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A Pilot Study to Evaluate the Safety and Efficacy of Oral Treprostinil in the Treatment of Calcinosis in Patients With Systemic Sclerosis

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ClinicalTrials.gov Identifier: NCT02663895
Recruitment Status : Recruiting
First Posted : January 26, 2016
Last Update Posted : April 19, 2018
Sponsor:
Collaborator:
United Therapeutics
Information provided by (Responsible Party):
Lorinda S Chung, Stanford University

Study Description
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Brief Summary:
This is a prospective open-label trial that will enroll 12 patients with systemic sclerosis (SSc) and at least one calcinotic lesion of the hands that is palpable on physical examination and also measureable on hand radiographs, at one single center. Each subject will receive treprostinil orally for 12 months, and follow-up evaluations will be performed every 3 months. Our main objective is to determine whether oral treprostinil is safe, and effective in reducing calcinosis in patients with SSc. We hypothesize that calcinosis is a result of microvascular injury and ischemic damage, and that therefore treprostinil may be beneficial in the treatment of calcinosis in patients with SSc.

Condition or disease Intervention/treatment Phase
Systemic Sclerosis Calcinosis Drug: Oral treprostinil Phase 2

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Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Study to Evaluate the Safety and Efficacy of Oral Treprostinil in the Treatment of Calcinosis in Patients With Systemic Sclerosis
Study Start Date : October 2016
Estimated Primary Completion Date : June 2019
Estimated Study Completion Date : June 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Scleroderma

Arms and Interventions
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Arm Intervention/treatment
Experimental: Oral treprostinil
Treprostinil 0.125 mg TID orally, which will be increased by 0.125 mg TID every 3 to 4 days as tolerated for 12 months
 Drug: Oral treprostinil
Treprostinil 0.125 mg TID orally, which will be increased by 0.125 mg TID every 3 to 4 days as tolerated
Other Name: Orenitram


Outcome Measures
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Primary Outcome Measures :
  1. To assess the number of participants with treatment-related adverse events following treatment with oral treprostinil [ Time Frame: 12 months ]
  2. To determine the mean rate of change of calcinosis in radiograph following treatment with oral treprostinil as assessed by a novel radiographic scoring system [ Time Frame: 12 months ]

Secondary Outcome Measures :
  1. To assess the effect of oral treprostinil on the change in Scleroderma Health Assessment Questionnaire (SHAQ) [ Time Frame: 12 months ]
  2. To assess the effect of oral treprostinil on the change in Cochin Hand Functional Scale [ Time Frame: 12 months ]
  3. To assess the effect of oral treprostinil on the change in SF-36 [ Time Frame: 12 months ]
  4. To assess the effect of oral treprostinil on the change in Mawdsley Calcinosis Questionnaire [ Time Frame: 12 months ]
  5. To assess the effect of oral treprostinil on the change in Raynaud Condition Score [ Time Frame: 12 months ]
  6. To assess the effect of oral treprostinil on the change in patient global assessment of calcinosis severity [ Time Frame: 12 months ]
  7. To assess the effect of oral treprostinil on the change in physician global assessment of calcinosis severity [ Time Frame: 12 months ]
  8. To assess changes in blood flow using SPY perfusion machine following treatment with treprostinil at 1 year compared to baseline. [ Time Frame: 12 months ]
  9. To assess the number of patients with changes in vascular and SSc-PAH associated biomarkers following treatment with treprostinil at 1 year compared to baseline [ Time Frame: 12 months ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Signed written informed consent
  • Age > 18 years of age
  • Diagnosis of limited or diffuse cutaneous systemic sclerosis (SSc) according to the revised 2013 ACR/EULAR classification criteria for SSc
  • Radiological and physical examination evidence of at least one subcutaneous calcium deposition in the hands that is clinically apparent as part of routine clinical care.
  • If female of childbearing potential, the patient must have a negative pregnancy test at screening and baseline visits
  • Oral corticosteroids (≤ 10 mg/day of prednisone or equivalent) and NSAIDs are permitted if the patient is on a stable dose regimen for ≥ 2 weeks prior to screening and throughout the study
  • Calcium channel blockers, alpha-1-antagonists, ACE-inhibitors, angiotensin receptor blockers, and protein-pump inhibitors are permitted as long as the doses are stable for 4 weeks prior to screening and throughout the study
  • Women of childbearing potential must agree to use adequate contraception when sexually active with any combination of at least 2 effective methods of birth control (except for women who have a partner who is sterile, i.e. due to vasectomy)

Exclusion Criteria:

  • Rheumatic disease other than SSc
  • Patients with pulmonary arterial hypertension (PAH), NYHA Class III or IV, as determined by right heart catheterization or on PAH approved medications for PAH
  • Patients with moderate or severe hepatic impairment (Child Pugh Class C), or transaminase elevation (ALT or AST) > 3 x the upper limit of normal at screening visit
  • Patients with diverticulosis
  • Hemoglobin < 75% of the lower limit of the normal range
  • Systolic blood pressure < 95 mmHg or diastolic blood pressure < 50 mmHg
  • Patients who are hemodynamically unstable, or have acute renal, cardiac or pulmonary failure, or any life-threatening condition.
  • Concurrent malignancy except non-melanoma skin cancers
  • Patients receiving specific (sildenafil, tadalafil) or unspecific phosphodiesterase-5 inhibitors (dipyridamole, theophylline), endothelin receptor antagonists, prostanoids, riociguat, or NO donors (nitrates) within 4 weeks of screening
  • Patients receiving bisphosphonates, warfarin, colchicine, minocycline, intravenous immunoglobulins, or biological agents including abatacept or rituximab within 4 weeks of screening
  • Patients receiving local treatments for calcinosis including surgical removal or intralesional steroid injections within 12 weeks of screening or throughout the study.
  • Patients who have participated in another clinical trial of an investigative agent within 30 days of screening (or 5 half-lives of the investigational drug, whichever is longer)
  • Pregnant or nursing women
  • Patients with a history of drug or alcohol abuse within 6 months of screening
  • Any medical condition that, in the opinion of the investigator, might interfere with the subject's participation in the study or poses an added risk for the subject
  • Inability to comply with study and follow-up procedures
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02663895


Contacts
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Contact: Lorinda S Chung, MD, MS (650) 723-6961

Locations
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United States, California
Stanford University School of Medicine Recruiting
Palo Alto, California, United States, 94304
Contact: Antonia Maria Valenzuela Vergara, MD, MS    650-723-6961    antoniav@stanford.edu   
Sponsors and Collaborators
Stanford University
United Therapeutics
Investigators
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Principal Investigator: Lorinda S Chung, MD, MS Stanford University
More Information
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Publications:

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Responsible Party: Lorinda S Chung, Associate professor of medicine (Immunology & Rheumatology), Stanford University
ClinicalTrials.gov Identifier: NCT02663895     History of Changes
Other Study ID Numbers: 36140
First Posted: January 26, 2016    Key Record Dates
Last Update Posted: April 19, 2018
Last Verified: April 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Additional relevant MeSH terms:
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Calcinosis
Sclerosis
Scleroderma, Systemic
Scleroderma, Diffuse
Pathologic Processes
Connective Tissue Diseases
 Skin Diseases
Calcium Metabolism Disorders
Metabolic Diseases
Treprostinil
Antihypertensive Agents