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

Plain English Summary

Open Label Treprostinil Raynaud's Study is a Phase 4 clinical trial sponsored by Brigham and Women's Hospital studying Raynaud's Disease, Raynaud Phenomena, Raynauds. This study tests if an oral medication called treprostinil can help people with severe Raynaud's phenomenon. It is for adults (18+) with Raynaud's that causes frequent, painful attacks and doesn't improve with usual medicines. Participants will take an oral medication and have their symptoms tracked for about 38 weeks. Alternatives include staying warm, lifestyle changes, and other prescription medications. The trial aims to enroll 30 participants.

Official Summary

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 tre

Who Can Participate

Here is what you need to know about eligibility for this trial. You can join if you are 18 or older and have Raynaud's that causes at least four attacks per week. You must have Raynaud's that is either primary (no other cause) or secondary to certain autoimmune diseases like scleroderma. You cannot join if you have uncontrolled high blood pressure or diabetes, or have had a recent stroke or heart attack. You also cannot join if you smoke, have a history of substance abuse, or have moderate to severe liver disease. This trial is studying Raynaud's Disease, Raynaud Phenomena, Raynauds, so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.

What They're Measuring

The primary outcome measures how much the treprostinil medication improves the severity and frequency of Raynaud's symptoms, aiming to reduce pain and discomfort for patients. The specific primary outcome measures are: Change in the Raynaud's Condition Score from baseline (6-week run in), comparing treprostinil treatment phase vs. baseline phase. (38 weeks). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This is a Phase 4 (post-marketing) study of a treatment that has already received FDA approval. Phase 4 trials monitor long-term safety, effectiveness in broader patient populations, and potential interactions with other treatments in real-world settings. These studies can involve thousands of patients and help identify rare side effects that may not have appeared in earlier, smaller trials.

Why This Trial Matters

This trial addresses a significant unmet need for patients with severe Raynaud's phenomenon whose symptoms are not adequately controlled by existing therapies, offering a potential new oral treatment This research targets Raynaud's Disease, Raynaud Phenomena, Raynauds, where improved treatment options are needed.

Investor Insight

This trial targets a niche but significant patient population with limited treatment options, potentially positioning oral treprostinil as a key therapy for refractory Raynaud's phenomenon. This treatment is already approved and on the market. This post-marketing study monitors real-world outcomes.

Is This Trial Right for Me?

Ask your doctor about the potential benefits and risks of oral treprostinil and how it compares to your current treatments. You will need to track your Raynaud's attacks daily for 6 weeks before starting the medication. The study involves regular clinic visits for check-ups and to adjust your medication dose as needed. This trial is currently recruiting participants. The trial is being conducted at 2 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: NA
• Model: SINGLE_GROUP
• Masking: NONE
• Enrollment: 30 participants

Interventions

• DRUG: Oral treprostinil (UT-15C) sustained release tablets — 30 patients with symptomatic primary or secondary Raynaud's phenomenon (RP) resistant to standard vasodilatory therapy will be enrolled. Prior to treatment, subjects will record each day for 42 days the number of RP attacks as well as the duration of each RP attack, considered to be baseline data. After the 6-week baseline run in, all subjects will receive oral treprostinil sustained release tablets. Dose escalations can occur every 48 hours in 0.125 mg increments. Subjects will be titrated as t

Primary Outcomes

• Change in the Raynaud's Condition Score from baseline (6-week run in), comparing treprostinil treatment phase vs. baseline phase. (38 weeks)

Secondary Outcomes

• Digital Ulcer Score (32 Weeks)

Full 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.

Trial Locations

• Brigham and Women's Hospital, Boston, Massachusetts, United States
• Brigham and Women's Hospital, Boston, Massachusetts, United States

Frequently Asked Questions

Related Conditions

• Raynaud's Disease
• Raynaud's Phenomenon
• Scleroderma
• Lupus
• Sjogren's Syndrome
• Mixed Connective Tissue Disease
• Rheumatoid Arthritis
• Connective Tissue Diseases
• Vasculitis
• Digital Ulcers

More Raynaud's Disease Trials

View all Raynaud's Disease clinical trials