Overview

Treatment of Preclinical Hypertrophic Cardiomyopathy With Diltiazem

Status:
Completed

Trial end date:
2013-12-01

Target enrollment:
0

Participant gender:
All

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.

Phase:

Phase 2/Phase 3

Accepts Healthy Volunteers?

Details

Lead Sponsor:

Brigham and Women's Hospital

Collaborators:

Boston Children's Hospital
Boston Children’s Hospital
National Heart, Lung, and Blood Institute (NHLBI)

Treatments:

Diltiazem

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