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

NCT04744207 | Completed Phase 2 Results

• 1 other identifier: GS-2001
• Study Type: interventional
• Target: 94
• Locations: 4 countries
• Sites: 12

Brief 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).

Conditions

Systemic Sclerosis

Outcome Measures

Primary Outcomes (1)

• Mean Change From Baseline to Week 4 in the Number of Raynaud Attacks Per Week.

  Patient reported number of Raynaud's attacks per day as registered in electronic diary.

  From baseline to week 4, i.e. the 7 most recent days prior to Visit 2 and Visit 4 respectively

Secondary Outcomes (4)

• Mean Change From Baseline to Week 4 in the Raynaud's Condition Score.

  From baseline to week 4, i.e. the 7 most recent days prior to Visit 2 and Visit 4 respectively

• Mean Change From Baseline to Week 4 in Pain Experienced During Raynaud Attacks.

  From baseline to week 4, i.e. the 7 most recent days prior to Visit 2 and Visit 4 respectively

• Mean Change From Baseline to Week 4 in the Mean Duration of Raynaud's Attacks

  From baseline to week 4, i.e. the 7 most recent days prior to Visit 2 and Visit 4 respectively

• Mean Change From Baseline to Week 4 in the Cumulative Duration of Raynaud Attacks.

  From baseline to week 4, i.e. the 7 most recent days prior to Visit 2 and Visit 4 respectively

Study Arms (2)

GS-248

EXPERIMENTAL

GS-248, capsule, 120 mg, once daily for 4 weeks

Drug: GS-248

Placebo

PLACEBO COMPARATOR

placebo, capsule, once daily for 4 weeks

Drug: Placebo

Interventions

GS-248 DRUG

120 mg, capsule, once daily for 4 weeks

GS-248

Placebo DRUG

capsule, once daily for 4 weeks

Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Gesynta Pharma AB: lead
• Ergomed: collaborator

Study Sites (12)

Investigator site

Ghent, Belgium

Location

Investigator Site

Nijmegen, Netherlands

Location

Investigator site

Gdansk, Poland

Location

Investigator Site

Krakow, Poland

Location

Investigator site

Lublin, Poland

Location

Investigator site

Bath, United Kingdom

Location

Investigator site

Cambridge, United Kingdom

Location

Investigator Site

Dundee, United Kingdom

Location

Investigator site

Leeds, United Kingdom

Location

Investigator Site

Liverpool, United Kingdom

Location

Investigator Site

London, United Kingdom

Location

Investigator site

Manchester, United Kingdom

Location

MeSH Terms

Conditions

Scleroderma, Systemic

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Results Point of Contact

• Title: Study Director
• Organization: Gesynta Pharma AB

ctg@gesynta.se

+46 73 386 4246

Study Officials

• Charlotte Edenius

  Gesynta Pharma

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 28, 2021
• First Posted: February 8, 2021
• Study Start: December 29, 2020
• Primary Completion: June 15, 2022
• Study Completion: June 15, 2022
• Last Updated: August 9, 2024
• Results First Posted: August 9, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

GB (7); PL (3); NL (1); BE (1)