Open Label Treprostinil Raynaud's Study

Mass General Brigham

Status and phase: Not yet enrolling; Phase 4

Conditions: Raynaud Phenomena; Raynaud's Disease; Raynauds

Treatments: Drug: Oral treprostinil (UT-15C) sustained release tablets

Study type: Interventional

Funder types: Other

Identifiers

NCT07112183

2025P001219

Details and patient eligibility

About

Raynaud's phenomenon is a condition where the blood vessels in participants fingers and toes get too narrow when cold or stressed. This makes participants fingers and toes change colors - they might turn white, then blue, and finally red as blood flow returns. It can be painful and cause numbness or tingling.

When participants have Raynaud's, blood vessels react too strongly to cold or stress. Fingers and toes may turn white (blood moves away from the area), blue (lack of oxygen), or red and feel painful or tingly when warming up. These episodes usually last from a few minutes to several hours.

There are two types of Raynaud's. Primary Raynaud's (also called Raynaud's disease) itself and isn't connected to other health problems. It's the most common type and affects mostly women under 30. Secondary Raynaud's (also called Raynaud's phenomenon) is caused by other diseases like lupus, scleroderma, or rheumatoid arthritis. This type tends to be more serious and may cause painful sores on fingertips called digital ulcers.

For mild cases, staying warm might be enough. But if symptoms are severe, participants doctor might prescribe various medications including calcium channel blockers - blood pressure medicines that help open blood vessels, or other vasodilators - medicines that widen blood vessels. About 40% of people with scleroderma develop painful sores on their fingertips called digital ulcers. These happen when there isn't enough blood flow to heal small injuries.

For severe cases with digital ulcers, doctors might use prostacyclin therapy - medicines that mimic a natural substance that opens blood vessels. Oral treprostinil is a newer pill form of prostacyclin therapy that helps improve blood flow. The investigators are conducting a research study testing whether oral treprostinil - a pill that mimics prostacyclin (a natural blood vessel opener) - can help people with severe Raynaud's that doesn't respond to usual treatments. This represents hope for better treatment options for people with the most challenging cases of this condition.

Enrollment: 30 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:•

• Patients aged ≥ 18 years
• Active Raynaud's Phenomenon defined as patients with refractory RP having four or more RP attacks per week in the 4 weeks before inclusion in the study despite stable background vasodilator treatment for at least 3 months.
• Patients with primary Raynaud's Phenomenon
• Patients with Raynaud's secondary to connective tissue diseases (including scleroderma (SSc), limited scleroderma (CREST), mixed connective tissue disease (MCTD), primary Sjogren's syndrome (SS), systemic lupus erythematosus (SLE), with diagnosis of the underlying rheumatic disease based on standard criteria.
• Patients on stable dose phosphodiesterase inhibitors (sildenafil, tadalafil or vardenafil), endothelin antagonists, alpha adrenergic antagonists, or calcium channel blockers defined as 3-months with no change in dose will be allowed to participate.

Exclusion Criteria:

• Uncontrolled hypertension, diabetes mellitus, acute coronary or cerebrovascular event within 3 months, evidence of malignancy, history of sympathectomy
• Smoking within 3 months or smoking cessation using nicotine products
• Subjects with history of diverticulosis
• Subjects with moderate to severe liver disease, Child Pugh Class B or C
• Subjects currently taking any other prostacyclin.
• Pregnant or breast feeding or considering pregnancy in next 4 months
• Participation in trial with an investigational drug within 30 days

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

An open label study to assess efficacy of oral treprostinil in patients with Raynaud's

Experimental group

Description:

A single center open label study to assess efficacy of oral treprostinil titrated to a tolerable goal dose of 3.0 mg three times per day (TID) in 30 patients with symptomatic primary or secondary Raynaud's phenomenon resistant to standard vasodilatory therapy. Eligible subjects at the time of signing an informed consent will have a diagnosis of primary or secondary Raynaud's phenomenon. Once enrolled, subjects will enter a run in period and record each day for 42 days the number of RP attacks as well as the duration of each RP attack. Frequency and duration of RP recorded within 6-weeks before active treatment phase will be baseline data. After the run in period subjects will be titrated as tolerated to a goal dose of 3mg TID over a 6-week period. Throughout the study (6-week titration - 26-week treatment phase) subjects will record each day the number of RP attacks as well as the duration of each RP attack.

Treatment:

Drug: Oral treprostinil (UT-15C) sustained release tablets