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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06253221




Registration number
NCT06253221
Ethics application status
Date submitted
2/02/2024
Date registered
12/02/2024

Titles & IDs
Public title
A Study to Evaluate Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy
Scientific 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
Secondary ID [1] 0 0
2023-505650-17-00
Secondary ID [2] 0 0
CV027-010
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cardiomyopathy, Hypertrophic 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Other cardiovascular diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Mavacamten
Treatment: Drugs - Placebo

Experimental: Mavacamten - Participants assigned to this arm will receive mavacamten (1 mg to 15 mg) from day 1 to end of treatment at week 200.

Experimental: Placebo - Participants assigned to this arm will receive mavacamten (1 mg to 15 mg) from week 28 to end of treatment at week 200.


Treatment: Drugs: Mavacamten
Specified dose on specified days

Treatment: Drugs: Placebo
Specified dose on specified days
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change from baseline in Valsalva left ventricular outflow tract (LVOT) (VLVOT) gradient
Timepoint [1] 0 0
At Week 28
Secondary outcome [1] 0 0
Change from baseline in resting LVOT gradient
Timepoint [1] 0 0
At Week 28
Secondary outcome [2] 0 0
Change from baseline in post-exercise peak LVOT gradient
Timepoint [2] 0 0
At Week 28
Secondary outcome [3] 0 0
Change from baseline in maximal wall thickness
Timepoint [3] 0 0
At Week 28
Secondary outcome [4] 0 0
Change from baseline in ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e')
Timepoint [4] 0 0
At Week 28
Secondary outcome [5] 0 0
Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2)
Timepoint [5] 0 0
From baseline up to Week 28
Secondary outcome [6] 0 0
Proportion of participants achieving a reduction from baseline to Week 28 in maximal LVOT gradient to < 30 mmHg
Timepoint [6] 0 0
From baseline up to Week 28
Secondary outcome [7] 0 0
Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) class from baseline to Week 28
Timepoint [7] 0 0
From baseline up to Week 28
Secondary outcome [8] 0 0
Proportion of participants with at least 1 grade improvement in mitral regurgitation at Week 28
Timepoint [8] 0 0
From baseline up to Week 28
Secondary outcome [9] 0 0
Number of participants with treatment-emergent adverse events (TEAEs)
Timepoint [9] 0 0
Up to Week 218
Secondary outcome [10] 0 0
Number of participants with treatment-emergent serious adverse events (TESAEs)
Timepoint [10] 0 0
Up to Week 218
Secondary outcome [11] 0 0
Change from baseline in electrocardiogram (ECG) (QT interval)
Timepoint [11] 0 0
At Week 28
Secondary outcome [12] 0 0
Number of participants with left ventricular ejection fraction (LVEF) = 30%
Timepoint [12] 0 0
Up to Week 200
Secondary outcome [13] 0 0
Number of participants with LVEF = 50%
Timepoint [13] 0 0
Up to Week 200
Secondary outcome [14] 0 0
Trough observed plasma concentration (Ctrough)
Timepoint [14] 0 0
Up to Week 200
Secondary outcome [15] 0 0
Post-dose plasma concentration of mavacamten
Timepoint [15] 0 0
Up to Week 200
Secondary outcome [16] 0 0
Maximum observed concentration (Cmax)
Timepoint [16] 0 0
Up to Week 200
Secondary outcome [17] 0 0
Area under the concentration-time curve (AUC)
Timepoint [17] 0 0
Up to Week 200
Secondary outcome [18] 0 0
Proportion of participants who evaluate taste and swallowability as neutral or better using taste and swallowability scales
Timepoint [18] 0 0
At Day 1 and Week 11
Secondary outcome [19] 0 0
Change from baseline in the Hypertrophic Cardiomyopathy Symptom Questionnaire - Shortness of Breath (HCMSQ SoB) domain
Timepoint [19] 0 0
At Week 28

Eligibility
Key inclusion criteria
* Diagnosis of HCM
* Presence of LVOT obstruction
* Presence of symptoms
Minimum age
12 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
17/04/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
28/03/2031
Actual
Sample size
Target
40
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Local Institution - 0020 - Sydney
Recruitment hospital [2] 0 0
Local Institution - 0042 - Clayton
Recruitment hospital [3] 0 0
Local Institution - 0028 - Parkville
Recruitment postcode(s) [1] 0 0
2145 - Sydney
Recruitment postcode(s) [2] 0 0
3168 - Clayton
Recruitment postcode(s) [3] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Colorado
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Georgia
Country [7] 0 0
United States of America
State/province [7] 0 0
Illinois
Country [8] 0 0
United States of America
State/province [8] 0 0
Indiana
Country [9] 0 0
United States of America
State/province [9] 0 0
Massachusetts
Country [10] 0 0
United States of America
State/province [10] 0 0
Michigan
Country [11] 0 0
United States of America
State/province [11] 0 0
Missouri
Country [12] 0 0
United States of America
State/province [12] 0 0
New Jersey
Country [13] 0 0
United States of America
State/province [13] 0 0
New York
Country [14] 0 0
United States of America
State/province [14] 0 0
North Carolina
Country [15] 0 0
United States of America
State/province [15] 0 0
Ohio
Country [16] 0 0
United States of America
State/province [16] 0 0
Pennsylvania
Country [17] 0 0
United States of America
State/province [17] 0 0
Tennessee
Country [18] 0 0
United States of America
State/province [18] 0 0
Texas
Country [19] 0 0
United States of America
State/province [19] 0 0
Utah
Country [20] 0 0
United States of America
State/province [20] 0 0
Virginia
Country [21] 0 0
United States of America
State/province [21] 0 0
Wisconsin
Country [22] 0 0
Canada
State/province [22] 0 0
Alberta
Country [23] 0 0
Canada
State/province [23] 0 0
Ontario
Country [24] 0 0
France
State/province [24] 0 0
Paris
Country [25] 0 0
France
State/province [25] 0 0
Pessac
Country [26] 0 0
Germany
State/province [26] 0 0
BE
Country [27] 0 0
Germany
State/province [27] 0 0
BY
Country [28] 0 0
Ireland
State/province [28] 0 0
Dublin 12, D12 N512
Country [29] 0 0
Italy
State/province [29] 0 0
FI
Country [30] 0 0
Italy
State/province [30] 0 0
GE
Country [31] 0 0
Italy
State/province [31] 0 0
Napoli
Country [32] 0 0
Spain
State/province [32] 0 0
B
Country [33] 0 0
Spain
State/province [33] 0 0
GR
Country [34] 0 0
Spain
State/province [34] 0 0
M
Country [35] 0 0
Spain
State/province [35] 0 0
A Coruna
Country [36] 0 0
Spain
State/province [36] 0 0
Madrid
Country [37] 0 0
United Kingdom
State/province [37] 0 0
BFS
Country [38] 0 0
United Kingdom
State/province [38] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Bristol-Myers Squibb
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Bristol-Myers Squibb
Address 0 0
Bristol-Myers Squibb
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
BMS Study Connect Contact Center www.BMSStudyConnect.com
Address 0 0
Country 0 0
Phone 0 0
855-907-3286
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
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

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Clinical study report (CSR)
When will data be available (start and end dates)?
See plan description
Available to whom?
See plan description
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosure-commitment.html


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06253221