Study on Mavacamten for Adults with Non-obstructive Hypertrophic Cardiomyopathy

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What is this study about?

This clinical trial is focused on studying a heart condition called Non-obstructive Hypertrophic Cardiomyopathy. This condition involves the thickening of the heart muscle, which can make it harder for the heart to pump blood. The study will evaluate a treatment called Mavacamten, which is taken as a capsule. Mavacamten is being tested to see if it can help improve symptoms and physical limitations in people with this heart condition.

The purpose of the study is to compare the effects of Mavacamten with a placebo over a period of 48 weeks. Participants will be randomly assigned to receive either Mavacamten or a placebo, and neither the participants nor the researchers will know who is receiving which treatment. The study will monitor changes in health status, including symptoms and exercise capacity, to determine the effectiveness of Mavacamten.

Throughout the study, participants will take the medication orally and will have regular check-ups to assess their condition. The study aims to provide valuable information on whether Mavacamten can be a beneficial treatment for individuals with Non-obstructive Hypertrophic Cardiomyopathy, potentially improving their quality of life by reducing symptoms and enhancing their ability to perform physical activities.

1 joining the study

Upon joining the study, the participant will be randomly assigned to receive either the active medication, mavacamten, or a placebo. This process is double-blind, meaning neither the participant nor the researchers know which treatment is being administered.

2 medication administration

The participant will take the assigned medication orally in the form of a capsule. The dosage and frequency will be determined by the study protocol, and the treatment will last for a duration of 48 weeks.

3 monitoring and assessments

Throughout the 48-week period, the participant’s health status, symptoms, and physical limitations will be regularly assessed. This includes monitoring exercise capacity and other health indicators.

4 primary outcome evaluation

The primary outcomes will be evaluated by measuring changes from the baseline in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ CSS) and peak oxygen consumption (pVO2) at week 48.

5 secondary outcome evaluation

Secondary outcomes will include changes in the VE/VCO2 slope, improvement in New York Heart Association (NYHA) class, and changes in biomarkers such as NT-pro BNP and cTn-T. Additionally, the time to first major adverse cardiovascular events (MACE-Plus) will be recorded.

6 completion of the study

At the end of the 48-week period, the participant will complete the study. Final assessments will be conducted to evaluate the overall impact of the treatment.

Who Can Join the Study?

Participants must be at least 18 years old or the local age of majority.
Female participants must use highly effective birth control methods or have proof that they cannot have children.
Male participants do not need to use additional birth control measures.
Participants must have a diagnosis of Hypertrophic Cardiomyopathy (HCM), which is a condition where the heart muscle becomes thickened, following specific medical guidelines.
The peak LVOT pressure gradient must be less than 30 mmHg at rest and less than 50 mmHg during certain tests. This measures the pressure difference in the heart.
Participants must have an oxygen level at rest greater than 90% during screening and be able to perform a special exercise test called cardiopulmonary stress test (CPET). This test checks how well the heart and lungs work during physical activity.
Participants must be in New York Heart Association (NYHA) Class II or III, which describes the severity of heart failure symptoms.
Participants must have a NT-proBNP level of 200 pg/mL or higher, or a BNP level of 70 pg/mL or higher. These are blood tests that help measure heart function.
The LVEF (Left Ventricular Ejection Fraction) must be 60% or higher on an echocardiogram, which is an ultrasound of the heart.
The KCCQ-23 CSS Score must be 85 or lower at screening. This score measures the impact of heart failure on a person’s life.

Who Cannot Join the Study?

