04349072\], MAVERICK \[NCT03442764\], or MEMENTO \[NCT2264899\])
• Previously treated with mavacamten

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

США · 62 центра

• Alaska Heart Institute — Anchorage
• Pima Heart and Vascular — Tucson
• UAMS — Little Rock
• UC San Diego School of Medicine — La Jolla
• Keck School of Medicine of USC-Usc — Los Angeles
• Stanford Health Care Hospital & Clinics — Palo Alto
• University Of California San Francisco Medical Center — San Francisco
• UC Denver, AMC — Aurora
• … и ещё 54 центра

Испания · 8 центров

Список центров уточняется — проверьте первичный протокол.

Австрия · 7 центров

• KH St.Josef Braunau am Inn — Braunau am Inn
• LKH-Univ. Klinikum Graz — Graz
• Local Institution - 0088 — Innsbruck
• Local Institution - 0084 — Klagenfurt
• Kepler Universitätsklinikum Linz — Linz
• Local Institution - 0085 — Sankt Pölten
• Local Institution - 0083 — Vienna

Франция · 7 центров

• Centre Hospitalier Haguenau — Haguenau
• CHU Nantes Hopital Nord Laennec — Saint-Herblain
• Hôpital Saint Vincent-de-Paul — Lille
• CHU Amiens-Picardie — Amiens
• Hôpital Sainte Musse — Toulon
• CHU De Poitiers - Hopital De La Miletrie — Poitiers
• Local Institution - 0117 — Montpellier

Германия · 7 центров

• Ostalb-Klinikum Aalen — Aalen
• Local Institution - 0101 — Erlangen
• Universitatsklinikum Muenster — Münster
• Praxisklinik Herz Und Gefasse-Klinikum Weisser Hirsch — Dresden
• … и ещё 3 центра

Великобритания · 7 центров

Список центров уточняется — проверьте первичный протокол.

Пуэрто-Рико · 1 центр

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT05489705 · CV027-012

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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