At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Aficamten (drug), Placebo (drug)
- How long the study runs
- Study runs about 68 months (dates as stated)
- About the drug or intervention
- Aficamten — drug: Oral Tablet · Placebo — drug: Oral Tablet
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 12 Years to 17 Years
- Who
- All
- Number of participants
- 65
- Started
- 2024-05-29
- Last checked
- 2026-08
Plain English Summary
What is this study?
- • Testing a new treatment for pediatric
- • Phase2/Phase3 - 65 participants
- • The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM)
Who can take part?
- • Ages 12 Years to 17 Years
- • Diagnosed with pediatric
Where?
- • Liverpool - Alder Hey Children's Hospital
- • London - Evelina Children's Hospital
- • London - Great Ormond Street Hospital for Children
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
More detail
The overall objective of the trial is to determine the efficacy, safety, and tolerability of administration of aficamten in adolescents (12 to \< 18 years old) and children (6 to \< 12 years old) with symptomatic oHCM. Adolescents and children will be studied in a staged approach involving established favorable pharmacodynamic and safety profiles of aficamten in adolescents followed by further pharmacokinetic modeling to inform the dosing regimen in children. The 12 to \<18 years old cohort has enrolled all participants and is no longer recruiting, the 6 to \< 12 year old cohort is not yet recruiting. The trial will consist of 3 periods: 1. Period 1 is the randomized, double-blind, placebo-controlled treatment period that will assess the efficacy, safety and tolerability of aficamten in pediatric participants. 2. Period 2 is the open-label extension trial that will assess the long-term safety of aficamten in pediatric participants, and further assess efficacy and tolerability. 3. Period 3 is the long-term extension trial that will assess the long-term safety of aficamten in pediatric participants, and further assess efficacy and tolerability.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 12 Years - 17 Years
- Who can join: All genders
Biomarkers mentioned
What the study is looking for
- ✓Period 1: Treatment Period
- ✓Males and females between 12 and \< 18 years of age at screening and at Day 1.
- ✓Body weight ≥ 35 kg
- ✓Diagnosed with oHCM based on the following at screening:
- ✓Left ventricular (LV) hypertrophy with nondilated LV chamber in the absence of other heart disease.
Who cannot take part
- ✗Period 1: Treatment Period
- ✗Any of the following criteria will exclude potential participants from the trial:
- ✗Significant valvular heart disease.
- ✗Moderate or severe valvular aortic stenosis or fixed subaortic obstruction.
- ✗Mitral regurgitation that is greater than mild in severity and not due to systolic anterior motion of the mitral...
See the full criteria
Where Is This Study? (3 UK sites)
Alder Hey Children's Hospital
Liverpool, United Kingdom
Evelina Children's Hospital
London SW3 6NP, United Kingdom
Great Ormond Street Hospital for Children
London WC1N 3BH, United Kingdom
Main Email: Research.Governance@gosh.nhs.uk
Research.Governance@gosh.nhs.uk0207 905 2700