Systemic Sclerosis, scleroderma digital ulcers MedDRA version: 9.1 Level: LLT Classification code 10042953 Term: Systemic sclerosis MedDRA version: 9.1 Level: LLT Classification code 10039710 Term: Scleroderma
Conditions
Sponsors
United Therapeutics Corporation
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Subject gives voluntary written informed consent to participate in the study. 2) Subject has been diagnosed with systemic sclerosis (SSc) as defined by American College of Rheumatology (ACR) criteria. 3) Males and females age greater than 18 years at time of Screening. 4)Presence of at least one active digital ulcer (meets protocol defined qualifications for active digital ulcer, Section 3.3.1.1) at Baseline. 5) Females of childbearing potential must be willing to use a reliable form of medically acceptable contraception and have a negative pregnancy test at Screening and confirmed at Baseline. Women who are surgically sterile or have been post-menopausal for at least 2 years are not considered to be of child-bearing potential. 6) Subject is able to communicate effectively with study personnel and be considered reliable, willing and cooperative in terms of compliance with the protocol requirements. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1)Has diagnosis of pulmonary arterial hypertension (PAH). 2)Body weight less than 40 kg at time of Screening, confirmed at Baseline. 3)The subject has a history of postural hypotension, unexplained syncope, a blood pressure that is less than 95 mmHg systolic or 50 mmHg diastolic at Screening or Baseline. 4)Hemoglobin concentration less than 75% of the lower limit of the normal range at time of Screening. 5)Moderate to severe hepatic impairment, i.e., Child-Pugh Class B or C. 6)Intractable diarrhea, or severe malabsorption, defined as greater than 15% unintentional loss of body weight in the last 6 months prior to Screening; any severe organ failure (e.g., lung, kidney), bleeding diathesis or platelet disorder, or any life-threatening condition. 7)Pregnancy or breast-feeding. 8) Simultaneously fulfills criteria for a second connective tissue disease including systemic lupus erythematosus, rheumatoid arthritis, or inflammatory myopathy 9)Sympathectomy of the upper limb performed within 12 months of Baseline. 10)Receipt of prostanoid treatment (epoprostenol, treprostinil sodium, or other prostacyclin analog) within the previous 3 months of Baseline for conditions including Reynaud’s phenomenon, rest pain and / or digital ulcers. 11)Patients who have required systemic antibiotics for infected digital ulcers within 2 weeks of Screening. 12)Local injection of botulinum toxin in an affected finger within 1 month prior to Baseline. 13)Treatment with endothelin receptor antagonists within 1 month prior to Baseline. 14)Treatment with phosphodiesterase inhibitors such as sildenafil, except for intermittent treatment of male erectile dysfunction. 15)Treatment with statin within 1 month prior to Screening, unless for management of hyperlipidemia. 16)Received an investigational product within 1 month preceding Screening. 17)Known hypersensitivity to treprostinil diethanolamine or any of the excipients. 18)Tobacco or nicotine use at any level within the past 6 months prior to Screening. 19)Any condition or laboratory value that in the opinion of the investigator might interfere with the subject's participation in the study, poses an added risk for the subject, could prevent understanding the objectives, nature, or consequences of the trial, compliance with the protocol, adherence to therapy or that would interfere with the interpretation of study assessments.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the efficacy of treprostinil diethanolamine on net ulcer burden compared to placebo in patients with systemic sclerosis (SSc);Secondary Objective: To assess the effect of treprostinil diethanolamine as compared to placebo on: 1. Formation of new ischemic digital ulcers 2. Healing of active baseline ulcers 3. Digital ulcer related pain (as measured by Visual Analogue Scale [VAS-Pain] and Short Form McGill Pain Questionnaire [SF MPQ]) 4. Raynaud’s phenomenon (as measured by the Scleroderma Specific Raynaud’s Visual Analogue Scale [SSRVAS] -- a subcomponent of the Scleroderma Health Assessment Questionnaire [SHAQ]) 5.Patient function (as measured by the SHAQ and Cochin Hand Function Scale [CHFS]) 6.Skin thickening (as measured by the modified Rodnan Skin Score [mRSS]) 7.Patient quality of life (as measured by the SF 36) 8.Patient and physician global assessment of ulcer (as measured by patient impression of change questionnaire [PIC] and Visual Analogue Scale [VAS-Global]) 9. Vascular and SSc associated biomarkers ;Primary end point(s): Reduction in net ulcer burden | — |
Countries
United Kingdom
Outcome results
None listed