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Dilated CardiomyopathyPhase 3REALM-DCMNCT03439514

A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation

A Phase 3 study testing ARRY-371797 (PF-07265803) in people with Dilated Cardiomyopathy and Lamin A/C Gene Mutation, run by Pfizer at 107 sites in 10 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This is a randomized, double-blind, placebo-controlled study in patients with dilated cardiomyopathy (DCM) due to a mutation of the gene encoding the lamin A/C protein (LMNA). The study will further evaluate a dose level of study drug (ARRY-371797) that has shown preliminary efficacy and safety in this patient… Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
ARRY-371797 (PF-07265803)
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
77 participants
Where
107 sitesin 10 countries
Study dates
17 Apr 2018to 13 Oct 2022
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Selected Key Inclusion Criteria:

  • Patients with symptomatic lamin A/C protein (LMNA)-related cardiomyopathy Class II/III/ or Class IV defined as:
  • Gene positive for a pathogenic, likely pathogenic, or VUS mutation in the LMNA gene as determined by an accredited clinical laboratory.
  • Evidence of cardiac impairment in LVEF <= 50%
  • Patient will have an implantable cardioverter defibrillator/cardiac resynchronization therapy defibrillator (ICD/CRT-D). ICD implanted at least 4 weeks prior to initiation of study treatment or CRT-D initiated at least 6 months prior to initiation of study treatment and defibrillation function activated at least 4 weeks prior to initiation of study treatment.
  • Class II/III patients must have objective functional impairment evidenced by a reduction in 6-minute walk test (6MWT); a. Screening: 6MWT distance >100 m but ≤450 m, AND b. Day -1 visit: 6MWT distance >100 m but ≤485 m, AND c. Baseline visit (Day 1): 6MWT distance >100 m but ≤485
  • Class II/III patients must be stable for at least 3 months
  • Stable medical and/or device therapy consistent with regional American Heart Association (AHA) / American College of Cardiology (ACC) or European Society of Cardiology (ESC) guidelines at the investigator discretion, without change in heart failure drug(s) dose in the past 1 month.
  • Patients must meet acceptable hematology, hepatic and renal laboratory values within 35 days prior to Day 1 as specified in the protocol.

Selected Key Exclusion Criteria:

  • Presence of other form(s) of cardiomyopathy contributing to HF (eg, inflammatory or infiltrative cardiomyopathy), clinically significant cardiac anatomic abnormality (eg,LV aneurysm), clinically significant coronary artery disease (eg, coronary revascularization, exercise induced angina) or uncorrected, hemodynamically significant (ie, moderate-severe) primary structural valvular disease not due to HF, per investigator judgment.
  • Currently receiving intermittent or continuous IV inotrope infusion, or presence of a ventricular assist device, or history of prior heart transplantation. Participants listed for cardiac transplantation may be enrolled provided transplantation is not likely to occur in the next 6 months.
  • Myocardial infarction, cardiac surgical procedures (other than for pacemaker/ICD/CRT-D implantation or replacement), acute coronary syndrome, serious systemic infection with evidence of septicemia, or any major surgical procedure requiring general anesthesia within 3 months prior to screening.
  • Currently receiving or deemed at high risk of requiring chronic renal replacement therapy (eg, hemodialysis or peritoneal dialysis) within 6 months.
  • Initiation of CRT within 6 months prior to screening.
  • Treatment with any investigational agent(s) for HF within 35 days prior to Day 1.
  • Malignancy that is active or has been diagnosed within 3 years prior to screening, except surgically curatively resected in situ malignancies or surgically cured early breast cancer, prostate cancer, skin cancer (basal cell carcinoma, squamous cell carcinoma), thyroid cancer, or cervical cancer, or, with prior review by the medical monitor, other early stage surgically curatively resected malignancies with less than a 20% expected 2 year recurrence rate.
  • Non-cardiac condition that limits lifespan to < 1 year.
  • Serum positive for hepatitis B surface antigen, viremic hepatitis C, or human immunodeficiency virus (HIV) at screening.

Trial locations and contacts

107 sites in 10 countries.

