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

NCT04744207

CompletedPhase 2

Sponsor: Gesynta Pharma AB

Conditions: Systemic Sclerosis

Interventions: GS-248, Placebo

Countries: Belgium, Netherlands, Poland, United Kingdom

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).

Eligibility overview

Sex: ALL

Age: 18 Years to 75 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility 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.
Locations (12)
  • Ghent, Belgium
  • Nijmegen, Netherlands
  • Gdansk, Poland
  • Krakow, Poland
  • Lublin, Poland
  • Bath, United Kingdom
  • Cambridge, United Kingdom
  • Dundee, United Kingdom
  • Leeds, United Kingdom
  • Liverpool, United Kingdom
  • London, United Kingdom
  • Manchester, United Kingdom