Valsartan for Attenuating Disease Evolution In Early Sarcomeric HCM

Completed · Phase 2 · Has a placebo group

Conditions studied: Hypertrophic Cardiomyopathy

In brief

The purpose of this trial is to determine whether treatment with valsartan will have beneficial effect in early hypertrophic cardiomyopathy (HCM) by assessing many domains that reflect myocardial structure, function and biochemistry.

Key facts

Study ID

NCT01912534

Run by

Carelon Research

People needed

211

Starts

2014-03-01

Expected to finish

2019-07-01

Last updated by the study team

2021-01-11

Who can join

Age: 8 and older, up to 45. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• All subjects must have a Pathogenic or Likely Pathogenic HCM Sarcomere Mutation
• a. The following categories of mutations are considered acceptable for subjects who have previously undergone clinical genetic testing. If results are ambiguous, they will be reviewed by the Clinical Coordinating Center to determine eligibility.
• Laboratory for Molecular Medicine (Pathogenic, Likely Pathogenic)
• Transgenomics/ PGXHealth (Class I)
• GeneDx (Disease causing; Variant; likely disease-causing; Published, disease-causing mutation; Novel, likely disease-causing, mutation)
• Correlagen (Associated; Probably Associated)
• Group 1 (Overt HCM Cohort)
• LV wall thickness ≥12 mm and ≤25 mm or z score ≥3 and ≤18 as determined by rapid assessment by the echocardiographic core laboratory
• NYHA functional class I or II; no perceived or only slight limitations in physical activities
• No resting or provokable LV obstruction (peak gradient ≤ 30 mmHg) on clinically-obtained Exercise Tolerance Test (ETT)-echo within the past 24 months or transthoracic echo with Valsalva maneuver within the past 12 months
• Age 8-45 years
• Able to attend follow-up appointments, complete all study assessments, and provide written informed consent
• Group 2 (Preclinical HCM Cohort (G+/LVH-))
• LV Wall Thickness <12 mm and z score <3 , as determined by rapid assessment by the echocardiographic core laboratory
• Age 10-25 years
• E' z score ≤ -1.5 OR ECG abnormalities other than NSSTW changes (Q waves, T wave inversion, repolarization changes) OR LV wall thickness z-score 1.5-2.9 combined with LV thickness to dimension ratio ≥0.19 (as determined by rapid assessment by the echocardiographic core laboratory)
• Able to attend follow-up appointments, complete all study assessments, and provide written informed consent
• Subject Exclusion Criteria
• Contraindication to angiotensin receptor blocker (ARB) administration, including impaired renal function, hyperkalemia (serum K>5.0 mmol/L), prior history of angioedema
• Medical conditions associated with increased collagen turnover that may confound interpretation of biomarkers of collagen synthesis (liver, pulmonary or renal fibrosis, inflammatory states, cancer, trauma or surgery within 6 months of enrollment)
• Concomitant use of Spironolactone, Lithium, or Aliskiren, ARB or ACE-inhibitors. If these drugs are in active use but not necessary for medical care, they may be discontinued and baseline studies can be performed after a 2-week washout period.
• Pregnant or breastfeeding females - Females of childbearing potential with no effective contraceptive method (including abstinence)
• Uncontrolled systemic HTN [persistent SBP>160 and/or DBP>90 in adult or equivalent in children (e.g., SBP>99th or DBP>95th percentile for sex, age, and height centile based on the American Academy of Pediatrics normal values)]
• Obstructive physiology, defined by resting, Valsalva-provoked or exercise-induced gradient >30mmHg within the past 24 months
• Prior septal myectomy or alcohol septal ablation

Where it is running

• Stanford University — Stanford, California, United States
• University of Colorado — Aurora, Colorado, United States
• University of Chicago — Chicago, Illinois, United States
• Johns Hopkins University — Baltimore, Maryland, United States
• Brigham & Women's Hospital — Boston, Massachusetts, United States
• Children's Hospital Boston — Boston, Massachusetts, United States
• University of Michigan — Ann Arbor, Michigan, United States
• Washington University School Medicine — St Louis, Missouri, United States
• Cinncinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Cleveland Clinic Foundation — Cleveland, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• University of Pennsylvania — Philadelphia, Pennsylvania, United States
• Vanderbilt University — Nashville, Tennessee, United States
• Toronto General Hospital — Toronto, Ontario, Canada
• Toronto Sick Kids — Toronto, Ontario, Canada

Full record on ClinicalTrials.gov

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