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 : Completed
First Posted : January 26, 2016
Results First Posted : June 10, 2021
Last Update Posted : June 10, 2021
|Condition or disease||Intervention/treatment||Phase|
|Systemic Sclerosis Calcinosis||Drug: Oral treprostinil||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Actual Primary Completion Date :||May 13, 2020|
|Actual Study Completion Date :||May 13, 2020|
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
- Number of Participants With Treatment-related Adverse Events Following Treatment With Oral Treprostinil [ Time Frame: 12 months ]Number of participants with treatment-related adverse events following treatment with oral treprostinil at 12 months. We defined adverse event as any untoward medical experience occurring to a subject during a clinical trial whether or not it is related to the study drug.
- 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 ]
Median rate of change of calcinosis in radiograph following treatment with oral treprostinil as assessed by the Scleroderma Clinical Trial Consortium (SCTC) radiographic scoring system. Historical average SCTC scores in this patient population have ranged from 4.08 to 472.88, with higher scores indicating more severe symptoms.
The SCTC radiographic score for calcinosis is a validated radiographic scoring system to assess the severity of calcinosis affecting the hands of patients with SSc that accounts for area coverage, density, and anatomic location, higher scores mean worse calcinosis. Physician rates density and percentage of area for 22 regions of each hand, each deferentially weighted in the overall score; individual region values are multiplied my their weight then summed to create an overall score.
- Change in Scleroderma Health Assessment Questionnaire (SHAQ) [ Time Frame: Baseline, 12 months ]
The Scleroderma Health Assessment Questionnaire (SHAQ) has two parts.
The first part is a disability index (DI) that consists of the mean of 8 scores from 8 sections (dressing, arising, eating, walking, hygiene, reach, grip, and activities) ranging from 0 (without any difficulty) to 3 (unable to do), with scores summed and divided by 8. The result is the Health Assessment Questionnaire (HAQ)-DI score, which ranges between 0 and 3.
The second part consists of 6 visual analogue scales (VAS) each ranging from 0 (better outcome) to 10 (worse outcome).
- Pain (SHAQ-VAS-Pain)
- Intestinal (SHAQ-VAS-GI)
- Breathing (SHAQ-VAS-Breathing)
- Raynaud (SHAQ-VAS-Raynaud)
- Digital ulcers (SHAQ-VAS-DU)
- Disease Severity (SHAQ-VAS-Disease Severity)
- Change in Cochin Hand Functional Scale [ Time Frame: Baseline, 12 months ]Cochin Hand Functional Scale is a 18-question scale, each question ranges from 0 to 5, with a total score range of 0-90. Higher scores indicates higher disability.
- Change in Short Form (SF)-36 [ Time Frame: Baseline, 12 months ]The SF-36 consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale. The higher the score the less disability.
- Change in Mawdsley Calcinosis Questionnaire [ Time Frame: Baseline, 12 months ]Mawdsley Calcinosis Questionnaire is an 18-question survey with an overall score of 0-10; each individual question ranges from 0-10, scores are totaled and averaged to and divided by 18 to create the overall score. Higher scores indicate higher disability from calcinosis.
- Change in Raynaud Condition Score [ Time Frame: Baseline, 12 months ]The Raynaud Condition Score is a single question questionnaire, ranges from 0-10, where 10 indicates more active Raynaud phenomenon. Raynaud is an exaggerated vascular response to cold exposure or emotion with at least a 2-phase color change in finger(s) and often toe(s) consisting of pallor, cyanosis, and/or reactive hyperemia: usually one phase is pallor.
- Change in Patient Global Assessment of Calcinosis Severity [ Time Frame: Baseline, 12 months ]Patient global assessment of calcinosis severity at 1 year versus baseline. Score range: 0 to 10 (higher scores indicate more severe calcinosis).
- Change in Physician Global Assessment of Calcinosis Severity [ Time Frame: Baseline, 12 months ]Physician global assessment of calcinosis severity at 1 year versus baseline. Score range: 0 to 10 (higher scores indicate more severe calcinosis).
- Baseline Cortical Area Measured by HR-pQCT in SSc Patients With Calcinosis. [ Time Frame: Baseline visit (average approximately 3 hours for the scan) ]Bone cortical area is a bone microarchitecture measurement assessed by High Resolution peripheral Quantitative Computed Tomography (HRpQCT) at the distal tibia. Lower levels of cortical area are associated with more bone fragility.
- Baseline Cortical Porosity Measured by HR-pQCT in SSc Patients With Calcinosis. [ Time Frame: Baseline visit (average approximately 3 hours for the scan) ]Bone cortical porosity is a bone microarchitecture measurement assessed by High Resolution peripheral Quantitative Computed Tomography (HRpQCT) at the distal tibia. Higher levels of cortical porosity are associated with more bone fragility.
- 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 ]SSc-PAH associated biomarkers following treatment with treprostinil at 1 year compared to baseline. Ang-1 Ang-2 MMP-2, MMP-9 NT-proBNP, PIGF VEGFR1, sRAGE GLUT-1 Ficolin-1 MBL H62 plex (includes VEGF, PDGFBB, bFGF,IL-13, HGF, IL-6, IL-1β, IL-2, IL-4, IL-5, IL-8, IL-10, IL-12, IL-13,[TNF]-α,[IFN]-γ)
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
|United States, California|
|Stanford University School of Medicine|
|Palo Alto, California, United States, 94304|
|Principal Investigator:||Lorinda S Chung, MD, MS||Stanford University|