CompRehensive Phenotypic Characterization of Patients With Scleroderma-Associated ILD and PH

Study Purpose

Patients with interstitial lung disease (ILD) and scleroderma who develop pulmonary hypertension (PH) do not fit well into the current classification system and treatments for pulmonary hypertension. This study aims to better understand patients with ILD-PH and scleroderma and to determine if treatment with Macitentan is beneficial.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 18 Years and Over
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - Patients who have scleroderma ILD will be defined as having a total lung capacity of less than 80% predicted and CT evidence of fibrosis.
The degree of fibrosis will be scored by a radiologist using the CT comparative scoring method of Wells et al (13).
  • - Pulmonary Hypertension (PH) as defined as resting mean pulmonary arterial pressure (mPAP) ≥ 25 mmHg with a wedge pressure of ≤ 15 mmHg during right heart catheterization.
  • - Stable ILD as evident by a stable FEV1 and FVC for 3 months prior to the initiation of the study, and be pulmonary arterial hypertension (PAH)-targeted treatment naïve.

Exclusion Criteria:

  • - Patients with a left ventricular ejection fraction <50% or clinical, echocardiographic, and/or catheterization data consistent with heart failure with preserved ejection fraction (HFpEF) and/or moderate-severe aortic or mitral valve abnormality - Patients with severe restrictive lung disease (FVC<40% predicted) and/or obstructive lung disease (FEV1 <55% predicted and FEV1/FVC <70%).
  • - Patients with radiographic combined pulmonary fibrosis/emphysema (CPFE) will also be excluded if imaging shows predominant emphysema and/or obstruction is moderately severe (FEV1<30%) - Patients with a history of pulmonary embolism within the last three months or evidence of chronic pulmonary embolism.
  • - Patients with a known contraindication to right heart catheterization.
  • - Patients whom have received active or previous pulmonary vasoactive medication within the previous 12 weeks.
  • - Patients with a contraindication to exercise testing based on American Heart Association/American College of Cardiology (AHA/ACC) guidelines.
  • - PAH associated with significant venous or capillary involvement (PCWP > 15 mmHg), known pulmonary veno-occlusive disease, and pulmonary capillary hemangiomatosis.
  • - Persistent pulmonary hypertension of the newborn.
  • - Pulmonary Hypertension belonging to groups 2 to 5 of the Venice classification.
  • - Moderate to severe hepatic impairment, i.e., Child-Pugh Class B or C.
  • - Estimated creatinine clearance < 30 mL/min - Serum aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 1.5 times the upper limit of normal.
  • - Hemoglobin < 75% of the lower limit of the normal range.
  • - Systolic blood pressure < 100 mmHg.
  • - Acute or chronic physical impairment (other than dyspnea), limiting the ability to comply with study requirements.
  • - Pregnant or breast-feeding.
  • - Known concomitant life-threatening disease with a life expectancy < 12 months.
  • - Body weight < 40 kg.
  • - Any condition that prevents compliance with the protocol or adherence to therapy.
  • - Treatment with endothelin receptor antagonists (ERAs) within 3 months prior to randomization.
  • - Systemic treatment within 4 week prior to randomization with cyclosporine A or tacrolimus, everolimus, sirolimus (calcineurin or mammalian target of rapamycin (mTOR) inhibitors).
  • - Treatment with cytochrome P3A (CYP3A) inducers within 4 weeks prior to randomization - Known hypersensitivity to drugs of the same class as the study drug, or any of their excipients.
- Planned treatment, or treatment, with another investigational drug within 1 month prior to randomization

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT03726398
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 2/Phase 3
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Franz Rischard, DO
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Franz P. Rischard, DO
Principal Investigator Affiliation University of Arizona
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

OtherOtherOther
Overall Status Recruiting
Countries United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Interstitial Lung Disease, Scleroderma, Pulmonary Hypertension
Additional Details

The investigators aim to use pressure-volume loop derived right ventriculo-vascular coupling, pulmonary impedance, and invasive cardiopulmonary exercise testing (CPET) to: 1. Comprehensively phenotype patients with scleroderma ILD-PH and pulmonary vascular exercise limitation (PVL) relative to scleroderma ILD-PH without PVL. 2. Compare the efficacy of chronic Macitentan therapy in improving 1) right ventricular hemodynamics 2) exercise capacity and 3) symptoms in scleroderma ILD-PH patients with and without PVL.

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

University of Arizona, Tucson, Arizona

Status

Recruiting

Address

University of Arizona

Tucson, Arizona, 85724

Site Contact

Valerie Bloss, MS

vbloss@email.arizona.edu

520-626-8000

Terms of Service

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