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Intravenous Iloprost in Subjects With Symptomatic Raynaud's Phenomenon Secondary to Systemic Sclerosis (Phase 3)

Phase 3
Completed
Conditions
Raynaud's Phenomenon Secondary to Systemic Sclerosis
Interventions
Registration Number
NCT04040322
Lead Sponsor
Eicos Sciences, Inc.
Brief Summary

This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of iloprost on the frequency of and relief from symptomatic digital ischemic episodes in subjects with systemic sclerosis.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
198
Inclusion Criteria
  • Male or female subjects must be greater than or equal to 18 years of age.
  • Subjects must have a diagnosis of Systemic Sclerosis as defined by the 2013 American College of Rheumatology criteria/EULAR criteria
  • Subjects must have a diagnosis or history of Raynaud's Phenomenon, self-reported or reported by a physician, with at least a 2-phase color change in finger(s) of pallor, cyanosis, and/or reactive hyperemia in response to cold exposure or emotion
  • Subjects must have a minimum of 10 symptomatic Raynaud's Phenomenon attacks, documented in the electronic patient-reported outcomes (ePRO) diary, occurring over at least 3 separate days of the 3- to 5-day eligibility period
  • Subjects must complete a minimum of 80% of the daily ePRO diary entry during the baseline period
  • Female subjects of childbearing potential and male subjects must agree to use contraception for the duration of the study.
  • Subjects must be willing and able to comply with the study requirements and give informed consent for participation in the study
Exclusion Criteria
  • Female subjects who are pregnant or breastfeeding
  • Subjects with systolic blood pressure <85 mmHg
  • Subjects with an estimated glomerular filtration rate <15 mL/min/1.73 m2
  • Subjects with an alanine aminotransferase and/or aspartate aminotransferase value >3 × the upper limit of normal at screening
  • Subjects who have a digital ulcer infection within 30 days of screening
  • Subjects with a history of cervical or digital sympathectomy, or botulism toxin injections in their hands [for RP or digital ulcers] within 90 days of screening. Subjects should not have a planned botulism toxin or sympathectomy during their participation in the study.
  • Subjects with gangrene or digital amputation within 6 months of screening
  • Subjects with current intractable diarrhea or vomiting
  • Subjects with a risk of clinically significant bleeding events, including those with coagulation or platelet disorders at screening
  • Subjects with a history of major trauma or hemorrhage within 30 days of screening.
  • Subjects with clinically significant chronic intermittent bleeding, such as active gastric antral vascular ectasia or active peptic ulcer disease, within 60 days of screening
  • Subjects who have had any cerebrovascular events (eg, transient ischemic attack or stroke) within 6 months of screening
  • Subjects with a history of myocardial infarction or unstable angina within 6 months of screening. Subjects should not have a planned coronary procedure during their participation in the study
  • Subjects with acute or chronic congestive heart failure (New York Heart Association Class III [moderate] or Class IV [severe]) at screening
  • Subjects with a history of more than mild restrictive or congestive cardiomyopathy uncontrolled by medication or implanted device
  • Subjects with a history of life-threatening cardiac arrhythmias
  • Subjects with a history of hemodynamically significant aortic or mitral valve disease
  • Subjects with a history of known pulmonary hypertension, pulmonary arterial hypertension, or pulmonary veno-occlusive disease
  • Subjects with a history of significant restrictive lung disease, defined as forced vital capacity <45% predicted and diffusing capacity of the lungs for carbon monoxide <40% predicted (uncorrected for hemoglobin)
  • Subjects with scleroderma renal crisis within 6 months of screening
  • Subjects with a concomitant life-threatening disease with a life expectancy <12 months
  • Subjects who have a clinically significant disorder that, in the opinion of the Investigator, could contraindicate the administration of study drug, affect compliance, interfere with study evaluations, or confound the interpretation of study results
  • Subjects who have taken or are currently taking any parenteral, inhaled, or oral prostacyclin or prostacyclin receptor agonists (eg, epoprostenol, treprostinil, iloprost, and selexipag) within 8 weeks of screening
  • Subjects who have initiated or had a dose change of any of the following within 2 weeks of screening: oral, topical, or intravenous (IV) vasodilators (eg, calcium channel blockers, phosphodiesterase-5 (PDE5) inhibitors [eg, sildenafil, tadalafil, or vardenafil], nitrates, and fluoxetine)
  • Subjects with any history of acetaminophen intolerability (eg, allergic reaction to acetaminophen)
  • Subjects with any malignancy that requires treatment during the study period, that has required treatment within 1 year of screening (including excision of skin cancer) or that is currently not in remission
  • Subjects who have used any investigational medication or device for any indication within 30 days or 5 half-lives (whichever is longer)
  • Subjects who have participated in ES-201 or ES-301 studies and were randomized and treated with study drug

