Trial Outcomes & Findings for A Study of Mavacamten in Participants With HFpEF and Elevation of NT-proBNP With or Without Elevation of cTnT (NCT NCT04766892)
NCT ID: NCT04766892
Last Updated: 2025-03-20
Results Overview
Treatment-emergent Adverse event (TEAE) is defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product; it does not necessarily have to have a causal relationship with this treatment.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
30 participants
Primary outcome timeframe
From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
Results posted on
2025-03-20
Participant Flow
30 participants enrolled and treated
Participant milestones
| Measure |
Mavacamten
Mavacamten
|
|---|---|
|
Overall Study
STARTED
|
30
|
|
Overall Study
Safety Population
|
30
|
|
Overall Study
Intent to Treat Population
|
30
|
|
Overall Study
COMPLETED
|
24
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
Mavacamten
Mavacamten
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Lost to Follow-up
|
1
|
|
Overall Study
Adverse Event
|
3
|
|
Overall Study
Left ventricular ejection fraction (LVEF) Criteria Met
|
1
|
Baseline Characteristics
A Study of Mavacamten in Participants With HFpEF and Elevation of NT-proBNP With or Without Elevation of cTnT
Baseline characteristics by cohort
| Measure |
Mavacamten
n=30 Participants
Mavacamten
|
|---|---|
|
Age, Continuous
|
75.0 Years
STANDARD_DEVIATION 7.58 • n=5 Participants
|
|
Sex: Female, Male
Female
|
16 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
23 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
26 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)Population: Safety Analysis Population
Treatment-emergent Adverse event (TEAE) is defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product; it does not necessarily have to have a causal relationship with this treatment.
Outcome measures
| Measure |
Mavacamten
n=30 Participants
Mavacamten
|
|---|---|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
|
23 Participants
|
PRIMARY outcome
Timeframe: From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)Population: Safety Analysis Population
Adverse event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product; it does not necessarily have to have a causal relationship with this treatment. AEs of special interest include: Symptomatic overdose, teratogenicity, and LVEF ≤30%
Outcome measures
| Measure |
Mavacamten
n=30 Participants
Mavacamten
|
|---|---|
|
Number of Participants With Adverse Events of Special Interest (AESIs)
Symptomatic Overdose
|
0 Participants
|
|
Number of Participants With Adverse Events of Special Interest (AESIs)
Outcomes of Pregnancy
|
0 Participants
|
|
Number of Participants With Adverse Events of Special Interest (AESIs)
Left Ventricular Ejection Fraction (LVEF) ≤30%
|
0 Participants
|
PRIMARY outcome
Timeframe: From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)Population: Safety Analysis Population
Treatment-emergent serious adverse event (TESAE) is defined as any untoward medical occurrence at any dose that: Results in death, Is immediately life-threatening (places the participant at immediate risk of death from the event as it occurred), Requires inpatient hospitalization or prolongation of existing hospitalization, Results in persistent or significant disability or incapacity or substantial disruption of the ability to conduct normal life functions, Results in a congenital abnormality or birth defect, Is an important medical event that may not result in death, be life-threatening, or require hospitalization, but may be considered an SAE when, based upon appropriate medical judgment, it may require medical or surgical intervention to prevent any of the outcomes listed above.
Outcome measures
| Measure |
Mavacamten
n=30 Participants
Mavacamten
|
|---|---|
|
Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs)
|
5 Participants
|
PRIMARY outcome
Timeframe: From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)Population: Safety Analysis Population
Blood samples were collected to assess the abnormalities in laboratory parameters.
Outcome measures
| Measure |
Mavacamten
n=30 Participants
Mavacamten
|
|---|---|
|
Number of Participants With Laboratory Abnormalities Reported as Treatment-Emergent Adverse Events (TEAEs)
|
4 Participants
|
PRIMARY outcome
Timeframe: From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)Population: Safety Analysis Population
Treatment-emergent Adverse event (TEAE) is defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product; it does not necessarily have to have a causal relationship with this treatment.