Patients with other types of heart conditions that are not related to Non-obstructive Hypertrophic Cardiomyopathy cannot participate. This is a condition where the heart muscle becomes thickened without blocking blood flow.
Individuals who are not within the specified age range for the study are excluded. The study is designed for certain age groups only.
Participants who are unable to follow the study procedures or take the study medication as required will not be eligible.
People with certain medical conditions or who are taking medications that might interfere with the study treatment are not allowed to join.
Pregnant or breastfeeding women are not eligible to participate in the study.
Individuals who have participated in another clinical trial recently may be excluded to avoid interference with the study results.
Patients with a history of drug or alcohol abuse that could affect their ability to follow the study requirements are not eligible.
Anyone who has a known allergy or adverse reaction to the study medication or similar drugs cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Unidade Local De Saúde De Santa Maria, E.P.E.	Lisbon	Portugal
Medical University Of Vienna	Vienna	Austria
University Hospital Jena KöR	Jena	Germany
Center For Pediatric And Adolescent Medicine Of The Johannes Gutenberg University Mainz	Mainz	Germany
Hospital Universitario De Salamanca	Salamanca	Spain
Medical University Of Graz	Graz	Austria
Centre Hospitalier Regional Et Universitaire De Brest	Brest	France
Medizinische Universitaet Innsbruck	Innsbruck	Austria
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Oncopole Claudius Regaud	Toulouse	France
Katholieke Universiteit te Leuven	Leuven	Belgium
Oslo Universitetssykehus HF	Oslo	Norway
Medizinische Hochschule Hannover	Hanover	Germany
Bellvitge University Hospital	L'hospitalet De Llobregat	Spain
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
CHU Grenoble Alpes	La Tronche	France
Université Libre de Bruxelles – Hôpital Erasme	Brussels	Belgium
4 Wojskowy SzpitaKliniczny Z Polikliniką Samodzielny Publiczny ZakładOpieki ZdrowotneWe Wrocławiu	Wroclaw	Poland
Universitaetsmedizin Goettingen	Goettingen	Germany
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France
Uniwersytecki Szpital Kliniczny Im Jana Mikulicza Radeckiego We Wroclawiu	Wroclaw	Poland

Other Sites

Site Name	City	Country
Hospital Da Luz S.A.	Lisbon	Portugal
Semmelweis University	Budapest	Hungary
Centre Hospitalier Universitaire De Nantes	Nantes	France
Hopital Beaujon	Clichy	France
Hospital Son Llatzer	Palma	Spain
Instytut Centrum Zdrowia Matki Polki	Lodz	Poland
A.O. Krankenhaus St. Josef Braunau GmbH	Braunau Am Inn	Austria
Unidade Local De Saude Do Alto Ave E.P.E.	Guimaraes	Portugal
Kardio Brynow Sp. z o.o.	Katowice	Poland
Hdhfmfrq Vsuk doswqtwh	Barcelona	Spain
Hqjcgivp Uokdfwgclmbjf dy A Caopum	A Coruna Galicia	Spain
Uejsbgyism Hmiliivq Cvprfwf	Cologne	Germany
Alvoakgnc Uei	Amsterdam	The Netherlands
Umoqnqxlfpbj Mxjwzet Cjiudll Uthwjpg	Utrecht	The Netherlands
Aydsajvj Uzrrjlcsgl Hcspqtus	Lorenskog	Norway
Hyavalmf Cklswk Di Banylxvhr	Barcelona	Spain
Clulimn Uuueslhilmuhjblvcdtu Bxmppq Kmq	Berlin	Germany
Erjyknm Ugwbyjlenukx Mhcijtg Czquyvi Razllgxyo (uyhpdgh Mko	Rotterdam	The Netherlands
Csmmcj Hftunpwfino Rvngvoux Ucopyijrhsbak Dw Tgwmd	Tours	France
Umgvowjmmx Cwajywpn Htfegodu Viodyb Dz Lk Aenbhryr	Murcia	Spain
Uvskafggkiffnbdnxymmv Mdokekph Alm	Munster	Germany
Uhdkmjkdncluyoisaubbb Hpppwyatuy Atj	Heidelberg	Germany
Ksygnoay dlu Upuzbsknepqx Myprhdtu Ayg	Munich	Germany
Uhaunksluo Om Svaldz	Szeged	Hungary
Voohfpkyn Fcnrhnid Namacgxna V Pwwlt	Prague	Czechia
Uvygletpfw Hffjeixn Mjgkbzsfdh	Maastricht	The Netherlands
Csdsqc Hwpxkgitgpq Uliewpsmlrtlx Dk Roupjm	Rennes	France
Ubypcaoykm Ob Ajlcqan	Edegem	Belgium
Ogewjummulmldg Lpqy Gtcm	Linz	Austria
Ieyvpwtji For Chxaybwd Alp Ecynjcmapcjg Mpzddcak	Prague	Czechia
Cquobs Hehzwzedotk Lmjj Sge	Pierre Benite	France
Hbcrltec Uqglxvjvbwosu Pomnch Dg Hqioeh Da Moijnmobnve	Majadahonda	Spain
Hxvkaaus Uuzlecsfvlnfu Vkdbhr Dy Luw Nkoedp	Granada	Spain
Ejobye sitjhu	Nachod	Czechia
Hcznuslv Upslyyiecifut Vvybfh Dn Lh Vfulctbu	Malaga	Spain
Uumxypovophzbssoxqxqa Rhedmsevgb Amk	Regensburg	Germany
Dcylmduqe Hsyyvsjtzks Mdtmiqyt Drv Ftvzujpuuby Bhoqwf Kbscir Ax Dwd Tdtovlcxhuy Ujnobqtqqquo Mexyeegm	Munich	Germany
Svlkiagyp Rhvgwoz Uynxcwonow Mivmntt Chlmjc	Nijmegen	The Netherlands
Akgjnn Upxmifabsf Hyjcwoxi	Aarhus	Denmark
Fplmmdbm Nveywzzzt U Sk Aqga V Bpvv	Brno-Stred	Czechia
Pabghvqqa Ibramnoi Miharehk Mcwgmnriwgbd Sixgx Wpanuqysvqld I Aeqxvopgkjvvm	Warsaw	Poland
Kjbmkpanrjlxhqei Gxcs	Bad Nauheim	Germany
Zjtgkhrbtq Ofbh Lmzyzut	Genk	Belgium
Swisluj Srefniimsicdsn Srieuhqfmnv	Aabenraa	Denmark
Bkarzcghkzhrop Autsph Sobsrvraey	Balatonfured	Hungary
Awwsgpr Oivpnjlvitatzrhwgzyktzldx Szjm Arasd	Rome	Italy
Fbmctxhgtu Tkvpcfu Geaohznv Makziddhkp	Pisa	Italy
Hesl Uei Dzvlcfnvpaikwux Noo Bhj Osgyjsseke Ulsigpaxasjhrkghcbd Dhy Rofianoiqvyowbssi Btzrfk	Bad Oeynhausen	Germany
Utndbxn Lkggl Dk Sjzli Dg Gliadwpakhdp Ebszpx	Vila Nova De Gaia	Portugal
Skm Offhb Hzowpzxw Hq	Trondheim	Norway
Wiiocyilbf Sszumfyqqxnidgf Skghakw Im Dk Wx Brcwwqrpbcki	Lodz	Poland
Utfpyrcihdclbe Carpiyj Krxfdcewx Wgcndszpfmwke Utenrjcpfngx Mxogbwndhf	Warsaw	Poland
Kqayvfsv Blpypbyru gzoyo	Bielefeld	Germany