107 sites in 10 countries

United States56 sites

  • Centers for Heart Failure Therapy

    Atlanta, Georgia, 30322

  • Emory University Hospital

    Atlanta, Georgia, 30322

  • University of Colorado Academic Offices Building

    Aurora, Colorado, 80045

  • University of Colorado Clinical and Translational Research Center

    Aurora, Colorado, 80045

  • University of Colorado Hospital

    Aurora, Colorado, 80045

  • University of Alabama at Birmingham Hospital

    Birmingham, Alabama, 35233

  • University of Alabama at Birmingham the Kirklin Clinic

    Birmingham, Alabama, 35233

  • IDS Pharmacy at UAB Hospital

    Birmingham, Alabama, 35249

  • Cardiovascular Clinical Trials Unit

    Birmingham, Alabama, 35294

  • Saint Luke's Idaho Cardiology Associates

    Boise, Idaho, 83712-6246

  • Brigham and Women's Hospital

    Boston, Massachusetts, 02115

  • Chandler Regional Medical Center

    Chandler, Arizona, 85224

  • Valley Heart Rhythm Specialists, PLLC

    Chandler, Arizona, 85224

  • Medical University of South Carolina - PPDS

    Charleston, South Carolina, 29425-8911

  • Medical University of South Carolina - PPDS

    Charleston, South Carolina, 29425

  • Innovative Research of West Florida

    Clearwater, Florida, 33756

  • The Cleveland Clinic Foundation

    Cleveland, Ohio, 44195

  • Columbus Cardiology Associates Research - Centricity Research - HyperCore - PPDS

    Columbus, Georgia, 31904-6877

  • Columbus Cardiology Associates Research - IACT - HyperCore - PPDS

    Columbus, Georgia, 31904-6877

  • Columbus Regional Research Institute at Talbotton - Centricity Research - HyperCore - PPDS

    Columbus, Georgia, 31904-8946

  • Columbus Regional Research Institute

    Columbus, Georgia, 31904

  • Ohio State University Wexner Medical Center

    Columbus, Ohio, 43210

  • Baylor Scott and White Heart and Vascular Hospital

    Dallas, Texas, 75226

  • Baylor Annette C and Harold C Simmons Transplant Institute

    Dallas, Texas, 75246

  • Stern Cardiovascular Foundation Inc

    Germantown, Tennessee, 38138

  • Cardiovascular and Stem Cell Consultants

    Gilbert, Arizona, 85297-0425

  • Cardiovascular Research Clinic

    Gilbert, Arizona, 85297-0425

  • Dignity Health, Mercy Gilbert Medical Center

    Gilbert, Arizona, 85297-0425

  • Mercy Gilbert Medical Center

    Gilbert, Arizona, 85297

  • Ahmanson Cardiomyopathy Center Cardiovascular Genetics

    Los Angeles, California, 90095

  • Meriter Hospital

    Madison, Wisconsin, 53713

  • MyMichigan Medical Center Midland

    Midland, Michigan, 48670

  • CB Flock Research Corporation

    Mobile, Alabama, 36608

  • Intermountain Medical Center

    Murray, Utah, 84107

  • NYU School of Medicine / NYU Langone Health

    New York, New York, 10016

  • Columbia University Irving Medical Center

    New York, New York, 10032

  • Columbia University/Presbyterian Hospital - Vivian & Seymour Milstein Family Heart Center

