Cardiomyopathy, HypertrophicPhase 3NCT06253221
A Study to Evaluate Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy
A Phase 3 study testing Mavacamten in people with Cardiomyopathy, Hypertrophic, run by Bristol-Myers Squibb at 47 sites in 9 countries.
Trial summary
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of mavacamten in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM). Read more about this study
- Who is it for?
- Ages 12 to 17 years
- Conditions the study is for
- Cardiomyopathy, Hypertrophic
- What is being tested?
- Mavacamten
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 44 participants
- Where
- 47 sitesin 9 countries
- Study dates
- 17 Apr 2024to 28 Mar 2031
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 12 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosis of HCM
- Presence of LVOT obstruction
- Presence of symptoms
Who cannot join
- Phenocopy diseases resulting in myocardial hypertrophy not related to sarcomere dysfunction
- Evidence of LVEF <50% in prior 6 months
- Planned escalation in HCM therapy or upcoming intervention (eg, major cardiac surgery, HCM medication dose increase)
- Other protocol-defined Inclusion/Exclusion criteria apply.
Trial locations and contacts
47 sites in 9 countries.
47 sites in 9 countries
United States31 sites
Local Institution - 0002
Ann Arbor, Michigan, 48109
Local Institution - 0053
Atlanta, Georgia, 30329-3117
Local Institution - 0038
Aurora, Colorado, 80045-7106
Local Institution - 0045
Austin, Texas, 78712
Local Institution - 0017
Birmingham, Alabama, 35294-0004
Local Institution - 0043
Boston, Massachusetts, 02115-5724
Local Institution - 0015
Charlotte, North Carolina, 28204
Local Institution - 0012
Charlottesville, Virginia, 22908-0816
Local Institution - 0013
Chicago, Illinois, 60637-1447
Local Institution - 0019
Cincinnati, Ohio, 45229
Local Institution - 0001
Cleveland, Ohio, 44195-0001
Local Institution - 0029
Columbus, Ohio, 43205
Local Institution - 0039
Durham, North Carolina, 27705
Local Institution - 0054
Houston, Texas, 77030
Local Institution - 0009
Indianapolis, Indiana, 46202-5272
Local Institution - 0032
Los Angeles, California, 90027-6062
Local Institution - 0008
Los Angeles, California, 90095-8344
Local Institution - 0034
Memphis, Tennessee, 38105
Local Institution - 0052
Morristown, New Jersey, 07960
Local Institution - 0010
New Hyde Park, New York, 11042
Local Institution - 0024
New York, New York, 10029-6574
Local Institution - 0036
New York, New York, 10032-1559
Local Institution - 0033
Palo Alto, California, 94304-1601
Local Institution - 0030
Philadelphia, Pennsylvania, 19104-4319
Local Institution - 0050
Phoenix, Arizona, 85016-7710
Local Institution - 0005
Pittsburgh, Pennsylvania, 15224-1334
Local Institution - 0003
Salt Lake City, Utah, 84113-1103
Local Institution - 0044
San Diego, California, 92123-4223
Local Institution - 0037
St Louis, Missouri, 63110
Local Institution - 0031
St. Petersburg, Florida, 33701-4634
Local Institution - 0040
The Bronx, New York, 10467-2403
Italy3 sites
Local Institution - 0016
Florence, FI, 50100
Local Institution - 0051
Genova, GE, 16147
Local Institution - 0027
Naples, 80131
Australia2 sites
Local Institution - 0042
Clayton, Victoria, 3168
Local Institution - 0020
Sydney, New South Wales, 2145
Canada2 sites
Local Institution - 0041
Edmonton, Alberta, T6G 2R7
Local Institution - 0046
Toronto, Ontario, M5G 1X8
France2 sites
Local Institution - 0022
Paris, 75015
Local Institution - 0026
Pessac, 33604
Germany2 sites
Local Institution - 0018
Berlin, State of Berlin, 13353
Local Institution - 0006
Munich, Bavaria, 80636
Spain2 sites
Local Institution - 0014
Barcelona, B, 8950
Local Institution - 0025
Madrid, 28046
United Kingdom2 sites
Local Institution - 0049
Belfast, BFS, BT12 6BA
Local Institution - 0007
London, WC1N 3JH
About this study
Official title: A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adolescents (Age 12 Years to < 18 Years) With Symptomatic Obstructive Hypertrophic Cardiomyopathy
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of mavacamten in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).
