Ambrisentan for the Improvement in Right Ventricular Strain in Scleroderma Associated Pulmonary Arterial Hypertension (AMERICA-PAH)

National Jewish Health

Status: Terminated

Conditions: Pre-Pulmonary Atrial Hypertension

Treatments: Drug: Ambrisentan

Study type: Interventional

Funder types: Other

Identifiers

NCT02169752

HS-2716

Details and patient eligibility

About

This research study is looking at the use of the drug ambrisentan and if it can improve right ventricle function in people with systemic sclerosis-associated pre-pulmonary arterial hypertension. It is also looking at using right ventricle function changes as a marker of disease severity.

Full description

This research study is looking at the use of the drug ambrisentan and if it can improve right ventricle function in people with systemic sclerosis-associated pre-pulmonary arterial hypertension. It is also looking at using right ventricle function changes as a marker of disease severity.

Pre-pulmonary arterial hypertension is a borderline elevation in blood pressure in the lungs. Pre-pulmonary arterial hypertension is not currently treated with the drug therapies that are used for pulmonary arterial hypertension. It represents a group of patients that are at risk for developing pulmonary arterial hypertension.

The administration of ambrisentan is the experimental part of this study. Ambrisentan has not been approved by the FDA for use of improving right ventricle function in people with pre-pulmonary arterial hypertension. You will still receive your normal clinical care.

The purpose of this study is to learn more about ambrisentan and its role in treating systemic sclerosis-associated pre-pulmonary arterial hypertension. Ambrisentan is already approved for use in people with systemic sclerosis-associated pulmonary arterial hypertension, but this study wants to research its role in people with pre-pulmonary arterial hypertension.

Enrollment: 7 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age >18 years, < 80 years

• Systemic sclerosis with any of the following features:

  1. Duration of Raynaud's phenomena >8 years
  2. Anticentromere antibody positivity
  3. isolated nucleolar-pattern ANA positivity
  4. Extensive telangiectasias
  5. DLCO < 60% in the absence of extensive ILD
  6. FVC%/DLCO% >1.6
  7. Unexplained dyspnea

• Right heart catheterization-proven pre PAH (mean PAP 20-25 mmHg and pulmonary capillary wedge pressure <15 mmHg)

• Systolic blood pressure >100 mmHg

• Reliable contraception for women of childbearing age

• Informed consent

Exclusion criteria

• < 18 years or > 80 years
• Left ventricular ejection fraction < 55%
• Systolic or diastolic left ventricular congestive heart failure
• Liver disease (abnormal AST/ALT, chronic hepatitis, or cirrhosis)
• Extensive ILD or FVC< 60%
• Pregnant
• Breast-feeding women
• Cyclosporine use

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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7 participants in 2 patient groups, including a placebo group

Ambrisentan

Active Comparator group

Description:

Subjects will be randomly assigned in a 1:1 ration according to the computer generated random numbers to receive either placebo (sugar pill) or ambrisentan.

Treatment:

Drug: Ambrisentan

Placebo

Placebo Comparator group

Description:

Subjects will be randomly assigned in a 1:1 ration according to the computer generated random numbers to receive either placebo (sugar pill) or ambrisentan.

Treatment:

Drug: Ambrisentan