Systemic SclerosisPhase 2NCT04744207
A Study to Investigate Safety of GS-248 and Efficacy on Raynauds' Phenomenon in Systemic Sclerosis
A Phase 2 study testing GS-248 in people with Systemic Sclerosis, run by Gesynta Pharma AB at 12 sites in 4 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Systemic Sclerosis
- What is being tested?
- GS-248
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 94 participants
- Where
- 12 sitesin 4 countries
- Study dates
- 29 Dec 2020to 15 Jun 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
12 sites in 4 countries.
12 sites in 4 countries
United Kingdom7 sites
Investigator site
Bath
Investigator site
Cambridge
Investigator Site
Dundee
Investigator site
Leeds
Investigator Site
Liverpool
Investigator Site
London
Investigator site
Manchester
Poland3 sites
Investigator site
Gdansk
Investigator Site
Krakow
Investigator site
Lublin
About this study
Official 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)
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).
Read the detailed description
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).
This is a randomized, double-blind, placebo-controlled study conducted in multiple sites in 4 countries in Europe. Approximately 80 subjects will be randomized in a 1:1 allocation to receive either GS-248 (120 mg) or placebo once daily. The study will comprise an enrolment period, a treatment period, and a follow-up period, with a total of 5 study visits over approximately 10 weeks.
- Conditions
- Systemic Sclerosis
- Being tested
- GS-248, Placebo
- Medical subject headings
- Scleroderma, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GS-248Drug
120 mg, capsule, once daily for 4 weeks
PlaceboDrug
capsule, once daily for 4 weeks
| Study group | Role | What they receive |
|---|---|---|
| GS-248 | Experimental | GS-248, capsule, 120 mg, once daily for 4 weeks Receives: Drug: GS-248 |
| Placebo | Placebo comparator | placebo, capsule, once daily for 4 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Patients reported Raynaud's Condition Score (RCS) once a day in an electronic diary.
RCS is a validated numeric rating scale (from 0 to 10) answering the question "What difficulty did you have today with your Raynaud's condition?" where a score of '0' = 'No difficulty', and a score of '10' = 'Extreme difficulty'.
Measured: 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.
The patient reported the experienced pain of each Raynaud attack using a Numeric Rating Scale (NRS) from 0 to 10 in an electronic diary where '0'='No pain' and '10'='Worst imaginable pain'.
Measured: 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
The patient reported the start time (hh:mm) and stop time (hh:mm) of each Raynaud's attack in the electronic diary.
Measured: 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.
The patient reported the start time (hh:mm) and stop time (hh:mm) of each Raynaud's attack in the electronic diary.
Measured: From baseline to week 4, i.e. the 7 most recent days prior to Visit 2 and Visit 4 respectively
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Feb 2021
- Study start29 Dec 2020
- Primary completion15 Jun 2022
- Study completion15 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Systemic SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 94 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gesynta Pharma AB
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Ergomed
- Responsible party
- Sponsor
- Investigators
- Charlotte Edenius, Study director, Gesynta Pharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04744207
- Sponsor's protocol ID
- GS-2001
- Status last verified
- Mar 2024
- First submitted
- 28 Jan 2021
- Last updated
- 9 Aug 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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)
- ClinicalTrials.gov ID
- NCT04744207
- Acronym / sponsor ID
- GS-2001
- Conditions
- Systemic Sclerosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 94 participants (actual)
- Lead sponsor
- Gesynta Pharma ABIndustry (pharma, biotech, devices)
- Collaborators
- Ergomed
- Study start
- 29 Dec 2020 (actual)
- Primary completion
- 15 Jun 2022 (actual)
- Study completion
- 15 Jun 2022 (actual)
- First posted
- 8 Feb 2021 (actual)
- Results
- Posted 9 Aug 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
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).
This is a randomized, double-blind, placebo-controlled study conducted in multiple sites in 4 countries in Europe. Approximately 80 subjects will be randomized in a 1:1 allocation to receive either GS-248 (120 mg) or placebo once daily. The study will comprise an enrolment period, a treatment period, and a follow-up period, with a total of 5 study visits over approximately 10 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| GS-248 | Experimental | Drug: GS-248GS-248, capsule, 120 mg, once daily for 4 weeks |
| Placebo | Placebo comparator | Drug: Placeboplacebo, capsule, once daily for 4 weeks |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GS-248120 mg, capsule, once daily for 4 weeks | Drug | GS-248 |
| Placebocapsule, once daily for 4 weeks | Drug | Placebo |
Primary outcome
Mean Change From Baseline to Week 4 in the Number of Raynaud Attacks Per Week.
Time frame: From baseline to week 4, i.e. the 7 most recent days prior to Visit 2 and Visit 4 respectively
Patient reported number of Raynaud's attacks per day as registered in electronic diary.
4 secondary outcomes
Mean Change From Baseline to Week 4 in the Raynaud's Condition Score.
Time frame: 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.
Time frame: 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
Time frame: 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.
Time frame: From baseline to week 4, i.e. the 7 most recent days prior to Visit 2 and Visit 4 respectively
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Aug 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Charlotte Edenius study director
Gesynta Pharma
12 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GS-248 in people with Systemic Sclerosis, run by Gesynta Pharma AB at 12 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United Kingdom, Poland, Belgium, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean Change From Baseline to Week 4 in the Number of Raynaud Attacks Per Week. (time frame: From baseline to week 4, i.e. the 7 most recent days prior to Visit 2 and Visit 4 respectively). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Aug 2024.