Open-label oral treprostinil for Raynaud's

An Open Label Study to Assess Efficacy of Oral Treprostinil Titrated to 3.0mg Three Times Daily or Highest Tolerable Dose in 30 Patients With Symptomatic Primary or Secondary Raynaud's Phenomenon Resistant to Standard Vasodilatory Therapy

This trial will test whether oral sustained-release treprostinil (UT-15C) can reduce the number and severity of Raynaud's attacks in adults with frequent primary or secondary Raynaud's.

Quick facts

• Phase: Phase 4
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Brigham and Women's Hospital (Academic / other)
• Drugs / interventions: immunotherapy
• Locations: 2 sites (Boston, Massachusetts and 1 other locations)
• Trial ID: NCT07112183 on ClinicalTrials.gov

What this trial studies

This open-label Phase 4 trial gives adults with frequent Raynaud's oral sustained-release treprostinil (UT-15C) and monitors their symptoms and attack frequency. Eligible participants must have at least four Raynaud's attacks per week and may have primary Raynaud's or Raynaud's secondary to connective tissue disease; background vasodilator medications are allowed if doses have been stable for at least three months. All participants receive the active medication (no placebo control) and outcomes focus on changes in attack frequency, severity, and related complications. The study is conducted at Brigham and Women's Hospital in Boston, Massachusetts.

Who should consider this trial

Good fit: Adults (≥18 years) with refractory Raynaud's experiencing four or more attacks per week, including primary Raynaud's or Raynaud's secondary to connective tissue diseases, and who are on stable background vasodilator therapy if applicable.

Not a fit: Patients with infrequent attacks (fewer than four per week), pregnant individuals, or those with contraindications to prostacyclin analogs are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, the medication could reduce the frequency and severity of Raynaud's attacks and decrease the risk of painful digital complications.

How similar studies have performed: Prostacyclin analogs such as treprostinil have shown benefit for digital ischemia and pulmonary hypertension in prior studies, but oral treprostinil specifically for Raynaud's has limited published evidence.

Eligibility criteria

Inclusion Criteria:

1. Patients aged ≥ 18 years
2. 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.
3. Patients with either:

   1. Primary Raynaud's Phenomenon
   2. 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.
4. If patients are on phosphodiesterase inhibitors (sildenafil, tadalafil or vardenafil), endothelin antagonists, alpha adrenergic antagonists, or calcium channel blockers, their dose must be stable, defined as 3-months with no change in dose or frequency.
5. If female of childbearing potential (FOCP), has demonstrated a negative beta HCG (human chorionic gonadotropin) serum pregnancy test, and agrees to comply with any applicable contraceptive requirements of the protocol
6. If female of non-childbearing potential, meets either of the following criteria:

   1. Is in a postmenopausal state defined as no menses for 12 months without an alternative medical cause and follicle-stimulating hormone (FSH) level consistent with postmenopause (\>25 mIU/mL). If a participant is \>60 years old and has been amenorrheic for \>5 years, the participant may be enrolled with an FSH \<25 mIU/mL after discussion with the PI.
   2. Has a documented hysterectomy, bilaterial oophorectomy, or salpingectomy.

Exclusion Criteria:

1. Uncontrolled hypertension, diabetes mellitus, acute coronary or cerebrovascular event within 3 months, history of sympathectomy
2. Smoking within 3 months or smoking cessation using nicotine products
3. History of alcohol or other substance abuse within the previous year
4. Subjects with diverticulosis confirmed via endoscopic evaluation of the sigmoid colon with at least three diverticula noted
5. Subjects with moderate to severe liver disease, Child Pugh Class B or C
6. Subjects currently taking any other prostacyclin.
7. Pregnant or breast feeding or considering pregnancy in next 4 months
8. Was dosed in any clinical research study evaluating another investigational drug (including biologics) or therapy (including specific immunotherapy) within 30 days or 5 half-lives (whichever is longer) of an investigational biologic drug before the start of the screening period.
9. Any serious chronic, and/or unstable pre-existing medical, surgical, psychiatric or other condition that could interfere with the participant's safety, obtaining informed consent, or compliance with study procedures per investigator's discretion.
10. Hospitalization for any indication within 7 days before the start of screening.

Where this trial is running

Boston, Massachusetts and 1 other locations

• Brigham and Women's Hospital — Boston, Massachusetts, United States (Not_yet_recruiting)
• Brigham and Women's Hospital — Boston, Massachusetts, United States (Recruiting)

Study contacts

• Principal investigator: Aaron B Waxman, MD, PhD — Brigham and Women's Hospital, Pulmonary Vascular Disease Program
• Study coordinator: Olivia G Vayer

Conditions Raynaud's Disease; Raynaud Phenomena; Raynauds; Raynaud' s; Raynaud's Phenomena; Connective Tissue Disease; Systemic Sclerosis