Safety and Efficacy of Oral Treprostinil in the Treatment of Calcinosis in Patients With Systemic Sclerosis

• Register: ClinicalTrials.gov
• Last refreshed on: 21 June 2021
• Main ID: NCT02663895
• Date of registration: 19/01/2016
• Prospective Registration: Yes
• Primary sponsor: Stanford University
• Public title: Safety and Efficacy of Oral Treprostinil in the Treatment of Calcinosis in Patients With Systemic Sclerosis
• Scientific title: A Pilot Study to Evaluate the Safety and Efficacy of Oral Treprostinil in the Treatment of Calcinosis in Patients With Systemic Sclerosis
• Date of first enrolment: October 2016
• Target sample size: 12
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02663895
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Lorinda S Chung, MD, MS
• Affiliation: Stanford University

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Calcinosis

Systemic Sclerosis

Intervention(s)

Drug: Oral treprostinil

Primary Outcome(s)

Median Rate of Change of Calcinosis in Radiograph Following Treatment With Oral Treprostinil as Assessed by a Novel Radiographic Scoring System [Time Frame: Baseline, month 12]

Number of Participants With Treatment-related Adverse Events Following Treatment With Oral Treprostinil [Time Frame: 12 months]

Secondary Outcome(s)

Change in Mawdsley Calcinosis Questionnaire [Time Frame: Baseline, 12 months]

Change in Cochin Hand Functional Scale [Time Frame: Baseline, 12 months]

Number of Patients With Changes in Vascular and SSc-Pulmonary Arterial Hypertension (PAH) Associated Biomarkers Following Treatment With Treprostinil at 1 Year Compared to Baseline [Time Frame: 12 months]

Baseline Cortical Area Measured by HR-pQCT in SSc Patients With Calcinosis. [Time Frame: Baseline visit (average approximately 3 hours for the scan)]

Change in Scleroderma Health Assessment Questionnaire (SHAQ) [Time Frame: Baseline, 12 months]

Change in Short Form (SF)-36 [Time Frame: Baseline, 12 months]

Change in Physician Global Assessment of Calcinosis Severity [Time Frame: Baseline, 12 months]

Change in Patient Global Assessment of Calcinosis Severity [Time Frame: Baseline, 12 months]

Baseline Cortical Porosity Measured by HR-pQCT in SSc Patients With Calcinosis. [Time Frame: Baseline visit (average approximately 3 hours for the scan)]

Change in Raynaud Condition Score [Time Frame: Baseline, 12 months]

Secondary ID(s)

36140

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

United Therapeutics

Results

• Results available: Yes
• Date Posted: 10/06/2021
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02663895