HS-18-638
Research type
Research Study
Full title
An Exploratory Trial Assessing Digital Vascular Perfusion, Pharmacokinetics, Safety and Tolerability Following a Single Dose of CAM2043 (Treprostinil Subcutaneous Depot) in Patients with Raynaud’s Phenomenon Secondary to Systemic Sclerosis
IRAS ID
272415
Contact name
Ariane Herrick
Contact email
Sponsor organisation
Camurus AB
Eudract number
2019-002444-24
Duration of Study in the UK
0 years, 3 months, 29 days
Research summary
Raynaud’s phenomenon is an exaggeration of the normal physiological response to cold exposure or to emotional stress. Raynaud’s phenomenon typically manifests in 3 stages: extremities turn white, followed by blue, and finally red, usually associated with pain. Raynaud’s phenomenon is common and exists as primary Raynaud’s phenomenon or as secondary Raynaud’s phenomenon secondary due to underlying disease, for example in connective tissue diseases such as systemic sclerosis. Systemic sclerosis, also referred to as scleroderma, is a rare rheumatic disease which frequently presents as fibrosis of the skin and internal organs. More than 95% of patients with systemic sclerosis experience Raynaud’s phenomenon.
Raynaud’s phenomenon in systemic sclerosis may have a great negative impact on the patient’s quality of life. Approximately half of patients with systemic sclerosis develop digital (finger) ulcers.
Changes in lifestyle may be enough to reduce symptoms, for example, avoiding exposure to cold or stop smoking, but many patients also require drug treatment or surgical treatment. There are only a limited number of medicinal products which are approved for the treatment of Raynaud’s phenomenon. As not all patients respond adequately to those treatments which are available, there is an unmet medical need for alternative treatments.
CAM2043 is an investigational drug, which means it has not yet been approved for the treatment of Raynaud’s phenomenon secondary to systemic sclerosis.
The active substance in CAM2043 is called treprostinil. Treprostinil widens blood vessels. As Raynaud’s phenomenon is characterized by changes in finger and toe blood flow, a drug which affects the blood flow in the fingers may also have a beneficial effect in Raynaud’s phenomenon. In this small study, the effect of CAM2043 on finger blood flow will be investigated by a technique called thermography following a cold challenge. The purpose is to understand if there is a potential effect of CAM2043 on Raynaud’s phenomenon.REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
20/NW/0037
Date of REC Opinion
18 Feb 2020
REC opinion
Favourable Opinion