- Conditions
- Cardiomyopathy, Hypertrophic
- Being tested
- Mavacamten, Placebo
- Keywords
- HCM, mavacamten, Obstructive Hypertrophic Cardiomyopathy, SCOUT, SCOUT-HCM, Pediatric HCM, Pediatric hypertrophic cardiomyopathy, Symptomatic HCM, oHCM
- Medical subject headings
- Cardiomyopathy, Hypertrophic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MavacamtenDrug
Also called BMS-986427
Specified dose on specified days
PlaceboDrug
Specified dose on specified days
| Study group | Role | What they receive |
|---|---|---|
| Mavacamten | Experimental | Participants assigned to this arm will receive mavacamten (1 mg to 15 mg) from day 1 to end of treatment at week 200. Receives: Drug: Mavacamten |
| Placebo | Experimental | Participants assigned to this arm will receive mavacamten (1 mg to 15 mg) from week 28 to end of treatment at week 200. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in Valsalva left ventricular outflow tract (LVOT) (VLVOT) gradient
Measured: At Week 28
Secondary outcomes (19)
Change from baseline in resting LVOT gradient
Measured: At Week 28
Change from baseline in post-exercise peak LVOT gradient
Measured: At Week 28
Change from baseline in maximal wall thickness
Measured: At Week 28
Change from baseline in ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e')
Measured: At Week 28
Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2)
Measured: From baseline up to Week 28
Proportion of participants achieving a reduction from baseline to Week 28 in maximal LVOT gradient to < 30 mmHg
Measured: From baseline up to Week 28
Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) class from baseline to Week 28
Measured: From baseline up to Week 28
Proportion of participants with at least 1 grade improvement in mitral regurgitation at Week 28
Measured: From baseline up to Week 28
Number of participants with treatment-emergent adverse events (TEAEs)
Measured: Up to Week 218
Number of participants with treatment-emergent serious adverse events (TESAEs)
Measured: Up to Week 218
Change from baseline in electrocardiogram (ECG) (QT interval)
Measured: At Week 28
Number of participants with left ventricular ejection fraction (LVEF) ≤ 30%
Measured: Up to Week 200
Number of participants with LVEF < 50%
Measured: Up to Week 200
Trough observed plasma concentration (Ctrough)
Measured: Up to Week 200
Post-dose plasma concentration of mavacamten
Measured: Up to Week 200
Maximum observed concentration (Cmax)
Measured: Up to Week 200
Area under the concentration-time curve (AUC)
Measured: Up to Week 200
Proportion of participants who evaluate taste and swallowability as neutral or better using taste and swallowability scales
Measured: At Day 1 and Week 11
Change from baseline in the Hypertrophic Cardiomyopathy Symptom Questionnaire - Shortness of Breath (HCMSQ SoB) domain
Measured: At Week 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Feb 2024
- Study start17 Apr 2024
- Primary completion25 Nov 2025
- Study completion28 Mar 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Cardiomyopathy, HypertrophicStudies of the same condition, open and under way.
- More trials run by Bristol-Myers SquibbThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bristol-Myers Squibb
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bristol-Myers Squibb, Study director, Bristol-Myers Squibb
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06253221
- Sponsor's protocol ID
- CV027-010
- Other IDs
- 2023-505650-17-00, U1111-1290-1175, 2023
- Status last verified
- Dec 2025
- First submitted
- 2 Feb 2024
- Last updated
- 19 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myer Squibb's data sharing policy and process can be found at: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosure-commitment.html
Publications
Rossano JW, Canter C, Wolf CM, Papez A, Gambra M, Bryant RM, Alejos J, McCulloch M, Sarquella Brugada G, Bock MJ, Jeewa A, Pearce FB, Desai MY, Favatella N, Javidialsaadi A, Phung V, Rano T, Zhu L, Dyme JL, Mital S; SCOUT-HCM Investigators. Mavacamten in Adolescents with Obstructive Hypertrophic Cardiomyopathy. N Engl J Med. 2026 Jul 23;395(4):362-373. doi: 10.1056/NEJMoa2601103. Epub 2026 Mar 29.
PubMed 41910394Rossano J, Canter C, Wolf C, Favatella N, Lockman J, Puli S, Javidialsaadi A, Dyme J, Crevar C, Mital S. Mavacamten in symptomatic adolescent patients with obstructive hypertrophic cardiomyopathy: design of the phase 3 SCOUT-HCM trial. Am Heart J. 2026 Feb;292:107283. doi: 10.1016/j.ahj.2025.107283. Epub 2025 Sep 30.