    New York, New York, 10032

  • Rutgers New Jersey Medical School - Doctors Office Center

    Newark, New Jersey, 07103

  • Newark Beth Israel Medical Center

    Newark, New Jersey, 07112

  • University of Pennsylvania Hospital

    Philadelphia, Pennsylvania, 19104

  • University of Washington Medical Center

    Seattle, Washington, 98195

  • Washington University Center For Advanced Medicine

    St Louis, Missouri, 63110

  • Washington University Center for Outpatient Health

    St Louis, Missouri, 63110

  • Washington University School of Medicine

    St Louis, Missouri, 63110

  • Stanford Hospital and Clinics

    Stanford, California, 94305

  • University of South Florida

    Tampa, Florida, 33606-3601

  • USF Health

    Tampa, Florida, 33606

  • Florida Cardiovascular Institute PA

    Tampa, Florida, 33609

  • Children's Clinic

    Tucson, Arizona, 85712

  • Banner - Banner University Medical Center South

    Tucson, Arizona, 85713

  • Banner - University Medical Center Tucson

    Tucson, Arizona, 85719

  • Banner University Medicine North

    Tucson, Arizona, 85719

  • Banner University Medical Center Tuscon

    Tucson, Arizona, 85724

  • University of Arizona Sarver Heart Center

    Tucson, Arizona, 85724

  • Tennessee Center for Clinical Trials

    Tullahoma, Tennessee, 37388

  • Medstar Washington Hospital Center

    Washington D.C., District of Columbia, 20010

Other trials in United States

Italy16 sites

  • AO Ospedale Policlinico Consorziale di Bari

    Bari, 70124

  • Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari

    Bari, 70124

  • Azienda Ospedaliera Spedali Civili di Brescia

    Brescia, 25123

  • Azienda Ospedaliera Universitaria Careggi

    Florence, Tuscany, 50134

  • Lacopo Olivotto, Azienda Ospedaliera Universitaria Careggi

    Florence, Tuscany, 50134

  • Azienda Ospedaliera Universitaria Careggi

    Florence, 50134

  • Fondazione IRCCS Policlinico San Matteo di Pavia

    Pavia, PV, 27100

  • IRCCS Pavia - Istituti Clinici Scientifici Maugeri Spa ? Società Benefit

    Pavia, 27100

  • A.O.U. di Perugia Ospedale Santa Maria della Misericordia

    Perugia, Umbria, 06156

  • A.O.U. di Perugia Ospedale Santa Maria Della Misericordia

    Perugia, 06132

  • A.O.U. di Perugia Ospedale Santa Maria della Misericordia

    Perugia, 06156

  • Ospedale Santa Maria Della Misericordia Perugia

    Perugia - Località S. Andrea Delle Fratte, Perugia, 6132

  • Azienda Ospedaliera Sant'Andrea

    Rome, Lazio, 00189

  • Presidio Ospedaliero Madonna del Soccorso

    San Benedetto del Tronto, 63074

  • Ospedale Di Cattinara

    Trieste, Friuli Venezia Giulia, 34149

  • Ospedale Di Cattinara

    Trieste, 34149

Other trials in Italy

Spain11 sites

  • Complexo Hospitalario Universitario A Coruña

    A Coruña, 15006

  • Hospital Universitario Vall d'Hebrón - PPDS

    Barcelona, 08035

  • Hospital Clinic de Barcelona

    Barcelona, 08036

  • C.H. Regional Reina Sofia - PPDS

    Córdoba, 14004

  • Hospital General Universitario Reina Sofia

    Córdoba, 14004

  • Hospital Universitario Virgen de la Arrixaca

    El Palmar, Murcia, 30120

  • Hospital Universitario Puerta de Hierro de Majadahonda

    Majadahonda, Madrid, 28222

  • Hospital Universitario Puerta de Hierro - Majadahonda

    Majadahonda, 28222

  • Hospital Universitario Son Llatzer

    Palma de Mallorca, 07198

  • Centro de Farmacovigilancia de Galicia

    Santiago de Compostela, A Coruña, 15781

  • Complejo Hospitalario Universitario de Vigo - H. Alvaro Cunqueiro

    Vigo, Pontevedra, 36312

Other trials in Spain

Canada7 sites

  • Nova Scotia Health Authority (Capital District Health Authority)

    Halifax, Nova Scotia, B3H 1V7

  • NSHA QEII Health Sciences Halifax Infirmary

    Halifax, Nova Scotia, B3H 3A7

  • Clinical Laboratories of Montreal Heart Institute

    Montreal, Quebec, H1T 1C8

  • University of Ottawa Heart Institute

    Ottawa, Ontario, K1Y 4W7

  • Kawartha Cardiology Clinical Trials

    Peterborough, Ontario, K9J 0B2

  • Centre Intégré Universitaire de santé et de services sociaux de l'Estrie Centre hospitalier

    Sherbrooke, Quebec, J1G 2E8

  • St. Paul's Hospital

    Vancouver, British Columbia, V6Z 1Y6

Other trials in Canada

Argentina5 sites

  • Fundacion Favaloro para la Docencia e Investigacion Medica

    Buenos Aires, Buenos Aires F.D., C1093AAS

  • Hospital Británico de Buenos Aires

    Buenos Aires, C1280AEB

  • Instituto CAICI

    Rosario, Santa Fe Province, S2000 PBJ

  • Hospital Provincial Del Centenario

    Rosario, Santa Fe Province, S2000KDS

  • Hospital Provincial Dr Jose Maria Cullen

    Santa Fe, S3000EOZ

Other trials in Argentina

Mexico4 sites

  • Hospital Boutique Riobamba

    Mexico City, Mexico City, 07300

  • Fundación de Atención e Investigación Médica Lindavista S.C.

    Mexico City, 07300

  • Cardiolink Clin Trials S.C.

