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Phase 2 N=69 Randomized Triple-blind Treatment

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

Systemic Sclerosis

Enrolled (actual)
69
Serious AEs
1.5%
Results posted
Aug 2024
Primary outcome: Primary: Mean Change From Baseline to Week 4 in the Number of Raynaud Attacks Per Week. — -3.41; -4.22 number of attacks — p=<0.05

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
GS-248 (Drug); Placebo (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Gesynta Pharma AB
Primary completion
Jun 2022

Outcome Measures

OutcomeResultp-value
PRIMARY
Mean Change From Baseline to Week 4 in the Number of Raynaud Attacks Per Week.
-3.41; -4.22 <0.05 sig
SECONDARY
Mean Change From Baseline to Week 4 in the Raynaud's Condition Score.
-0.99; -0.95
SECONDARY
Mean Change From Baseline to Week 4 in Pain Experienced During Raynaud Attacks.
-0.65; -0.60
SECONDARY
Mean Change From Baseline to Week 4 in the Mean Duration of Raynaud's Attacks
1.06; 0.89
SECONDARY
Mean Change From Baseline to Week 4 in the Cumulative Duration of Raynaud Attacks.
0.70; 0.61

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).

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 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.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04744207). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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