Mavacamten in symptomatic adolescent patients with obstructive hypertrophic cardiomyopathy: design of the phase 3 SCOUT-HCM trial
Bibliographic record
Abstract
BACKGROUND: Mavacamten, a first-in-class cardiac myosin inhibitor, is approved internationally for the treatment of symptomatic adult patients with obstructive hypertrophic cardiomyopathy (HCM) and has been shown to improve cardiac function and symptoms in adult patients across multiple phase 3 trials. The efficacy and safety of mavacamten in pediatric patients with obstructive HCM has not been evaluated. METHODS: SCOUT-HCM is a phase 3, randomized, placebo-controlled, double-blind, parallel-group, multicenter, international study in symptomatic adolescent patients (12 years to <18 years old) with obstructive HCM. The aim of the study is to assess the efficacy, safety, and pharmacokinetics of mavacamten in this population. Participants will be randomized 1:1 to mavacamten or placebo for 28 weeks, followed by a 28-week active-treatment period (when patients randomized to placebo will cross over to mavacamten) and an open-label long-term extension period for ≤144 weeks. Participants will initiate mavacamten at a dosage of 2.5 mg/day or 5 mg/day; dose titration will be based on echocardiographic assessment of Valsalva left ventricular (LV) outflow tract (LVOT) gradient and LV ejection fraction. The primary endpoint is change from baseline to week 28 in Valsalva LVOT gradient. Secondary endpoints include efficacy parameters of resting and post-exercise LVOT gradients, peak oxygen consumption, symptoms, and health status, plus safety and pharmacokinetic parameters. CONCLUSIONS: SCOUT-HCM is the first clinical trial to evaluate a cardiac myosin inhibitor in adolescent patients with obstructive HCM. SCOUT-HCM will assess the utility of mavacamten in this patient population with an unmet clinical need. TRIAL REGISTRATION: ClinicalTrials.gov: NCT06253221.
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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 | 0.002 |
| 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 | 0.001 | 0.002 |
| Insufficient payload (model declined to judge) | 0.004 | 0.000 |
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".