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

NCT03726398 | Withdrawn Phase 2 FDA Drug

• 1 other identifier: IIS-02801
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Interstitial Lung Disease; Scleroderma; Pulmonary Hypertension

Outcome Measures

Primary Outcomes (1)

• Change in exercise pulmonary vascular resistance (PVR)

  Baseline to 6 months

Secondary Outcomes (3)

• Change in right ventricular pulmonary vascular hemodynamic coupling (RVPA).

  Baseline to 6 months

• Change in maximal oxygen consumption (V02 max).

  Baseline to 6 months

• Change in pulmonary impedance.

  Baseline to 6 months

Study Arms (1)

Opsumit

EXPERIMENTAL

Opsumit 10 mg tablet by mouth once daily

Drug: Opsumit 10 Mg Tablet

Interventions

Opsumit 10 Mg Tablet DRUG

Oral tablet taken once daily

Also known as: Macitentan

Opsumit

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Franz Rischard, DO: lead
• National Jewish Health: collaborator
• University of Pittsburgh: collaborator

Study Sites (1)

University of Arizona

Tucson, Arizona, 85724, United States

Location

MeSH Terms

Conditions

Lung Diseases, Interstitial; Scleroderma, Diffuse; Hypertension, Pulmonary

Interventions

macitentan; Tablets

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Scleroderma, Systemic; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases

Intervention Hierarchy (Ancestors)

Dosage Forms; Pharmaceutical Preparations

Study Officials

• Franz P. Rischard, DO

  University of Arizona

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Associate Professor, Medicine

Study Record Dates

• First Submitted: October 21, 2018
• First Posted: October 31, 2018
• Study Start: September 1, 2018
• Primary Completion: August 3, 2020
• Study Completion: August 3, 2020
• Last Updated: February 20, 2025

Record last verified: 2025-02

Locations

US (1)