Hypertrophic Regression With N-Acetylcysteine in HCM

• Status: Completed
• Trial end date: 2016-12-31
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: The University of Texas Health Science Center, Houston
• Collaborator: National Institutes of Health (NIH)
• Treatments: Acetylcysteine; N-monoacetylcystine

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)