GS-2001

• Research type

  Research Study

• Full title

  A Phase II, randomized, multi-center, placebo-controlled, double-blind study to investigate the safety of GS-248, and efficacy on Raynaud’s phenomenon (RP) and peripheral vascular blood flow, in subjects with systemic sclerosis (SSc)

• IRAS ID

  283532

• Contact name

  Ariane Herrick

• Contact email

  ariane.herrick@manchester.ac.uk

• Sponsor organisation

  Gesynta Pharma AB

• Eudract number

  2020-002081-13

• Clinicaltrials.gov Identifier

  NCT04744207

• Duration of Study in the UK

  0 years, 7 months, 0 days

• Research summary

  Summary of Research

  The study is being performed to determine the safety and clinical efficacy of the study drug (GS-248) on patients with a rare autoimmune disease called Systemic Sclerosis (SSc) who suffer with attacks of Raynaud's phenomenon (RP). This phenomenon affects blood circulation, causing recurrent episodes of restriction of blood flow in the fingers and toes in response to cold exposure or emotional stress.
  For this patient population, attacks of Raynaud's phenomenom are frequent and painful and can lead to ulcers on fingers and toes, result in patients making life adjustments and affect their quality of life. Current treatments are unsatisfactory in a large proportion of patients and therefore this study could be important for improving the quality of life of these patients.

  The study drug is an inhibitor of an enzyme involved in inflammatory processes.
  This study will include 80 male and female, adult participants with systemic sclerosis. Participants will be in the study for approximately 10 weeks and will be randomised in a 1:1 allocation to active treatment or placebo. The participants will take the study medication once a day for 28 days. There are 5 visits and participants will undergo a series of tests and procedures at each visit, including blood sampling, thermal imaging and questionnaires. Participants will also be required to complete a diary to record their Raynaud's attacks.
  The study is taking place at approximately 10 NHS hospital sites in the UK

  Summary of Results

  The study medicine (GS-248) was safe and well-tolerated in patients with Systemic Sclerosis (a chronic inflammatory disease) who suffer with Raynauds’ Phenomenon when they were given 120mg by mouth once a day for 28 days. The results for how well GS-248 works showed that GS-248 was no better than the dummy medicine (placebo) in reducing the number of Raynaud attacks per week, other Raynaud’s symptoms or recovery of blood flow in the fingers after exposure to cold temperatures.

• REC name

  East of England - Essex Research Ethics Committee

• REC reference

  20/EE/0168

• Date of REC Opinion

  30 Jul 2020

• REC opinion

  Further Information Favourable Opinion