    Monterrey, Nuevo León, 64060

  • Christus Muguerza Hospital Alta Especialidad

    Monterrey, Nuevo León, 64060

Other trials in Mexico

United Kingdom3 sites

  • Queen Elizabeth University Hospital - PPDS

    Glasgow, Glasgow CITY, G51 4TF

  • St Bartholomew's Hospital

    London, EC1A 7BE

  • Royal Brompton Hospital

    London, SW3 6NP

Other trials in United Kingdom

Belgium2 sites

  • Onze-Lieve-Vrouwziekenhuis

    Aalst, Oost-vlaanderen, 9300

  • UZ Leuven

    Leuven, 3000

Other trials in Belgium

Norway2 sites

  • Sykehuset Innlandet HF Hamar

    Hamar, 2318

  • Oslo University Hospital, Rikshospitalet

    Oslo, 0424

Other trials in Norway

Netherlands1 site

  • Amsterdam UMC, Location Academic Medical Center

    Amsterdam, North Holland, 1105 AZ

Other trials in Netherlands

About this study

Official title: A Phase 3, Multinational, Randomized, Placebo-controlled Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation (REALM-DCM)

This is a randomized, double-blind, placebo-controlled study in patients with dilated cardiomyopathy (DCM) due to a mutation of the gene encoding the lamin A/C protein (LMNA). The study will further evaluate a dose level of study drug (ARRY-371797) that has shown preliminary efficacy and safety in this patient population. After the primary analysis has been performed, eligible patients may receive open-label treatment with ARRY-371797.

Being tested
ARRY-371797 (PF-07265803), Placebo
Keywords
cardiomyopathy, Lamin Type A, heart failure, ARRY-797, C4411002
Medical subject headings
Cardiomyopathy, Dilated, Cardiomyopathies, Limb-girdle muscular dystrophy, type 1B, Heart Failure

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ARRY-371797 (PF-07265803)Drug

    400 mg twice daily (BID)

  • PlaceboOther

    BID

Study groups in NCT03439514
Study groupRoleWhat they receive
Part 1 Double-blind TreatmentExperimentalARRY-371797 (PF-07265803) tablet orally OR matching placebo tablet orally
Receives: Drug: ARRY-371797 (PF-07265803); Other: Placebo
Part 2 Open-label TreatmentExperimentalARRY-371797 (PF-07265803) tablet orally
Receives: Drug: ARRY-371797 (PF-07265803)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change From Baseline in Six-Minute Walk Test (6 MWT) at Week 24

    The 6 MWT was an assessment where the distance that a participant could walk on a flat and hard surface in 6 minutes was measured. Participants were asked to perform the test at a pace that was comfortable to them, with as many breaks as they needed, and under supervision of a qualified professional. Study discontinuation \& death were incorporated into endpoint definition through ranking in hypothesis testing of treatment difference. Missing data resulting from study discontinuation were imputed using control-based multiple imputation method to estimate treatment effect.

    Measured: Baseline, Week 24

Secondary outcomes (12)

  1. Change From Baseline in 6 MWT at Weeks 4 and 12

    The 6 MWT was an assessment where the distance that a participant could walk on a flat and hard surface in 6 minutes was measured. Participants were asked to perform the test at a pace that was comfortable to them, with as many breaks as they needed, and under supervision of a qualified professional.

    Measured: Baseline, Week 4, Week 12

  2. Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Physical Limitation (PL) and Total Symptom Score (TSS) Domain Scores at Weeks 12 and 24

    The KCCQ measured the effects of symptoms, functional (physical) limitations, and psychological distress on an individual's health-related quality of life. It contains 23 items, which assessed the ability to perform activities of daily living, frequency and severity of symptoms, the impact of these symptoms, and health-related quality of life. PL was a single questionnaire with score range of 0 to 100, where higher scores reflected better physical functioning status. TSS included frequency and severity of symptoms, and the impact of these symptoms. TSS scores were transformed to a range of 0 to 100, where higher scores reflected better health status.

    Measured: Baseline, Week 12, Week 24

  3. Number of Participants With Improvement From Baseline in Patient Global Impression of Severity (PGI-S) Score at Weeks 12 and 24

    PGI-S is a global index that rate the severity of the disease using a 5-point scale. In this outcome the number of participants with improvements in PGI-S the severity of their heart failure symptoms and in the severity of their PL were reported. Measured by the scale of: none, mild, moderate, severe or very severe (listed from better to worse).

