Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil Diethanolamine

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00775463
• Date of registration: 17/10/2008
• Prospective Registration: Yes
• Primary sponsor: United Therapeutics
• Public title: Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil Diethanolamine DISTOL-1
• Scientific title: DISTOL-1: Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil Diethanolamine: A Randomized, Double-blind, Placebo-controlled, Multicenter Study
• Date of first enrolment: May 2009
• Target sample size: 148
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00775463
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

Canada, United Kingdom, United States

Contacts

• Name: James Seibold, MD
• Affiliation: Scleroderma Research Consultants LLC, Avon, CT,

Key inclusion & exclusion criteria

Inclusion Criteria:

- Subject gave 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 at Screening

- Presence of at least one active digital ulcer (met protocol defined qualifications for
active digital ulcer) at Baseline

- Females of childbearing potential willing to use a reliable form of medically
acceptable contraception and have a negative pregnancy test at Screening and Baseline

- 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 at Screening and confirmed at Baseline

- 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 at
Screening

- Moderate to severe hepatic impairment, i.e., Child-Pugh Class B or C, or ALT greater
than three times upper limit of normal

- 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

- Pregnant or breast-feeding

- Simultaneously fulfilled criteria for a second connective tissue disease including
systemic lupus erythermatosus, rheumatoid arthritis or inflammatory myopathy

- Sympathectomy of the upper limb, involving the hand, performed within 12 months of
Baseline. Sympathectomy performed on the non-target limb (hand 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
Raynaud'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 or nicotine use at any level within the past 6 months prior to Screening

- Any condition or laboratory that in the opinion of the investigator might interfere
with subject's participation, pose an additional 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 interpretation of study
assessments



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

Health Condition(s) or Problem(s) studied

Scleroderma

Systemic Sclerosis

Intervention(s)

Drug: placebo

Drug: treprostinil diethanolamine

Primary Outcome(s)

Net Ulcer Burden [Time Frame: Week 20]

Secondary Outcome(s)

Time to Ulcer Healing- Percentage of Subjects With Complete Healing [Time Frame: Week 20]

Cochin Hand Function Scale (CHFS) [Time Frame: Week 20]

Scleroderma Health Assessment Questionnaire (SHAQ) [Time Frame: Week 20]

Short Form 36 [Time Frame: Week 20]

Short-Form McGill Pain Questionnaire [Time Frame: Week 20]

Time to Ulcer Healing [Time Frame: Week 20]

Modified Rodnan Skin Score (mRSS) [Time Frame: Week 20]

Patient Impression of Change (PIC) Questionnaire [Time Frame: Week 20]

Digital Ulcer Pain VAS [Time Frame: Week 20]

Patient Global Assessment of Digital Ulcer Severity VAS [Time Frame: Week 20]

Physician Global Assessment of Digital Ulcer Severity VAS [Time Frame: Week 20]

Secondary ID(s)

TDE-DU-201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 17/03/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00775463