Abstract 4132223: Mavacamten in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy: design of the Phase 3 SCOUT-HCM trial
Bibliographic record
Abstract
Background: While most current treatments can improve symptoms in patients with obstructive hypertrophic cardiomyopathy (HCM), they may be poorly tolerated and do not target the underlying pathophysiology. Mavacamten is the first and only cardiac myosin inhibitor approved for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive HCM but has not been evaluated in a pediatric age group. SCOUT-HCM (Study of MavaCamten in AdOlescents with Symptomatic ObstrUcTive HCM; NCT06253221) will evaluate the efficacy, safety, and pharmacokinetics of mavacamten in adolescents with symptomatic obstructive HCM. Methods: SCOUT-HCM is a randomized, double-blind, placebo-controlled, international phase 3 clinical trial that will enroll 40 adolescents (age 12 to <18 years) with obstructive HCM ( Figure ). Patients will receive mavacamten or placebo during the first 28 weeks, followed by a 28-week double-blind active treatment period. Inclusion criteria include a diagnosis of HCM with presence of symptoms and left ventricular outflow tract (LVOT) obstruction. Patients with HCM phenocopies, LVEF <50% at rest in the past 6 months, planned escalation in HCM therapy or upcoming intervention (eg, major cardiac surgery) will be excluded. The primary endpoint is change in Valsalva LVOT gradient from baseline to week 28. Key secondary endpoints include other echocardiographic parameters, safety, pharmacokinetics, and exercise capacity. Conclusions: SCOUT-HCM is the first randomized controlled trial evaluating a targeted myosin inhibitor in adolescents with symptomatic obstructive HCM. The results will inform the utility of mavacamten in adolescent patients with symptomatic obstructive HCM.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.002 | 0.001 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.002 | 0.001 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.002 | 0.002 |
| Insufficient payload (model declined to judge) | 0.006 | 0.001 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".