A Study to Investigate Safety of GS-248 and Efficacy on Raynauds' Phenomenon in Systemic Sclerosis

• Status: Recruiting
• Trial end date: 2021-12-31
• Target enrollment: 0
• Participant gender: All

Summary

The primary objective of this study is to determine the safety, and evaluate the efficacy of GS-248 versus placebo on Raynaud's Phenomenon (RP) in subjects with Systemic Sclerosis (SSc).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Gesynta Pharma AB
• Collaborator: Ergomed

Criteria

Inclusion Criteria:

• Subjects must provide signed and dated written informed consent before the conduct of any study-specific procedures.

• Male and female subjects aged 18-75 years inclusive.

• Systemic Sclerosis diagnosed according to European League Against Rheumatism (EULAR)/American College of Rheumatology (ACR) criteria (van den Hoogen F et al. 2013). Subjects with signs of other autoimmune diseases (e.g. Sjögren's syndrome, myositis, rheumatoid arthritis) could be included if SSc is the dominating phenotype.

• Raynaud attacks typically ≥7 times per week during the last 4 weeks prior to screening despite background medication (only allowed vasodilatory therapy is calcium channel blockers or PDE-5 inhibitors).

• Women of childbearing potential must be using a highly effective method of contraception to avoid pregnancy throughout the study and for 4 weeks after the last dose of Investigational Medicinal Product in such manner that the risk of pregnancy is minimised.

• Women must not be pregnant or breastfeeding.

• Male subjects to agree to use condom in combination with use of contraceptive methods with a failure rate of <1% to prevent pregnancy and drug exposure of a partner, and refrain from donating sperm from the first date of dosing until 3 months after last dosing of the IMP.

• Ability of subjects to participate fully in all aspects of this clinical trial.

Exclusion Criteria:

• Systemic Sclerosis disease duration of greater than 120 months from first non-Raynaud manifestation

• Current smokers or stopped smoking <3 months prior to Visit 1.

• Dose-change or initiation of vasodilating substances (calcium blockers or PDE-5 inhibitors) within 4 weeks prior to Visit 1.

• Use of iloprost or other intravenous (iv) or po prostacyclin receptor agonist within 4 weeks prior to Visit 1.

• Ongoing treatment with immunosuppressive therapies (other than mycophenolate) including, but not restricted to; cyclophosphamide, azathioprine, methotrexate, or cyclosporine, or use of those medications within 4 weeks of trial entry.

• Use of systemic corticosteroids during 4 weeks before screening and during the course of the study.

• Concurrent serious medical condition, with special attention to cardiovascular conditions, which in the opinion of the Investigator makes the subject not suitable for this study.

• Prolonged QTcF interval defined as a mean QTcF >450 msec.

• Creatinine clearance <50 mL/min (determined by Cockcroft-Gault equation) at Screening.

• Active digital ulcer (DU) within 4 weeks prior to Visit 1.

• Clinically meaningful laboratory abnormalities at Screening (Visit 1), as determined and documented by the Investigator.