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Phase 3 N=39 Randomized Quadruple-blind Treatment

Treatment of Preclinical Hypertrophic Cardiomyopathy With Diltiazem

Hypertrophic Cardiomyopathy

Enrolled (actual)
39
Serious AEs
0.0%
Results posted
Apr 2015
Primary outcome: Primary: Increase, Stability of, or Decrease in the Decline of Diastolic Function as Reflected by the Global Early Myocardial Relaxation (E') Velocity — -0.06; -0.21 cm/sec (difference final-baseline) — p=0.75

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
Diltiazem (Drug); Placebo (Drug)
Age
Pediatric, Adult · 5+ yrs
Sex
All
Sponsor
Brigham and Women's Hospital
Primary completion
Dec 2012

Outcome Measures

OutcomeResultp-value
PRIMARY
Increase, Stability of, or Decrease in the Decline of Diastolic Function as Reflected by the Global Early Myocardial Relaxation (E') Velocity
-0.06; -0.21 0.75
SECONDARY
Safety and Tolerability of Diltiazem Treatment
10; 12 0.99
SECONDARY
Impact of Diltiazem on Heart Rate
-4.9; 2.0 >0.06
SECONDARY
Left Ventricular Cavity Size
0.60; -0.53 <0.001 sig
SECONDARY
Development of Left Ventricular Hypertrophy
2; 2
SECONDARY
Adherence to Study Medication
83; 90 0.08
SECONDARY
Impact of Diltiazem on Systolic Blood Pressure
-1.4; 2.1 0.15

Summary

This is a pilot clinical trial to assess whether the administration of diltiazem may be able to decrease the development or progression of hypertrophic cardiomyopathy (HCM). Diltiazem is a commonly used medication for the treatment of high blood pressure and studies on animals with HCM suggest that diltiazem decreases disease development. This study specifically targets individuals in the "prehypertrophic" phase of HCM-- those with documented sarcomere gene mutations without echocardiographic or EKG evidence of LVH, and therefore without a clinical diagnosis of HCM. The hypothesis of this study is that starting diltiazem administration early in life (in the prehypertrophic phase) will decrease the progression of HCM in individuals with sarcomere gene mutations. This will be assessed by looking at an improvement in the heart's ability to relax using echocardiography, as well as exploratory analyses of a broad range of features reflecting the heart's structure and function.

Eligibility Criteria

Inclusion Criteria

  • Preclinical HCM (identified sarcomere mutation with no clinical evidence of left ventricular hypertrophy)
  • Able to provide informed consent (or parental consent)

Exclusion Criteria

  • Contraindication to diltiazem administration
  • Impaired hepatic or renal function
  • Age < 5 years
  • Pregnant or breastfeeding women
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00319982). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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