    Measured: Week 12, Week 24

  4. Number of Participants With Improvement From Baseline in Patient Global Impression of Change (PGI-C) Score at Weeks 12 and 24

    PGI-C is a global index that rate the severity of the disease using a 7-point scale. In this outcome the number participants with improvements in their heart failure symptoms and "in their physical activity limitations?", were reported. Measured by the scale of: very much better, moderately better, a little better, no change, a little worse, moderately worse, very much worse (listed from better to worse).

    Measured: Week 12, Week 24

  5. Change From Baseline in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP) at Weeks 4, 12, and 24

    NT pro-BNP is a cardiac biomarker that is released in the blood in response to changes in the pressure inside of the heart. Levels go up when heart failure develops or gets worse, and levels go down when heart failure is stable or improves. This biomarker helps to measure the changes in the severity of heart failure over time in response to therapy.

    Measured: Baseline, Week 4, Week 12, Week 24

  6. Composite Time to First Occurrence of All-Cause Mortality or Worsening Heart Failure (WHF)

    Defined as the time from randomization to the first occurrence of any event of death due to any cause, or worsening heart failure (HF-related hospitalization or HF-related urgent care visit). Kaplan-Meier method and cox regression model were used for analysis.

    Measured: Maximum up to 212.28 weeks (maximum exposure was 208 weeks)

  7. Overall Survival (OS)

    OS was defined as time from randomization to death due to any cause. Participants who did not have a death date were censored for OS at their last contact date. Kaplan-Meier method and cox regression model were used for analysis.

    Measured: From randomization up to death due to any cause or censored date, maximum up to 212.28 weeks (maximum exposure was of 208 weeks)

  8. Number of Participants With Treatment Emergent Adverse Events (AEs) and by Severity

    An AE was any untoward medical occurrence in a participant who received investigational product without regard to possibility of causal relationship. Treatment-emergent AEs were events that occurred between first dose of study drug and up to 30 days after last dose. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and all Non-SAEs. Grade >=3 AEs meant severe AEs.

    Measured: Maximum up to 212.28 weeks (maximum exposure was of 208 weeks)

  9. Number of Participants With Laboratory Test Abnormalities

    Following parameters were analyzed for laboratory examination: hematology (eosinophils, erythrocytes, hemoglobin, hematocrit, granulocytes, leukocytes, lymphocytes, monocytes, platelets, neutrophils, nucleated erythrocytes); blood chemistry (alanine aminotransferase, albumin, alkaline phosphatase, aspartate aminotransferase, bicarbonate, bilirubin, blood urea nitrogen, C-reactive protein, calcium, chloride, creatinine, creatine kinase, epidermal growth factor receptor, follicle stimulating hormone, gamma glutamyl transferase, glucose, magnesium, N-Terminal ProB-type natriuretic peptide, phosphate, potassium, protein, sodium, potassium, thyrotropin, troponin I, troponin T, urate).

    Measured: Maximum up to 212.28 weeks (maximum exposure was of 208 weeks)

  10. Number of Participants According to Categorization of Abnormal Vital Signs

    Following vital sign parameters were assessed: diastolic blood pressure, systolic blood pressure, heart rate, and body weight. Vital sign abnormalities criteria included: a) systolic blood pressure (mmHg): decrease (change <= -20, or value <90) and increase (change >=20, or value >140); b) diastolic blood pressure (mmHg): decrease (change <= -15, or value <60) and increase (change >=15, or value >90); c) heart Rate (bpm) decrease: (change <= -15, or value <50) and increase (change >=15, or value >100); d) weight: (kg) decrease (Change <= -7%) and increase (Change > =7%).

    Measured: Maximum up to 212.28 weeks (maximum exposure was of 208 weeks)

  11. Number of Participants According to Categorization of Electrocardiogram (ECG) Data

    Following parameters were analyzed: heart rate, QT interval, corrected QT (QTc) interval, Bazett's correction QT (QTcB) interval, and Fridericia's correction (QTcF) interval. Criteria for notable ECG values were as follows: QT interval (in millisecond [msec]) new (newly occurring post-baseline value) greater than (>) 450, 480, 500, increase from baseline >30, increase from baseline >60; corrected QT interval by Fredericia formula (QTcF) in msec new (newly occurring post-baseline value) > 450, 480, 500, increase from baseline >30, increase from baseline >60; corrected QT interval by Bazett's formula (QTcB) in msec new (newly occurring post-baseline value) > 450, 480, 500, increase from baseline >30, increase from baseline >60; heart rate in bpm new (newly occurring post-baseline value) <60 and >100.

