Open Label Treprostinil Raynaud's Study

NCT ID: NCT07112183

Last Updated: 2025-08-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

PHASE4

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-08-01

Study Completion Date

2029-03-01

Brief Summary

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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.

Detailed Description

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Conditions

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Raynaud's Disease Raynaud Phenomena Raynauds

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

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 tolerated to a goal dose of 3mg three times daily over a 6-week period. Titration can be done as quickly as tolerated. Once at goal dose subjects will enter the 26-week treatment phase. Throughout the study (6-week titration - 26-week treatment phase) subjects will record each day the number of RP attacks.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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

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.

Group Type EXPERIMENTAL

Oral treprostinil (UT-15C) sustained release tablets

Intervention Type DRUG

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 tolerated to a goal dose of 3mg three times daily over a 6-week period. Titration can be done as quickly as tolerated. Once at goal dose subjects will enter the 26-week treatment phase. Throughout the study (6-week titration - 26-week treatment phase) subjects will record each day the number of RP attacks.

Interventions

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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 tolerated to a goal dose of 3mg three times daily over a 6-week period. Titration can be done as quickly as tolerated. Once at goal dose subjects will enter the 26-week treatment phase. Throughout the study (6-week titration - 26-week treatment phase) subjects will record each day the number of RP attacks.

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Brigham and Women's Hospital

OTHER

Sponsor Role lead

Responsible Party

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Aaron Waxman MD PhD

Executive Director, Center for Pulmonary Heart Diseases, Director, Pulmonary Vascular Disease Program

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Aaron B Waxman, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Brigham and Women's Hospital, Pulmonary Vascular Disease Program

Locations

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Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status

Countries

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United States

Central Contacts

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Olivia G Vayer

Role: CONTACT

6175259733

Ruth Marrero

Role: CONTACT

6175259733

Facility Contacts

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Aaron B Waxman, MD, PhD

Role: primary

6175259733

B

Role: backup

6175259733

References

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Maverakis E, Patel F, Kronenberg DG, Chung L, Fiorentino D, Allanore Y, Guiducci S, Hesselstrand R, Hummers LK, Duong C, Kahaleh B, Macgregor A, Matucci-Cerinic M, Wollheim FA, Mayes MD, Gershwin ME. International consensus criteria for the diagnosis of Raynaud's phenomenon. J Autoimmun. 2014 Feb-Mar;48-49:60-5. doi: 10.1016/j.jaut.2014.01.020. Epub 2014 Feb 1.

Reference Type BACKGROUND
PMID: 24491823 (View on PubMed)

Shah AA, Schiopu E, Hummers LK, Wade M, Phillips K, Anderson C, Wise R, Boin F, Seibold JR, Wigley F, Rollins KD. Open label study of escalating doses of oral treprostinil diethanolamine in patients with systemic sclerosis and digital ischemia: pharmacokinetics and correlation with digital perfusion. Arthritis Res Ther. 2013 Apr 18;15(2):R54. doi: 10.1186/ar4216.

Reference Type BACKGROUND
PMID: 23597147 (View on PubMed)

Milio G, Corrado E, Genova C, Amato C, Raimondi F, Almasio PL, Novo S. Iloprost treatment in patients with Raynaud's phenomenon secondary to systemic sclerosis and the quality of life: a new therapeutic protocol. Rheumatology (Oxford). 2006 Aug;45(8):999-1004. doi: 10.1093/rheumatology/kel038. Epub 2006 Feb 16.

Reference Type BACKGROUND
PMID: 16484290 (View on PubMed)

Roustit M, Blaise S, Allanore Y, Carpentier PH, Caglayan E, Cracowski JL. Phosphodiesterase-5 inhibitors for the treatment of secondary Raynaud's phenomenon: systematic review and meta-analysis of randomised trials. Ann Rheum Dis. 2013 Oct;72(10):1696-9. doi: 10.1136/annrheumdis-2012-202836. Epub 2013 Feb 20.

