Working… Menu

Treatment of Preclinical Hypertrophic Cardiomyopathy With Diltiazem

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00319982
Recruitment Status : Completed
First Posted : April 27, 2006
Results First Posted : April 7, 2015
Last Update Posted : April 7, 2015
National Heart, Lung, and Blood Institute (NHLBI)
Boston Children’s Hospital
Information provided by (Responsible Party):
Carolyn Yung Ho, MD, Brigham and Women's Hospital

Brief 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.

Condition or disease Intervention/treatment Phase
Hypertrophic Cardiomyopathy Drug: Diltiazem Drug: Placebo Phase 2 Phase 3

  Show Detailed Description

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 39 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Treatment of Preclinical Hypertrophic Cardiomyopathy With Diltiazem
Study Start Date : January 2006
Actual Primary Completion Date : December 2012
Actual Study Completion Date : December 2013

Arm Intervention/treatment
Experimental: I- Diltiazem
Diltiazem- study medication
Drug: Diltiazem
Sustained release formulation titrated to a target dose of 360 mg daily, or a maximum of 5 mg/kg/day in pediatric subjects for the duration of the study period

Placebo Comparator: II- Placebo
Placebo Comparator
Drug: Placebo
Placebo comparator (double-blind allocation of study medication)

Primary Outcome Measures :
  1. Increase, Stability of, or Decrease in the Decline of Diastolic Function as Reflected by the Global Early Myocardial Relaxation (E') Velocity [ Time Frame: Baseline and final study visits ]
    The change in E' velocity (difference between final value - baseline value) was compared between participants who received diltiazem and those who received placebo to gauge treatment response. Please note that the total duration on treatment varied between study subjects to maximize time on treatment for the trial. Specifically, subjects that enrolled earliest had the longest duration of treatment; those who enrolled latest had the shortest duration of treatment with a minimum treatment duration of 1 year. All analyses examine the final study visit on treatment to the baseline visit.

Secondary Outcome Measures :
  1. Safety and Tolerability of Diltiazem Treatment [ Time Frame: Baseline through final study visits ]
    Adverse events were compared between participants assigned to diltiazem and those assigned to placebo

  2. Impact of Diltiazem on Heart Rate [ Time Frame: Baseline and final study visits ]
    Change in Value (Difference between Final and Baseline Visits)

  3. Left Ventricular Cavity Size [ Time Frame: Baseline and final study visits ]
    Change in Left Ventricular End-Diastolic Diameter z-score (Final Value - Baseline Value)

  4. Development of Left Ventricular Hypertrophy [ Time Frame: Baseline through final study visits ]
    The number of participants who developed overt left ventricular hypertrophy during the duration of the trial was analyzed

  5. Adherence to Study Medication [ Time Frame: Duration of the trial ]
    Adherence to study medication was assessed by pill count

  6. Impact of Diltiazem on Systolic Blood Pressure [ Time Frame: Baseline and final study visits ]
    Change in Value (Difference between Final and Baseline Visits)

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   5 Years to 39 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00319982

Layout table for location information
United States, Massachusetts
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
Children's Hospital
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
Brigham and Women's Hospital
National Heart, Lung, and Blood Institute (NHLBI)
Boston Children’s Hospital
Layout table for investigator information
Principal Investigator: Carolyn Y Ho, MD Brigham and Women's Hospital


Publications automatically indexed to this study by Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Carolyn Yung Ho, MD, Associate Physician, Brigham and Women's Hospital Identifier: NCT00319982     History of Changes
Other Study ID Numbers: 001936
K23HL078901 ( U.S. NIH Grant/Contract )
First Posted: April 27, 2006    Key Record Dates
Results First Posted: April 7, 2015
Last Update Posted: April 7, 2015
Last Verified: March 2015
Keywords provided by Carolyn Yung Ho, MD, Brigham and Women's Hospital:
Hypertrophic Cardiomyopathy
Left ventricular hypertrophy
Additional relevant MeSH terms:
Layout table for MeSH terms
Cardiomyopathy, Hypertrophic
Heart Diseases
Cardiovascular Diseases
Pathological Conditions, Anatomical
Aortic Stenosis, Subvalvular
Aortic Valve Stenosis
Heart Valve Diseases
Antihypertensive Agents
Calcium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Calcium-Regulating Hormones and Agents
Physiological Effects of Drugs
Vasodilator Agents