Outcome measures
| Measure |
Mavacamten
n=30 Participants
Mavacamten
|
|---|---|
|
Number of Participants With Abnormal Vital Signs Reported as Treatment-Emergent Adverse Events (TEAEs)
|
4 Participants
|
PRIMARY outcome
Timeframe: From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)Population: Safety Analysis Population
Treatment-emergent Adverse event (TEAE) is defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product; it does not necessarily have to have a causal relationship with this treatment.
Outcome measures
| Measure |
Mavacamten
n=30 Participants
Mavacamten
|
|---|---|
|
Number of Participants With Cardiac Rhythm Abnormalities Reported as Treatment-Emergent Adverse Events (TEAEs)
|
5 Participants
|
PRIMARY outcome
Timeframe: At Week 26Population: All Intention-To-Treat Participants
Ratio to baseline in N-terminal pro B-type natriuretic peptide levels. The baseline value is defined as the last available value before the first administration of study drug.
Outcome measures
| Measure |
Mavacamten
n=30 Participants
Mavacamten
|
|---|---|
|
Ratio to Baseline at Week 26 in Resting N-Terminal Pro B-Type Natriuretic Peptide (NT-proBNP) Levels
|
0.7354 Ratio
Interval 0.5612 to 0.9637
|
PRIMARY outcome
Timeframe: At Week 26Population: All Intention-To-Treat Participants
Ratio to Baseline in resting high sensitivity cardiac troponin T (hs-cTnT) levels. The baseline value is defined as the last available value before the first administration of study drug.
Outcome measures
| Measure |
Mavacamten
n=30 Participants
Mavacamten
|
|---|---|
|
Ratio to Baseline at Week 26 in Resting High Sensitivity Cardiac Troponin T (Hs-cTnT) Levels Assessed by High-Sensitivity Assay
|
0.8645 Ratio
Interval 0.7711 to 0.9693
|
Adverse Events
Mavacamten
Serious events: 5 serious events
Other events: 18 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Mavacamten
n=30 participants at risk
Mavacamten
|
|---|---|
|
Cardiac disorders
Atrial fibrillation
|
3.3%
1/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
|
Cardiac disorders
Cardiac failure acute
|
3.3%
1/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
|
Eye disorders
Eye haemorrhage
|
3.3%
1/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
|
Infections and infestations
Gastroenteritis
|
3.3%
1/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
|
Metabolism and nutrition disorders
Dehydration
|
3.3%
1/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma of skin
|
3.3%
1/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
|
Renal and urinary disorders
Renal impairment
|
3.3%
1/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
Other adverse events
| Measure |
Mavacamten
n=30 participants at risk
Mavacamten
|
|---|---|
|
Cardiac disorders
Atrial flutter
|
6.7%
2/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
|
General disorders
Asthenia
|
6.7%
2/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
|
Infections and infestations
COVID-19
|
13.3%
4/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
|
Infections and infestations
Upper respiratory tract infection
|
10.0%
3/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
|
Infections and infestations
Urinary tract infection
|
6.7%
2/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
|
Injury, poisoning and procedural complications
Fall
|
13.3%
4/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
|
Nervous system disorders
Dizziness
|
13.3%
4/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
|
Nervous system disorders
Headache
|
10.0%
3/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
10.0%
3/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
|
Vascular disorders
Hypertension
|
10.0%
3/30 • Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
All-Cause Mortality, Serious Adverse Events and Other (Not Including Serious) Adverse Events represents all participants that received at least 1 dose of study medication. Pre-specified for all doses to be collected combined per treatment.
|
Additional Information
Bristol-Myers Squibb Study Director
Bristol-Myers Squibb
Phone: Please email
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Bristol-Myers Squibb Co. agreements with investigators vary; constant is our right to embargo communications regarding trial results prior to public release for a period ≤60 days from submittal for review. We will not prohibit investigators from publishing but will prohibit the disclosure of previously undisclosed confidential information other than study results, and request postponement of single-center publications until after disclosure of the clinical trial's primary publication.
- Publication restrictions are in place
Restriction type: OTHER