Treatment of Preclinical Hypertrophic Cardiomyopathy With Diltiazem

This study has been completed.
Sponsor:
Collaborators:
Children's Hospital Boston
Information provided by (Responsible Party):
Carolyn Yung Ho, MD, Brigham and Women's Hospital
ClinicalTrials.gov Identifier:
NCT00319982
First received: April 27, 2006
Last updated: March 24, 2015
Last verified: March 2015
Results First Received: January 26, 2015  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Hypertrophic Cardiomyopathy
Interventions: Drug: Diltiazem
Drug: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Participants were recruited from the HCM clinics at Brigham and Women’s Hospital (Boston, MA), Boston Children’s Hospital (Boston, MA), and Royal Prince Alfred Hospital (Sydney, Australia). Participants were recruited from 2006 through 2010.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
I- Diltiazem (Active Arm) Diltiazem: Titrated to a target dose of 360 mg daily (sustained release formulation) for the duration of the study period
II- Placebo

Placebo Comparator

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


Participant Flow:   Overall Study
    I- Diltiazem (Active Arm)     II- Placebo  
STARTED     19     20  
COMPLETED     18     20  
NOT COMPLETED     1     0  
Withdrawal by Subject                 1                 0  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Participants enrolled through Brigham and Women’s Hospital (Boston, MA), Boston Children’s Hospital, and Royal Prince Alfred Hospital (Sydney, Australia) were randomized 1:1 to diltiazem or placebo. Participants were 5-39 years old, carried the sarcomere mutation presumed to cause HCM in the family, and had normal LV wall thickness by echo.

Reporting Groups
  Description
I- Diltiazem Diltiazem: Titrated to a target dose of 360 mg daily (sustained release formulation) for the duration of the study period
II- Placebo

Placebo Comparator

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

Total Total of all reporting groups

Baseline Measures
    I- Diltiazem     II- Placebo     Total  
Number of Participants  
[units: participants]
  18     20     38  
Age  
[units: years]
Mean (Standard Deviation)
  14.1  (1.7)     17.3  (2.1)     15.8  (8.6)  
Gender  
[units: participants]
     
Female     11     11     22  
Male     7     9     16  
Region of Enrollment  
[units: participants]
     
United States     18     19     37  
Australia     0     1     1  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   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 ]

2.  Secondary:   Safety and Tolerability of Diltiazem Treatment   [ Time Frame: Baseline through final study visits ]

3.  Secondary:   Impact of Diltiazem on Heart Rate   [ Time Frame: Baseline and final study visits ]

4.  Secondary:   Left Ventricular Cavity Size   [ Time Frame: Baseline and final study visits ]

5.  Secondary:   Development of Left Ventricular Hypertrophy   [ Time Frame: Baseline through final study visits ]

6.  Secondary:   Adherence to Study Medication   [ Time Frame: Duration of the trial ]

7.  Secondary:   Impact of Diltiazem on Systolic Blood Pressure   [ Time Frame: Baseline and final study visits ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Small number of participants and short follow-up duration. Currently available tools to monitor treatment response/ phenotypic progression are may lack adequate resolution. The penetrance of sarcomere mutations is variable and may not be complete.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Carolyn Ho, MD
Organization: Brigham and Women's Hospital
phone: 617-732-5685
e-mail: cho@partners.org


Publications:


Responsible Party: Carolyn Yung Ho, MD, Brigham and Women's Hospital
ClinicalTrials.gov Identifier: NCT00319982     History of Changes
Other Study ID Numbers: 001936, K23HL078901
Study First Received: April 27, 2006
Results First Received: January 26, 2015
Last Updated: March 24, 2015
Health Authority: United States: Food and Drug Administration