Trial · NCT03439514
A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation
NCT03439514, Phase 3, Terminated, Pfizer; results first posted 2024-01-09
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation. Trials, record NCT03439514. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03439514
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation
- NCT number
- NCT03439514
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 77 participants, actual
- Lead sponsor
- Pfizer
- Conditions
- Dilated Cardiomyopathy, Lamin A/C Gene Mutation
- Interventions
- ARRY-371797 (PF-07265803), Placebo
- First posted
- 2018-02-20
- Last updated
- 2024-01-09
- Other registries' IDs
- eudract:2017-004310-25
- Results first posted
- 2024-01-09
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Six-Minute Walk Test (6 MWT) at Week 24 | Baseline, 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. |
| Secondary | Change From Baseline in 6 MWT at Weeks 4 and 12 | Baseline, Week 4, Week 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. |
| Secondary | Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Physical Limitation (PL) and Total Symptom Score (TSS) Domain Scores at Weeks 12 and 24 | Baseline, Week 12, Week 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. |
| Secondary | Number of Participants With Improvement From Baseline in Patient Global Impression of Severity (PGI-S) Score at Weeks 12 and 24 | Week 12, Week 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). |
| Secondary | Number of Participants With Improvement From Baseline in Patient Global Impression of Change (PGI-C) Score at Weeks 12 and 24 | Week 12, Week 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). |
| Secondary | Change From Baseline in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP) at Weeks 4, 12, and 24 | Baseline, Week 4, Week 12, Week 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. |
| Secondary | Composite Time to First Occurrence of All-Cause Mortality or Worsening Heart Failure (WHF) | Maximum up to 212.28 weeks (maximum exposure was 208 weeks) | 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. |
| Secondary | Overall Survival (OS) | From randomization up to death due to any cause or censored date, maximum up to 212.28 weeks (maximum exposure was of 208 weeks) | 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. |
| Secondary | Number of Participants With Treatment Emergent Adverse Events (AEs) and by Severity | Maximum up to 212.28 weeks (maximum exposure was of 208 weeks) | 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. |
| Secondary | Number of Participants With Laboratory Test Abnormalities | Maximum up to 212.28 weeks (maximum exposure was of 208 weeks) | 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). |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Six-Minute Walk Test (6 MWT) at Week 24 | Baseline, Week 24 | Efficacy analysis set (EAS): NYHA functional Class II / III randomized participants. 'Number of Participants Analyzed' = participants evaluable for the outcome measure. Five participants (ARRY-371797 \[n=3\], placebo \[n=2\]) discontinued the study before week 24 due to the sponsor's decision to terminate and were excluded from the primary analysis. All participants reported under 'Number of Participants Analyzed' contributed data to table but may not have evaluable data for every row. | Meter | MEDIAN |
| SECONDARY | Change From Baseline in 6 MWT at Weeks 4 and 12 | Baseline, Week 4, Week 12 | EAS included all NYHA functional Class II or III randomized participants. All participants reported under 'Overall Number of Participants Analyzed' contributed data to the table but may not have evaluable data for every row. Number Analyzed signifies number of participants evaluable for specified rows of respective arms. | Meter | MEDIAN |
| SECONDARY | Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Physical Limitation (PL) and Total Symptom Score (TSS) Domain Scores at Weeks 12 and 24 | Baseline, Week 12, Week 24 | EAS included all NYHA functional Class II or III randomized participants. All participants reported under 'Overall Number of Participants Analyzed' contributed data to the table but may not have evaluable data for every row. Here, Number Analyzed signifies participants evaluable for specified rows. | Units on a scale | MEAN |
| SECONDARY | Number of Participants With Improvement From Baseline in Patient Global Impression of Severity (PGI-S) Score at Weeks 12 and 24 | Week 12, Week 24 | EAS included all NYHA functional Class II or III randomized participants. All participants reported under 'Overall Number of Participants Analyzed' contributed data to the table but may not have evaluable data for every row. Number Analyzed signifies number of participants evaluable for specified rows of respective arms. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Improvement From Baseline in Patient Global Impression of Change (PGI-C) Score at Weeks 12 and 24 | Week 12, Week 24 | EAS included all NYHA functional Class II or III randomized participants. All participants reported under 'Overall Number of Participants Analyzed' contributed data to the table but may not have evaluable data for every row. Number Analyzed signifies number of participants evaluable for specified rows of respective arms. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP) at Weeks 4, 12, and 24 | Baseline, Week 4, Week 12, Week 24 | EAS included all NYHA functional Class II or III randomized participants. All participants reported under 'Overall Number of Participants Analyzed' contributed data to the table but may not have evaluable data for every row. Number Analyzed signifies number of participants evaluable for specified rows of respective arms. | picomoles per liter | MEAN |
