Trial record 9 of 194 for:
Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil Diethanolamine (DISTOL-1)
This study has been completed.
Information provided by (Responsible Party):
First received: October 17, 2008
Last updated: February 26, 2013
Last verified: January 2011
This study will evaluate the effect of treprostinil diethanolamine (UT-15C) sustained release tablets(compared to placebo) on digital ulcers in patients with scleroderma. Treprostinil diethanolamine is an analog of prostacyclin. Prostacyclin is a naturally occuring 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.
Drug: treprostinil diethanolamine
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
||DISTOL-1: Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil Diethanolamine: A Randomized, Double-blind, Placebo-controlled, Multicenter Study
Primary Outcome Measures:
- Reduction in net ulcer burden [ Time Frame: Through week 20 ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Scleroderma Health Assessment Questionnaire (SHAQ) and other functional and quality of life scales [ Time Frame: Through week 20 ] [ Designated as safety issue: No ]
- modified Rodnan Skin Score (mRSS) [ Time Frame: Baseline and week 20 ] [ Designated as safety issue: No ]
- serum biomarkers [ Time Frame: Baseline and week 20 ] [ Designated as safety issue: No ]
- adverse events [ Time Frame: Throughout week 20 ] [ Designated as safety issue: Yes ]
| Study Start Date:
| Study Completion Date:
| Primary Completion Date:
||March 2011 (Final data collection date for primary outcome measure)
Experimental: treprostinil diethanolamine
Drug: treprostinil diethanolamine
oral sustained release tablet. Maximum tolerable dose not exceeding 16 mg twice daily (BID)
Placebo Comparator: placebo (sugar pill)
|Ages Eligible for Study:
||18 Years and older
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
- 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.
- 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 refer to this study by its ClinicalTrials.gov identifier: NCT00775463
||James Seibold, MD
||Scleroderma Research Consultants LLC, Avon, CT,
No publications provided
History of Changes
|Other Study ID Numbers:
|Study First Received:
||October 17, 2008
||February 26, 2013
||United States: Food and Drug Administration
United Kingdom: Research Ethics Committee
Keywords provided by United Therapeutics:
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on December 12, 2013
Connective Tissue Diseases