    Measured: Maximum up to 212.28 weeks (maximum exposure was of 208 weeks)

  12. Number of Participants With a New Clinically Significant Ventricular or Atrial Arrhythmias

    Arrhythmia assessment: incidence of new and clinically significant ventricular or atrial arrhythmias was assessed by an implantable cardioverter defibrillator (ICD) or CRT defibrillator (CRT-D) applicable device interrogations.

    Measured: Baseline, Week 12, Week 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Feb 2018
  2. Study start17 Apr 2018
  3. Primary completion13 Oct 2022
  4. Study completion13 Oct 2022

Why it stopped: The Sponsor terminated the study due to futility. The decision to stop the study was not based on safety concerns.

Resources

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
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
The study will be conducted in 2 parts: a randomized, double-blind treatment period for at least 24 weeks, followed by an ARRY-371797 (PF-07265803) open-label treatment period.
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
77 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03439514
Sponsor's protocol ID
ARRAY-797-301
Other IDs
C4411002, 2017-004310-25, 2017
Acronym
REALM-DCM
Status last verified
Dec 2023
First submitted
1 Feb 2018
Last updated
9 Jan 2024
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.

Publications

  • Garcia-Pavia P, Cai X, Di J, Li H, Demanuele C, Bruno J, Hinnershitz TM, Angeli FS, MacRae CA. Actigraphy-Quantified Physical Activity Measurements in Patients with Symptomatic LMNA-Related Dilated Cardiomyopathy in REALM-Dilated Cardiomyopathy. Digit Biomark. 2026 Jan 6;10(1):32-40. doi: 10.1159/000550259. eCollection 2026 Jan-Dec.

    PubMed 41693767

  • Garcia-Pavia P, Lakdawala NK, Sinagra G, Ripoll-Vera T, Afshar K, Priori SG, Ware JS, Owens A, Li H, Angeli FS, Elliott P, MacRae CA, Judge DP. Characterization and natural history of patients with LMNA-related dilated cardiomyopathy in the phase 3 REALM-DCM trial. ESC Heart Fail. 2024 Dec;11(6):4201-4208. doi: 10.1002/ehf2.14955. Epub 2024 Aug 15.

    PubMed 39145700

  • Garcia-Pavia P, Palomares JFR, Sinagra G, Barriales-Villa R, Lakdawala NK, Gottlieb RL, Goldberg RI, Elliott P, Lee P, Li H, Angeli FS, Judge DP, MacRae CA; REALM-DCM Investigators. REALM-DCM: A Phase 3, Multinational, Randomized, Placebo-Controlled Trial of ARRY-371797 in Patients With Symptomatic LMNA-Related Dilated Cardiomyopathy. Circ Heart Fail. 2024 Jul;17(7):e011548. doi: 10.1161/CIRCHEARTFAILURE.123.011548. Epub 2024 Jul 9.

    PubMed 38979608

  • Judge DP, Taylor MR, Li H, Oliver C, Angeli FS, Lee PA, MacRae CA. Long-term effectiveness of ARRY-371797 in people with dilated cardiomyopathy and a faulty LMNA gene: a plain language summary. Future Cardiol. 2023 Mar;19(3):117-126. doi: 10.2217/fca-2022-0125. Epub 2023 Apr 3.

    PubMed 37010012

  • MacRae CA, Taylor MR, Mestroni L, Moses J, Ashley EA, Wheeler MT, Lakdawala NK, Hershberger RE, Sandor V, Saunders ME, Oliver C, Lee PA, Judge DP. Plain Language Summary of Publication of the safety and efficacy of ARRY-371797 in people with dilated cardiomyopathy and a faulty LMNA gene. Future Cardiol. 2023 Feb;19(2):55-63. doi: 10.2217/fca-2022-0099. Epub 2023 Jan 31.

    PubMed 36718638

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing ARRY-371797 (PF-07265803) in people with Dilated Cardiomyopathy and Lamin A/C Gene Mutation, run by Pfizer at 107 sites in 10 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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: stopped early, as of the sponsor's last update on 9 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 107 sites in United States, Italy, Spain, Canada, Argentina and 5 more countries. 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.

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?

Yes. Results are posted in the registry (first posted 9 Jan 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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 From Baseline in Six-Minute Walk Test (6 MWT) at Week 24 (time frame: Baseline, Week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03439514, which also lists 5 linked publications.

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 9 Jan 2024.

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