Oral Treprostinil for Raynaud's Disease

Overseen By Ruth Marrero

Age: 18+

Sex: Any

Trial Phase: Phase 4

Sponsor: Brigham and Women's Hospital

Must be taking: Vasodilators

Must not be taking: Other prostacyclin

Disqualifiers: Uncontrolled hypertension, diabetes, malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 4

Duration: 26 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new pill called oral treprostinil, which might improve blood flow for people with severe Raynaud's disease. Raynaud's causes fingers and toes to change colors and feel painful or tingly when exposed to cold or stress. The trial seeks participants who experience four or more attacks per week and whose symptoms do not improve with usual treatments. Participants will take the medication and track their symptoms to assess improvement. As a Phase 4 trial, this research aims to understand how the already FDA-approved and effective treatment benefits more patients.

Do I have to stop taking my current medications for the trial?

The trial does not require you to stop taking your current medications if you are on a stable dose of certain drugs like phosphodiesterase inhibitors, endothelin antagonists, alpha adrenergic antagonists, or calcium channel blockers for at least 3 months. However, you cannot participate if you are currently taking any other prostacyclin.

What is the safety track record for oral treprostinil?

A previous study found that oral treprostinil was not well tolerated by patients with scleroderma (a disease affecting connective tissue) who also had calcinosis (calcium deposits in the skin). Despite this, 80% of these patients maintained stable conditions, although they faced challenges with treatment tolerance. Another study showed that a single dose of treprostinil warmed fingers and improved symptoms in people with Raynaud's, suggesting potential benefits for blood flow issues.

This treatment is currently in a Phase 4 trial, indicating FDA approval for other conditions and some established safety. However, side effects remain under close observation. Prospective participants should discuss potential risks and benefits with their healthcare provider. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Unlike the standard vasodilatory therapies for Raynaud’s disease, which often include calcium channel blockers and vasodilators like nifedipine, oral treprostinil offers a unique approach. Treprostinil is a prostacyclin analog, which means it mimics the effects of a natural substance in the body that dilates blood vessels and inhibits platelet aggregation, thus potentially improving blood flow more effectively. Additionally, oral administration provides a more convenient delivery method compared to traditional intravenous options for similar prostacyclin-based therapies. Researchers are excited about treprostinil because it may offer relief for patients who have not responded well to existing treatments, offering a new ray of hope for those with difficult-to-treat Raynaud’s phenomenon.

What evidence suggests that oral treprostinil might be an effective treatment for Raynaud's?

Research has shown that oral treprostinil, a pill form of a specific therapy, improves blood flow for people with Raynaud's disease. In earlier studies, patients experienced quicker warming of their fingers and fewer Raynaud's attacks with this medication. One study found that oral treprostinil was easily absorbed and improved skin symptoms in patients with scleroderma, a related condition. This trial will assess the efficacy of oral treprostinil in patients with severe Raynaud's who haven't found relief from other treatments, suggesting it could provide significant benefits. ^{1 3 5 6 7}

Who Is on the Research Team?

Aaron B Waxman, MD, PhD

Principal Investigator

Brigham and Women's Hospital, Pulmonary Vascular Disease Program

Are You a Good Fit for This Trial?

This trial is for individuals with severe Raynaud's Disease or secondary Raynaud's associated with connective tissue disorders like scleroderma, who haven't found relief from standard treatments. It aims to help those especially with painful sores on their fingertips due to poor blood flow.

Inclusion Criteria

Patients with primary Raynaud's Phenomenon

I have Raynaud's Phenomenon with 4+ attacks weekly despite treatment for 3 months.

I've been on a stable dose of certain heart or blood pressure medicines for 3 months.

Exclusion Criteria

Participation in trial with an investigational drug within 30 days

I do not have uncontrolled high blood pressure, diabetes, recent heart or brain events, cancer, or a history of nerve surgery.

I have smoked or used nicotine products within the last 3 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Run-in

Participants record the number and duration of Raynaud's attacks daily for baseline data

6 weeks

Daily self-reporting

Titration

Participants are titrated to a goal dose of 3mg TID of oral treprostinil

6 weeks

Regular monitoring visits

Treatment

Participants continue treatment with oral treprostinil and record daily Raynaud's attacks

26 weeks

Regular monitoring visits every 4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Oral treprostinil

Trial Overview The study tests oral treprostinil (UT-15C) sustained release tablets, a medication that mimics prostacyclin and may improve blood flow by widening the vessels. This could potentially offer better treatment for severe cases of Raynaud's phenomenon.

How Is the Trial Designed?

Group I: An open label study to assess efficacy of oral treprostinil in patients with Raynaud's Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Brigham and Women's Hospital

Lead Sponsor

Trials

1,694

Recruited

14,790,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07112183

NCT07112183 | Open Label Treprostinil Raynaud's Study 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 ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5011881/

Open label study of escalating doses of oral treprostinil ... Our data show that the oral SR formulation of treprostinil diethanolamine was effectively absorbed in patients with SSc, was associated with improved cutaneous ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT02583789?cond=Raynaud's%20Syndrome&viewType=Table&rank=6

Assess Efficacy of of Oral Treprostinil in Patients With ... The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure.

4. academic.oup.com academic.oup.com/rheumatology/article/64/10/5498/8152875

An exploratory trial of a single dose of CAM2043 (treprostinil ... A single dose of CAM2043 was associated with increased finger rewarming and improvement in Raynaud's. CAM2043 could be further investigated in ...

5. trial.medpath.com trial.medpath.com/clinical-trial/f6f99a106bf45c5d/nct07112183-oral-treprostinil-raynauds-phenomenon-vasodilatory-therapy

Open Label Treprostinil Raynaud's Study - MedPath 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) ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9308380/

A pilot study to evaluate the safety and efficacy of ... Oral treprostinil was poorly tolerated in SSc patients with calcinosis. Of five patients who completed treatment, most (80%) had documented stability of ...

7. withpower.com withpower.com/trial/phase-4-raynaud-disease-7-2025-2792d

Oral Treprostinil for Raynaud's Disease · Info for Participants This Phase 4 medical study run by Brigham and Women's Hospital needs participants to evaluate whether Oral treprostinil will have tolerable side effects ...