PubMed 41038603
Study at a glance
From the registry record, last updated by the sponsor on 19 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adolescents (Age 12 Years to < 18 Years) With Symptomatic Obstructive Hypertrophic Cardiomyopathy
- ClinicalTrials.gov ID
- NCT06253221
- Acronym / sponsor ID
- CV027-010
- Conditions
- Cardiomyopathy, Hypertrophic
- Keywords
- HCM, mavacamten, Obstructive Hypertrophic Cardiomyopathy, SCOUT, SCOUT-HCM, Pediatric HCM, Pediatric hypertrophic cardiomyopathy, Symptomatic HCM
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 44 participants (actual)
- Lead sponsor
- Bristol-Myers SquibbIndustry (pharma, biotech, devices)
- Study start
- 17 Apr 2024 (actual)
- Primary completion
- 25 Nov 2025 (actual)
- Study completion
- 28 Mar 2031 (estimated)
- First posted
- 12 Feb 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of mavacamten in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Mavacamten | Experimental | Drug: MavacamtenParticipants assigned to this arm will receive mavacamten (1 mg to 15 mg) from day 1 to end of treatment at week 200. |
| Placebo | Experimental | Drug: PlaceboParticipants assigned to this arm will receive mavacamten (1 mg to 15 mg) from week 28 to end of treatment at week 200. |
Who may qualify
- Ages
- 12 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Diagnosis of HCM
- Presence of LVOT obstruction
- Presence of symptoms
Key exclusion criteria (4 listed)
- Phenocopy diseases resulting in myocardial hypertrophy not related to sarcomere dysfunction
- Evidence of LVEF <50% in prior 6 months
- Planned escalation in HCM therapy or upcoming intervention (eg, major cardiac surgery, HCM medication dose increase)
- Other protocol-defined Inclusion/Exclusion criteria apply.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MavacamtenAlso: BMS-986427Specified dose on specified days | Drug | Mavacamten |
| PlaceboSpecified dose on specified days | Drug | Placebo |
Primary outcome
Change from baseline in Valsalva left ventricular outflow tract (LVOT) (VLVOT) gradient
Time frame: At Week 28
19 secondary outcomes
Change from baseline in resting LVOT gradient
Time frame: At Week 28
Change from baseline in post-exercise peak LVOT gradient
Time frame: At Week 28
Change from baseline in maximal wall thickness
Time frame: At Week 28
Change from baseline in ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e')
Time frame: At Week 28
Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2)
Time frame: From baseline up to Week 28
Proportion of participants achieving a reduction from baseline to Week 28 in maximal LVOT gradient to < 30 mmHg
Time frame: From baseline up to Week 28
Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) class from baseline to Week 28
Time frame: From baseline up to Week 28
Proportion of participants with at least 1 grade improvement in mitral regurgitation at Week 28
Time frame: From baseline up to Week 28
Number of participants with treatment-emergent adverse events (TEAEs)
Time frame: Up to Week 218
Number of participants with treatment-emergent serious adverse events (TESAEs)
Time frame: Up to Week 218
Change from baseline in electrocardiogram (ECG) (QT interval)
Time frame: At Week 28
Number of participants with left ventricular ejection fraction (LVEF) ≤ 30%
Time frame: Up to Week 200
Number of participants with LVEF < 50%
Time frame: Up to Week 200
Trough observed plasma concentration (Ctrough)
Time frame: Up to Week 200
Post-dose plasma concentration of mavacamten
Time frame: Up to Week 200
Maximum observed concentration (Cmax)
Time frame: Up to Week 200
Area under the concentration-time curve (AUC)
Time frame: Up to Week 200
Proportion of participants who evaluate taste and swallowability as neutral or better using taste and swallowability scales
Time frame: At Day 1 and Week 11
Change from baseline in the Hypertrophic Cardiomyopathy Symptom Questionnaire - Shortness of Breath (HCMSQ SoB) domain
Time frame: At Week 28
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Dec 2025.
- Derived from this study: PubMed 41910394Rossano JW, Canter C, Wolf CM, Papez A, Gambra M, Bryant RM, Alejos J, McCulloch M, Sarquella Brugada G, Bock MJ, Jeewa A, Pearce FB, Desai MY, Favatella N, Javidialsaadi A, Phung V, Rano T, Zhu L, Dyme JL, Mital S; SCOUT-HCM Investigators. Mavacamten in Adolescents with Obstructive Hypertrophic Cardiomyopathy. N Engl J Med. 2026 Jul 23;395(4):362-373. doi: 10.1056/NEJMoa2601103. Epub 2026 Mar 29.
- Derived from this study: PubMed 41038603Rossano J, Canter C, Wolf C, Favatella N, Lockman J, Puli S, Javidialsaadi A, Dyme J, Crevar C, Mital S. Mavacamten in symptomatic adolescent patients with obstructive hypertrophic cardiomyopathy: design of the phase 3 SCOUT-HCM trial. Am Heart J. 2026 Feb;292:107283. doi: 10.1016/j.ahj.2025.107283. Epub 2025 Sep 30.
- BMS Clinical Trial InformationLink provided by the sponsor.
- BMS Clinical Trial Patient RecruitingLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myer Squibb's data sharing policy and process can be found at: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosure-commitment.html
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bristol-Myers Squibb study director
Bristol-Myers Squibb
47 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Mavacamten in people with Cardiomyopathy, Hypertrophic, run by Bristol-Myers Squibb at 47 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. 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: under way, not looking for participants, as of the sponsor's last update on 19 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 47 sites in United States, Italy, Australia, Canada, France and 4 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?
This study is still under way. Results are not available in the source record yet.
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 Valsalva left ventricular outflow tract (LVOT) (VLVOT) gradient (time frame: At Week 28). 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 NCT06253221, which also lists 2 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 19 Dec 2025.