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Iloprost Injection, for intravenous useIloprost Injection, for intravenous useSubjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line. Study drug will be initiated at a starting dose 0.5 ng/kg/min up to 2.0 ng/kg/min.
PlaceboPlacebo IV infusionSubjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line. Study drug will be initiated at a starting dose 0.5 ng/kg/min up to 2.0 ng/kg/min.
Primary Outcome Measures
NameTimeMethod
Frequency of symptomatic RP attacksDay 6 - Day 21 will be compared to baseline

The primary efficacy parameter is the change in the weekly frequency of symptomatic RP attacks from baseline

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (30)

University of Pittsburgh Medical Center

🇺🇸

Pittsburgh, Pennsylvania, United States

University of California, Los Angeles Medical Center

🇺🇸

Los Angeles, California, United States

Arizona Arthritis & Rheumatology Research, PLLC

🇺🇸

Phoenix, Arizona, United States

University of Arizona - Arthritis Research Center

🇺🇸

Tucson, Arizona, United States

Cedars-Sinai Medical Center

🇺🇸

Los Angeles, California, United States

Stanford University Medical Center

🇺🇸

Palo Alto, California, United States

University of California San Francisco

🇺🇸

San Francisco, California, United States

Northwestern Medical Faculty Foundation

🇺🇸

Chicago, Illinois, United States

Georgetown University Medical Center - Department of Rheumatology

🇺🇸

Washington, District of Columbia, United States

University Medical Center New Orleans

🇺🇸

New Orleans, Louisiana, United States

Tufts Medical Center

🇺🇸

Boston, Massachusetts, United States

University of Michigan

🇺🇸

Ann Arbor, Michigan, United States

University of Nebraska Medical Center

🇺🇸

Omaha, Nebraska, United States

West Michigan Rheumatology PLLC

🇺🇸

Grand Rapids, Michigan, United States

University of Minnesota Maple Grove

🇺🇸

Minneapolis, Minnesota, United States

Robert Wood Johnson Medical School

🇺🇸

New Brunswick, New Jersey, United States

Columbia University Medical Center

🇺🇸

New York, New York, United States

Hospital for Special Surgery

🇺🇸

New York, New York, United States

University of Cincinnati - Scleroderma Center

🇺🇸

Cincinnati, Ohio, United States

Cleveland Clinic

🇺🇸

Cleveland, Ohio, United States

Ohio State University

🇺🇸

Columbus, Ohio, United States

The University of Toledo Medical Center (UTMC) - Ruppert Health Center

🇺🇸

Toledo, Ohio, United States

University of Pennsylvania

🇺🇸

Philadelphia, Pennsylvania, United States

Medical University of South Carolina (MUSC)

🇺🇸

Charleston, South Carolina, United States

University of Texas Houston - Division of Rheumatology and Clinical Immunogenetics

🇺🇸

Houston, Texas, United States

Froedtert Hospital and the Medical College of Wisconsin

🇺🇸

Milwaukee, Wisconsin, United States

University of Utah

🇺🇸

Salt Lake City, Utah, United States

Virginia Mason Medical Center

🇺🇸

Seattle, Washington, United States

Johns Hopkins University School of Medicine

🇺🇸

Baltimore, Maryland, United States

Mayo Clinic - Scottsdale

🇺🇸

Scottsdale, Arizona, United States

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