Open Label Treprostinil Raynaud's Study

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	18 Years and Over
Gender	All

More Inclusion & 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 Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT07112183
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 4
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	Brigham and Women's Hospital
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	Aaron B Waxman, MD, PhD
Principal Investigator Affiliation	Brigham and Women's Hospital, Pulmonary Vascular Disease Program
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Other
Overall Status	Not yet recruiting
Countries	United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Raynaud's Disease, Raynaud Phenomena, Raynauds

Arms & Interventions

Arms

Experimental: 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.

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 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.

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Brigham and Women's Hospital, Boston 4930956, Massachusetts 6254926

Status

Address

Brigham and Women's Hospital

Boston 4930956, Massachusetts 6254926, 02115-6110

Site Contact

Aaron B Waxman, MD, PhD

[email protected]

6175259733

Clinical Trial Finder