Trial status

Country	Status	Recruitment Start
Austria	Not recruiting	12.04.2023
Belgium	Not recruiting	12.04.2023
Czechia	Not recruiting	12.04.2023
Denmark	Not recruiting	12.04.2023
France	Not recruiting	12.04.2023
Germany	Not recruiting	12.04.2023
Hungary	Not recruiting	12.04.2023
Italy	Not recruiting	12.04.2023
Norway	Not recruiting	12.04.2023
Poland	Not recruiting	12.04.2023
Portugal	Not recruiting	12.04.2023
Spain	Not recruiting	12.04.2023
The Netherlands	Not recruiting	12.04.2023

Trial locations

Investigated Drugs:

Mavacamten

Mavacamten is a medication being studied for its potential to help adults with symptomatic nonobstructive hypertrophic cardiomyopathy. This condition involves the thickening of the heart muscle, which can lead to symptoms like shortness of breath and reduced exercise capacity. Mavacamten works by targeting the heart muscle to improve its function, potentially reducing symptoms and improving the ability to exercise. The clinical trial aims to evaluate how effective a 48-week course of mavacamten is in improving patients’ health status and physical limitations compared to not receiving the medication.

Investigated Diseases:

Non-obstructive cardiomyopathy

Non-obstructive Hypertrophic Cardiomyopathy – This is a condition where the heart muscle becomes abnormally thick without blocking blood flow. The thickened heart muscle can make it harder for the heart to pump blood efficiently. Over time, this can lead to symptoms such as shortness of breath, chest pain, and palpitations. The disease may progress slowly, and symptoms can vary widely among individuals. Some people may experience significant physical limitations, while others may have mild or no symptoms. The condition is often inherited and can be detected through family history.

Trial ID:

2023-506352-24-00

Protocol code:

CV027-031

NCT ID:

NCT05582395

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Mavacamten for Adults with Non-obstructive Hypertrophic Cardiomyopathy

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