Phase 1 N=42 Randomized Quadruple-blind Treatment

Hypertrophic Regression With N-Acetylcysteine in HCM

Hypertrophic Cardiomyopathy

Bottom Line

View on ClinicalTrials.gov: NCT01537926 ↗

Enrolled (actual)

Serious AEs

11.9%

Results posted

Nov 2021

Primary outcome: Primary: Recruitment as Assessed by Number of Participants Who Enrolled to the Study — 29; 13 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 1
Interventions: N-acetylcysteine (Drug); Placebo (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: The University of Texas Health Science Center, Houston
Primary completion: Dec 2016

Outcome Measures

Outcome	Result	p-value
PRIMARY Recruitment as Assessed by Number of Participants Who Enrolled to the Study	29; 13	—
PRIMARY Retention as Assessed by Number of Participants Who Completed the Study	24; 11	—
PRIMARY Compliance as Assessed by Percentage of Pills Taken by Participant	92	—
PRIMARY Number of Participants With Side Effects Attributable to the Intervention	0; 0	—
PRIMARY Interventricular Septal Thickness (IVST) as Assessed by Echocardiography	17.92; 17.82	—
PRIMARY Interventricular Septal Thickness (IVST) as Assessed by Echocardiography	17.92; 17.82	—
SECONDARY Left Ventricular End-systolic Diameter (LVESD) as Assessed by Echocardiography	22.13; 22.64	—
SECONDARY Left Ventricular End-systolic Diameter (LVESD) as Assessed by Echocardiography	22.13; 22.64	—
SECONDARY Left Ventricular Mass (LVM) as Assessed by Echocardiography	281.98; 290.44	—
SECONDARY Left Ventricular Mass (LVM) as Assessed by Echocardiography	281.98; 290.44	—

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 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)

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01537926). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.