Institutional Review Board, Hospital for Special Surgery
July 27, 2010
The safety of study participants is our top priority. The trial is approved and periodically reviewed by an Institutional Review Board (IRB), which includes doctors, administrators, ethicists, and members of the general public. The safety of clinical trials is reviewed by the U.S. Food and Drug Administration.
Before enrolling in a clinical trial, the investigator will explain the purpose of the trial, its expected benefits, any possible risks or side effects, and what your role will be. This is the time to ask questions! If you want to join the trial, you must sign the informed consent documents. You can leave a clinical trial at any time without penalty.
For further information, see Understanding Clinical Trials.
This study will evaluate the effect of treprostinil diethanolamine (UT-15C) sustained release tablets (compared to placebo) on digital ulcers in patients with scleroderma (limited or diffuse systemic sclerosis.) Treprostinil diethanolamine is an analogue of prostacyclin. Prostacyclin is a naturally occurring substance produced by the cells of blood vessels that inhibits platelet aggregation, induces vasodilation, and suppresses smooth muscle proliferation. Improvement in blood flow in lower limbs and fingers would be anticipated to result in a reduction in ischemic pain, Raynaud's phenomenon and promote healing of digital ulcers and other ischemic wounds. This is a double blinded, placebo-controlled trial lasting 20 weeks, at the end of which patients should be given the opportunity to receive the active drug (treprostinil diethanolamine) in an open-label extension phase. .
Inclusion Criteria:
• Subject gives voluntary written informed consent to participate in the study.
• Diagnosis of systemic sclerosis (SSc) as defined by American College of Rheumatology (ACR) criteria.
• Males and females age greater than 18 years
• Presence of at least one active digital ulcer (meets protocol defined qualifications for active digital ulcer)
• Females of childbearing potential must be willing to use a reliable form of medically acceptable contraception and have a negative pregnancy test
• Able to communicate effectively with study personnel and willing to comply with protocol requirements.
Exclusion Criteria:
• Diagnosis of pulmonary arterial hypertension (PAH).
• Body weight less than 40 kg
• History of postural hypotension, unexplained syncope, a blood pressure that is less than 90 mmHg systolic or 50 mmHg diastolic at Screening and Baseline.
• Hemoglobin concentration less than 75% of the lower limit of the normal range
• Moderate to severe hepatic impairment, i.e., Child-Pugh Class B or C.
• 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.
• Pregnancy or breast-feeding.
• Overlap with another connective tissue disease that could affect rest pain and hand function (e.g. diabetes mellitus, rheumatoid arthritis).
• Sympathectomy of the upper limb performed within 12 months of Baseline. Sympathectomy performed on the non-target limb (hands not presenting with qualifying ulcers) or which did not include the hand performed within 6 months of Baseline.
• 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.
• Required systemic antibiotics for infected digital ulcers within 2 weeks of Screening.
• Local injection of botulinum toxin in an affected finger within 1 month prior to Baseline.
• Treatment with endothelin receptor antagonists within 1 month prior to Baseline.
• Patients on phosphodiasterase inhibitors, such as sildenafil, or tadalafil, who have received treatment for less than 6 months prior to Baseline (unless for intermittent treatment of male erectile dysfunction).
• Treatment with statin within 1 month prior to Screening, unless for management of hyperlipidemia.
• Received an investigational product within 1 month preceding Screening.
• Known hypersensitivity to treprostinil diethanolamine or any of the excipients.
• Tobacco use at any level within the past 6 months prior to Screening.
*Please note this is an abbreviated list of criteria. Please contact the study coordinator.
Kamini Doobay
doobayk@hss.edu
212.774.2123