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New treatment option for Pediatric
Official title A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).
ClinicalTrials.gov ID: NCT06412666
What this study is testing
What is Aficamten?
Aficamten is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for pediatric.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The purpose of this study is to evaluate the efficacy, safety and PK of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 to 17
You may be able to join if
- Period 1: Treatment Period
- Males and females between 12 and \< 18 years of age at screening and at Day 1.
- Body weight ≥ 45 kg for the initial cohort and then body weight ≥ 35 kg after at least 10 participants in the initial cohort have undergone dose...
- Core laboratory confirmation of the following oHCM echocardiographic criteria at screening:
- Left ventricular (LV) hypertrophy with nondilated LV chamber in the absence of other cardiac disease.
You likely can't join if
- 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 valve (per judgment of Principal...
- Evidence of fixed left-sided obstruction (eg, subaortic membrane, aortic valve stenosis, or coarctation of the aorta).
- History of LV systolic dysfunction (LVEF \< 45%) or stress cardiomyopathy at any time during their clinical course.
See the full eligibility criteria
- Period 1: Treatment Period
- Males and females between 12 and \< 18 years of age at screening and at Day 1.
- Body weight ≥ 45 kg for the initial cohort and then body weight ≥ 35 kg after at least 10 participants in the initial cohort have undergone dose titration up to Week 4 without observed events of LVEF \< 50% at the...
- Core laboratory confirmation of the following oHCM echocardiographic criteria at screening:
- Left ventricular (LV) hypertrophy with nondilated LV chamber in the absence of other cardiac disease.
- LV end-diastolic wall thickness that meets a threshold of:
- Z-score \> 2.5 in the absence of family history OR
- Z-score \> 2 in the presence of positive family history or positive genetic test.
- LVEF ≥ 60% AND Valsalva LVOT-G ≥ 50 mmHg.
- oHCM of sarcomeric origin confirmed by genetic testing or, if unable to confirm by genetic testing, oHCM of sarcomeric origin may be presumed in the absence of history of metabolic disorders, mitochondrial...
- New York Heart Association (NYHA) Class ≥ II at screening.
- Adequate acoustic windows for echocardiography.
- Participants on beta blockers, verapamil, diltiazem, or disopyramide should have been on stable doses for more than 4 weeks prior to randomization.
- Period 2: Open-Label Extension
- Completed Period 1. If unable to complete Period 1 due to circumstances not related to compliance or safety, the Medical Monitor may review and determine eligibility.
- LVEF ≥ 55% after washout.
- Period 3: Long-term Extension Completed Period 2.
- 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 valve (per judgment of Principal Investigator or designee).
- Evidence of fixed left-sided obstruction (eg, subaortic membrane, aortic valve stenosis, or coarctation of the aorta).
- History of LV systolic dysfunction (LVEF \< 45%) or stress cardiomyopathy at any time during their clinical course.
- History of congenital heart disease other than oHCM (may be enrolled if not hemodynamically significant in the judgement of the Principal Investigator and study Medical Monitor).
- Has been treated with SRT (surgical myectomy or percutaneous alcohol septal ablation) within the preceding 6 months or has plans for either treatment during the trial period.
- History of paroxysmal or persistent atrial fibrillation or atrial flutter.
- History of syncope, symptomatic ventricular arrhythmia, or sustained ventricular tachyarrhythmia within 3 months prior to screening.
- History or evidence of any other clinically significant disorder, malignancy, active infection, other condition, or disease that, in the opinion of the Principal Investigator (or designee) or the Medical Monitor, would...
- Current or previous use of drugs known to cause cardiomyopathy (eg, anthracyclines, monoclonal antibodies [trastuzumab], alkylating agents [cyclophosphamide], and tyrosine kinase inhibitors [sunitinib and imatinib]).
- Currently participating in another investigational device or drug trial or received an investigational device or drug \< 1 month (or 5 half-lives for drugs, whichever is longer) prior to screening.
- Implantable cardioverter defibrillator (ICD) implantation within 6 weeks of screening or planned ICD implantation during the trial period.
- Has received prior treatment with aficamten or mavacamten.
- Currently listed for heart transplantation or anticipated to be listed for heart transplantation in the next 12 months.
The study team makes the final eligibility decision.
Where it's taking place
- Phoenix, Arizona, United States
- Los Angeles, California, United States
- San Francisco, California, United States
- Aurora, Colorado, United States
- Washington D.C., District of Columbia, United States
- Miami, Florida, United States
- Chicago, Illinois, United States
- Iowa City, Iowa, United States
- New Orleans, Louisiana, United States
- Ann Arbor, Michigan, United States
- Detroit, Michigan, United States
- Rochester, Minnesota, United States
- Kansas City, Missouri, United States
- Omaha, Nebraska, United States
- Morristown, New Jersey, United States
- New York, New York, United States
- The Bronx, New York, United States
- Durham, North Carolina, United States
- Portland, Oregon, United States
- Philadelphia, Pennsylvania, United States
+ 16 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Phoenix, Arizona, United States; Los Angeles, California, United States; San Francisco, California, United States; Aurora, Colorado, United States; Washington D.C., District of Columbia, United States; Miami, Florida, United States and 30 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.