Hypertrophic Regression With N-Acetylcysteine in HCM

• Register: ClinicalTrials.gov
• Last refreshed on: 15 November 2021
• Main ID: NCT01537926
• Date of registration: 15/02/2012
• Prospective Registration: No
• Primary sponsor: The University of Texas Health Science Center, Houston
• Public title: Hypertrophic Regression With N-Acetylcysteine in HCM HALT
• Scientific title: Pilot Feasibility Study With N-acetylcystein (NAC) in Patients With HCM Caused by Sarcomere Proteins Mutations
• Date of first enrolment: January 2012
• Target sample size: 42
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01537926
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Ali J. Marian, MD
• Affiliation: The University of Texas Health Science Center, Houston

Key inclusion & exclusion 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)



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Hypertrophic Cardiomyopathy

Intervention(s)

Drug: Placebo

Drug: N-acetylcysteine

Primary Outcome(s)

Recruitment as Assessed by Number of Participants Who Enrolled to the Study [Time Frame: at the time of enrollment]

Compliance as Assessed by Percentage of Pills Taken by Participant [Time Frame: from baseline to 12 months]

Retention as Assessed by Number of Participants Who Completed the Study [Time Frame: from baseline to 12 months]

Interventricular Septal Thickness (IVST) as Assessed by Echocardiography [Time Frame: baseline]

Interventricular Septal Thickness (IVST) as Assessed by Echocardiography [Time Frame: 12 months]

Number of Participants With Side Effects Attributable to the Intervention [Time Frame: from baseline to 12 months]

Secondary Outcome(s)

Left Ventricular End-systolic Diameter (LVESD) as Assessed by Echocardiography [Time Frame: baseline]

Left Ventricular Mass (LVM) as Assessed by Echocardiography [Time Frame: 12 months]

Left Ventricular Mass (LVM) as Assessed by Echocardiography [Time Frame: baseline]

Left Ventricular End-systolic Diameter (LVESD) as Assessed by Echocardiography [Time Frame: 12 months]

Secondary ID(s)

IR34HL105563

HALT-HCM

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Institutes of Health (NIH)

Results

• Results available: Yes
• Date Posted: 02/11/2021
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01537926