A Study of Mavacamten in Adolescent Participants with Symptomatic Obstructive HCM

• Research type

  Research Study

• Full 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 (SCOUT-HCM)

• IRAS ID

  1008713

• Contact name

  GSM-CT Representative

• Contact email

  mg-gsm-ct@bms.com

• Sponsor organisation

  Bristol-Myers Squibb Services Unlimited Company

• ISRCTN Number

  Not Available

• Clinicaltrials.gov Identifier

  NCT06253221

• Research summary

  This is a Phase 3, randomized, placebo-controlled, double-blind, multi-center, international, parallel-group study to evaluate the efficacy safety, and pharmacokinetics (PK) of mavacamten in adolescent patients with symptomatic obstructive HCM.
  The purpose of this study is to test the hypothesis that treatment with oral mavacamten compared to placebo improves health status (symptoms and physical limitations) and increases exercise capacity in symptomatic adolescents with obstructive HCM. The effect of treatment on health status and exercise capacity will be extrapolated using change from baseline in VLVOT.

  Double-blind treatment with oral mavacamten or placebo will occur for the first 28 weeks. The primary endpoint for all participants will be assessed at Week 28. Participants randomized to placebo will receive mavacamten starting at Week 28; all participants will receive mavacamten until the end of treatment visit at Week 56. A separate rollover/extension study may be offered to eligible participants who finish their study intervention per protocol.
  About 40 study participants will be taking part in this study globally and participation will last up to 79 weeks. This is counted from the date that the participant signs the Informed Consent Form, until their last study visit.

  Study intervention is defined as mavacamten or placebo.
  The study has 4 phases:
  • Screening (up to 5 weeks)
  • Double-blind Placebo-controlled (28 weeks)
  • Double-blind Active treatment (28 weeks)
  • Follow-up (participants not continuing to the rollover/extension: 8 or 18 weeks for CYP2C19 poor metabolizers)

  Patients will visit the study hospital for the following:
  • Day 1
  • Week 4
  • Week 5
  • Week 8
  • Week 9
  • Week 11
  • Week 12
  • Week 16
  • Week 20
  • Week 23
  • Week 24
  • Week 28
  • Week 32
  • Week 33
  • Week 36
  • Week 37
  • Week 39
  • Week 40
  • Week 44
  • Week 48
  • Week 51
  • Week 52
  • Week 56/EOT
  • Week 64/EOS 1
  • Week 74/EOS 2

• REC name

  West of Scotland REC 1

• REC reference

  24/WS/0016

• Date of REC Opinion

  18 Mar 2024

• REC opinion

  Further Information Favourable Opinion