| SECONDARY | Composite Time to First Occurrence of All-Cause Mortality or Worsening Heart Failure (WHF) | Maximum up to 212.28 weeks (maximum exposure was 208 weeks) | The safety analysis set (SAS) included all participants who received at least 1 dose of study intervention regardless of NYHA functional class. | Weeks | MEDIAN |
| SECONDARY | Overall Survival (OS) | From randomization up to death due to any cause or censored date, maximum up to 212.28 weeks (maximum exposure was of 208 weeks) | The SAS included all participants who received at least 1 dose of study intervention regardless of NYHA functional class. | Weeks | MEDIAN |
| SECONDARY | Number of Participants With Treatment Emergent Adverse Events (AEs) and by Severity | Maximum up to 212.28 weeks (maximum exposure was of 208 weeks) | The SAS included all participants who received at least 1 dose of study intervention regardless of NYHA functional class. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Laboratory Test Abnormalities | Maximum up to 212.28 weeks (maximum exposure was of 208 weeks) | The SAS included all participants who received at least 1 dose of study intervention regardless of NYHA functional class. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group340
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Six-Minute Walk Test (6 MWT) at Week 24 | PF-07265803 (ARRY-371797) | Baseline | 402.500 | Inter-Quartile Range 348.195 to 444.000 | Meter |
| Change From Baseline in Six-Minute Walk Test (6 MWT) at Week 24 | Placebo | Baseline | 393.935 | Inter-Quartile Range 360.000 to 425.500 | Meter |
| Change From Baseline in Six-Minute Walk Test (6 MWT) at Week 24 | PF-07265803 (ARRY-371797) | Week 24 | 420.234 | Inter-Quartile Range 358.902 to 459.117 | Meter |
| Change From Baseline in Six-Minute Walk Test (6 MWT) at Week 24 | Placebo | Week 24 | 393.455 | Inter-Quartile Range 347.409 to 450.826 | Meter |
| Change From Baseline in Six-Minute Walk Test (6 MWT) at Week 24 | PF-07265803 (ARRY-371797) | Week 24 change from baseline | 20.996 | Inter-Quartile Range -22.757 to 51.477 | Meter |
| Change From Baseline in Six-Minute Walk Test (6 MWT) at Week 24 | Placebo | Week 24 change from baseline | 2.679 | Inter-Quartile Range -11.530 to 33.698 | Meter |
| Change From Baseline in 6 MWT at Weeks 4 and 12 | PF-07265803 (ARRY-371797) | Change at Week 4 | 15.40 | Full Range -72.0 to 71.8 | Meter |
| Change From Baseline in 6 MWT at Weeks 4 and 12 | Placebo | Change at Week 4 | -2.60 | Full Range -85.0 to 34.5 | Meter |
| Change From Baseline in 6 MWT at Weeks 4 and 12 | PF-07265803 (ARRY-371797) | Change at Week 12 | 21.47 | Full Range -70.2 to 69.3 | Meter |
| Change From Baseline in 6 MWT at Weeks 4 and 12 | Placebo | Change at Week 12 | 10.00 | Full Range -100.9 to 89.5 | Meter |
1 to 10 of 340Source ClinicalTrials.gov
See all 340 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Double-Blind Treatment Period | STARTED | PF-07265803 (ARRY-371797) | 40 | |
| Double-Blind Treatment Period | STARTED | Placebo | 37 | |
| Double-Blind Treatment Period | COMPLETED | PF-07265803 (ARRY-371797) | 0 | |
| Double-Blind Treatment Period | COMPLETED | Placebo | 0 | |
| Double-Blind Treatment Period | NOT COMPLETED | PF-07265803 (ARRY-371797) | 40 | |
| Double-Blind Treatment Period | NOT COMPLETED | Placebo | 37 | |
| Open-Label Treatment Period | STARTED | PF-07265803 (ARRY-371797) | 0 | |
| Open-Label Treatment Period | STARTED | Placebo | 0 | |
| Open-Label Treatment Period | COMPLETED | PF-07265803 (ARRY-371797) | 0 | |
| Open-Label Treatment Period | COMPLETED | Placebo | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events106
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Angina pectoris | Cardiac disorders | PF-07265803 (ARRY-371797) | 3 | 40 | 3 |
| other | Angina pectoris | Cardiac disorders | Placebo | 1 | 37 | 1 |
| other | Atrial fibrillation | Cardiac disorders | PF-07265803 (ARRY-371797) | 6 | 40 | 7 |
| other | Atrial fibrillation | Cardiac disorders | Placebo | 5 | 37 | 10 |
| other | Atrial flutter | Cardiac disorders | PF-07265803 (ARRY-371797) | 3 | 40 | 4 |
| other | Atrial flutter | Cardiac disorders | Placebo | 2 | 37 | 5 |
| other | Cardiac failure acute | Cardiac disorders | PF-07265803 (ARRY-371797) | 2 | 40 | 2 |
| other | Cardiac failure acute | Cardiac disorders | Placebo | 2 | 37 | 2 |
| other | Ventricular tachycardia | Cardiac disorders | PF-07265803 (ARRY-371797) | 7 | 40 | 10 |
| other | Ventricular tachycardia | Cardiac disorders | Placebo | 8 | 37 | 12 |
1 to 10 of 106Source ClinicalTrials.gov
See all 106 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | PF-07265803 (ARRY-371797) | 3 | 40 |
| Total, all-cause mortality | Placebo | 3 | 37 |
| Total, other adverse events | PF-07265803 (ARRY-371797) | 31 | 40 |
| Total, other adverse events | Placebo | 27 | 37 |
| Total, serious adverse events | PF-07265803 (ARRY-371797) | 10 | 40 |
| Total, serious adverse events | Placebo | 21 | 37 |
Source ClinicalTrials.gov
Papers5
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| PF-07265803 (ARRY-371797) vs Placebo: deaths from any cause | Trial comparisons |
| PF-07265803 (ARRY-371797) vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03439514
GET https://conductscience.org/api/trials/v1/NCT03439514/results?page=2