Cardiac Assessment by PV Loop in IPAH and Scleroderma PAH

NCT04610788 | Completed

• 2 other identifiers: NA_00049022R01HL114910-06
• Study Type: observational
• Target: 43
• Locations: 1 country
• Sites: 1

Brief Summary

This observational study is being done to understand why people with scleroderma can develop pulmonary arterial hypertension (high blood pressure in the lungs, abbreviated PAH) and a weak heart muscle (heart failure). The study will also help the investigators understand why people with PAH from an unknown cause (called idiopathic PAH, or IPAH) can also develop a weakened heart muscle. The response of the right side of the heart or right ventricle (RV) to standard PAH therapy in scleroderma-associated PAH and in IPAH will be assessed. Blood and tissue samples will be collected from research participants during participants' normal standard of care procedures. People with scleroderma-associated PAH or idiopathic cause (IPAH) who need a right heart catheterization may join this study.

Conditions

Scleroderma; Pulmonary Artery Hypertension

Outcome Measures

Primary Outcomes (5)

• Right Ventricular Function as assessed by RHC

  Assessed on the clinical RHC as normal, moderately reduced, or severely reduced.

  Baseline

• Change in pulmonary vascular resistance

  Assessed as improved or decreased after 6 months by comparing the change in pulmonary vascular resistance in Wood units on the clinical RHC.

  Baseline and 6months

• Change in arterial elastance

  Assessed as improved or decreased after 6 months by comparing the change in arterial elastance in pressure volume (PV) loops.

  Baseline and 6 months

• Change in myofilament contractility

  Assessed as Normal or Abnormal after studying the collected samples in lab. Abnormal can be either reduced or increased; i.e. hyper- or hypo-contractile.

  up to 4 years

• Change in calcium sensitivity

  Assessed as either increased- or decreased- sensitivity after 6 months, by studying the collected samples in lab.

  up to 4 years

Secondary Outcomes (2)

• Number of genes expressed

  up to 4 years

• Number of proteins expressed

  up to 4 years

Study Arms (2)

SSc-PAH Group

Scleroderma patients referred for a clinically indicated right heart catheterization (RHC).

IPAH Group

Presumed/known IPAH patients referred for a clinically indicated right heart catheterization (RHC).

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients 18 years or older with clinically diagnosed scleroderma or presumed/known idiopathic pulmonary hypertension.

You may qualify if:

• Patients 18 years or older with clinically diagnosed scleroderma or presumed/known idiopathic pulmonary hypertension.

You may not qualify if:

• Patients found to have secondary pulmonary hypertension (PH due to left heart failure) on clinical RHC.
• Hemodynamically unstable patients (systolic blood pressure \< 90mmHg, vasopressor requirement).
• Patients whom are unable to give consent for themselves.
• Patients with RV clot or septal aneurysm will be excluded.
• In order to undergo the clinical right heart catheterization procedures, pregnancy testing (urine or serum) is standard of care.
• Pregnancy

Sponsors & Collaborators

• Johns Hopkins University: lead
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Sites (1)

Johns Hopkins

Baltimore, Maryland, 21287, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Cardiac biopsy, 4 teaspoons of blood

MeSH Terms

Conditions

Scleroderma, Diffuse; Familial Primary Pulmonary Hypertension

Condition Hierarchy (Ancestors)

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

Study Officials

• Paul Hassoun, MD

  Johns Hopkins University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 26, 2020
• First Posted: November 2, 2020
• Study Start: April 15, 2019
• Primary Completion: December 31, 2025
• Study Completion: December 31, 2025
• Last Updated: January 9, 2026

Record last verified: 2026-01

Locations

US (1)