Hypertrophic Regression With N-Acetylcysteine in HCM
Condition: Hypertrophic Cardiomyopathy
MeSH terms: Cardiomyopathies; Cardiomyopathy, Hypertrophic; Hypertrophy
Intervention: N-acetylcysteine; Placebo
Intervention description: NAC 600mg capsule or matching Placebo , 1 twice daily for 90 days, then increase to NAC 1,200mg or matching placebo, 2 capsules twice daily for 270 days.; sugar pill manufactured to minic NAC 600mg capsule
Intervention MeSH terms: Acetylcysteine; N-monoacetylcystine
Phase: Phase 1
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 42.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: HALT; HCM

Summary:

      The purpose of the sudy is to conduct a small study to gather the preliminary data for future
      lage scale clinical studies that will be designed test the potential beneficial effect of
      over-the counter study anti-oxidant drug called N-acetylcysteine (NAC) in patients with a
      heart muscle condition called Hypertrophic Cardiomyopathy (HCM). The present study is a pilot
      feasibility study, the investigators want to find out whether the investigators can recruit
      and retain patients with HCM in the study and whether these patients can tolerate this drug
      and can stay on one year. Likewise, the investigators want to find out any potential side
      effects that this drug might have and estimate whether it has any beneficial effects.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patients with primary cardiac hypertrophy, non-dilated LV cavity and preserved LV
             systolic function, hence, the diagnosis of HCM, who have at least an LV end diastolic
             (LVSD) wall thickness of at least 15 mm on a 2D echocardiogram and

          -  Known to have mutations in genes encoding sarcomeric proteins

        Exclusion Criteria:

          -  Hypersensitivity to NAC

          -  Individuals younger than 18 years old (in the pilot study)

          -  Phenocopy conditions, diagnosed clinically or genetically

          -  Patients who have undergone transcatheter (alcohol) septal ablation within 6 months.

          -  Individuals (typically family members) with causal mutations but an LVSD wall
             thickness of <15 mm

          -  Patients with concomitant diseases such as:

               -  Significant coronary artery disease >70% luminal diameter stenosis in ny of the
                  major coronary arteries (if known);

               -  Valvular heart diseases (more than mild aortic stenosis and mitral regurgitation,
                  the latter judged to be due to primary mitral valve abnormalities);

               -  Uncontrolled hypertension, defined as systolic blood pressure of

                    -  140 mmHg and diastolic blood pressure of ≥90 mmHg on medication, mean of
                       three measurements at rest);

               -  Other significant medical problems, such as moderate to severe chronic renal
                  failure (GFR<45 ml/min/1.73m2), advanced liver disease, cancer, or other
                  disabling conditions

          -  Pregnant women, nursing mothers and those who plan pregnancy during the study period

          -  Those with active asthma (albeit the concern is relevant to nebulizer form but not
             oral formulations)