Reference Type BACKGROUND
PMID: 23426043 (View on PubMed)

Denton CP, Hachulla E, Riemekasten G, Schwarting A, Frenoux JM, Frey A, Le Brun FO, Herrick AL; Raynaud Study Investigators. Efficacy and Safety of Selexipag in Adults With Raynaud's Phenomenon Secondary to Systemic Sclerosis: A Randomized, Placebo-Controlled, Phase II Study. Arthritis Rheumatol. 2017 Dec;69(12):2370-2379. doi: 10.1002/art.40242.

Reference Type BACKGROUND
PMID: 29193819 (View on PubMed)

Garcia de la Pena Lefebvre P, Nishishinya MB, Pereda CA, Loza E, Sifuentes Giraldo WA, Roman Ivorra JA, Carreira P, Rua-Figueroa I, Pego-Reigosa JM, Munoz-Fernandez S. Efficacy of Raynaud's phenomenon and digital ulcer pharmacological treatment in systemic sclerosis patients: a systematic literature review. Rheumatol Int. 2015 Sep;35(9):1447-59. doi: 10.1007/s00296-015-3241-1. Epub 2015 Apr 1.

Reference Type BACKGROUND
PMID: 25824427 (View on PubMed)

Costa E, Cunha-Santos F, Dourado E, Oliveira D, Falzon L, Romao V, Duarte AC, Cordeiro A, Santiago T, Sepriano A. Systematic literature review to inform the Portuguese recommendations for the management of Raynaud's phenomenon and digital ulcers in systemic sclerosis and other connective tissue diseases. ARP Rheumatol. 2024 Jul 1;3(Apr-Jun):128-144. doi: 10.63032/YHBL8967.

Reference Type BACKGROUND
PMID: 38306796 (View on PubMed)

Khouri C, Lepelley M, Bailly S, Blaise S, Herrick AL, Matucci-Cerinic M, Allanore Y, Trinquart L, Cracowski JL, Roustit M. Comparative efficacy and safety of treatments for secondary Raynaud's phenomenon: a systematic review and network meta-analysis of randomised trials. Lancet Rheumatol. 2019 Dec;1(4):e237-e246. doi: 10.1016/S2665-9913(19)30079-7.

Reference Type BACKGROUND
PMID: 38229380 (View on PubMed)

Flavahan NA, Flavahan S, Mitra S, Chotani MA. The vasculopathy of Raynaud's phenomenon and scleroderma. Rheum Dis Clin North Am. 2003 May;29(2):275-91, vi. doi: 10.1016/s0889-857x(03)00021-8.

Reference Type BACKGROUND
PMID: 12841295 (View on PubMed)

Stjernbrandt A, Pettersson H, Lundstrom R, Liljelind I, Nilsson T, Wahlstrom J. Incidence, remission, and persistence of Raynaud's phenomenon in the general population of northern Sweden: a prospective study. BMC Rheumatol. 2022 Jul 21;6(1):41. doi: 10.1186/s41927-022-00272-0.

Reference Type BACKGROUND
PMID: 35858907 (View on PubMed)

Maciejewska M, Sikora M, Maciejewski C, Alda-Malicka R, Czuwara J, Rudnicka L. Raynaud's Phenomenon with Focus on Systemic Sclerosis. J Clin Med. 2022 Apr 28;11(9):2490. doi: 10.3390/jcm11092490.

Reference Type BACKGROUND
PMID: 35566614 (View on PubMed)

Curtiss P, Svigos K, Schwager Z, Lo Sicco K, Franks AG Jr. Part I: Epidemiology, pathophysiology, and clinical considerations of primary and secondary Raynaud's phenomenon. J Am Acad Dermatol. 2024 Feb;90(2):223-234. doi: 10.1016/j.jaad.2022.06.1199. Epub 2022 Jul 7.

Reference Type BACKGROUND
PMID: 35809798 (View on PubMed)

Other Identifiers

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2025P001219

Identifier Type: -

Identifier Source: org_study_id

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