Rheumatoid ArthritisPhase 2/3NCT01830985
A Phase 2/3 Open-label Extension Study to Evaluate Long-Term Safety and Efficacy With VX-509 in Subjects With Rheumatoid Arthritis
A Phase 2/3 study testing VX-509 in people with Rheumatoid Arthritis, run by Vertex Pharmaceuticals Incorporated at 22 sites in 4 countries.
Trial summary
This study is designed to evaluate the long-term safety and tolerability of VX-509 in subjects with active rheumatoid arthritis (RA) on DMARD therapy. This study will enroll subjects who completed a previous designated study with VX-509 (e.g., Study VX12-509-103). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Rheumatoid Arthritis
- What is being tested?
- VX-509
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 39 participants
- Where
- 22 sitesin 4 countries
- Study dates
- Apr 2013to Jul 2014
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects must have completed the assigned study drug treatment phase of a previous VX-509 study (e.g., Study 103).
- Subjects must voluntarily sign and date the Study 104 informed consent document.
- Subject must be willing and able to comply with the scheduled visits, treatment plan, laboratory tests, contraceptive guidelines, and other study procedures.
Who cannot join
- Inflammatory and rheumatological disorders other than RA, where arthritis may be a prominent feature.
- History of any clinically significant illness that might, in the opinion of the investigator, confound the results of the study or pose an additional risk in administering study drug(s) to the subject
- History of tuberculosis (TB), regardless of history of antimycobacterial treatment.
- Planned surgery during the study.
- History of alcohol or drug abuse, or excessive alcohol consumption as determined by the investigator, during the previous 12 months before Day 1.
- Pregnant or nursing an infant or with a life partner who is pregnant, nursing, or planning to become pregnant
Trial locations and contacts
22 sites in 4 countries.
22 sites in 4 countries
United States18 sites
Vertex Investigational Site
Canton, Georgia
Vertex Investigational Site
Charleston, South Carolina
Vertex Investigational Site
Decatur, Georgia
Vertex Investigational Site
Duncansville, Pennsylvania
Vertex Investigational Site
Elizabethtown, Kentucky
Vertex Investigational Site
Fort Lauderdale, Florida
Vertex Investigational Site
Fredrick, Maryland
Vertex Investigational Site
Greensboro, North Carolina
Vertex Investigational Site
Katy, Texas
Vertex Investigational Site
Lincoln, Nebraska
Vertex Investigational Site
Memphis, Tennessee
Vertex Investigational Site
Rochester, New York
Vertex Investigational Site
San Antonio, Texas
Vertex Investigational Site
Spokane, Washington
Vertex Investigational Site
Upland, California
Vertex Investigational Site
Venice, Florida
Vertex Investigational Site
Webster, Texas
Vertex Investigational Site
West Palm Beach, Florida
South Africa2 sites
Vertex Investigational Site
Pretoria
Vertex Investigational Site
Stellenbosch
About this study
Official title: A Phase 2/3 Open-label Extension Study to Evaluate Long-Term Safety and Efficacy With VX-509 in a Treat to Target Setting in Subjects With Rheumatoid Arthritis on Disease-Modifying Antirheumatic Drugs
This study is designed to evaluate the long-term safety and tolerability of VX-509 in subjects with active rheumatoid arthritis (RA) on DMARD therapy. This study will enroll subjects who completed a previous designated study with VX-509 (e.g., Study VX12-509-103).
Read the detailed description
VX-509 is an oral, selective Janus kinase 3 (JAK3) inhibitor being developed by Vertex. In autoimmune diseases, JAK3 is an essential component of the immune signaling cascade. This cascade ultimately contributes to abnormal immune response that results in chronic inflammation and, in the case of rheumatoid arthritis (RA), irreversible damage to cartilage and bones. Selective inhibition of JAK3 offers a new disease modifying approach to the treatment of RA.
This study will follow a "treat to target" (T2T) paradigm. T2T strategies have been followed in non-rheumatologic fields for decades. T2T trials have been conducted for RA from the late 1990's, and have substantiated the concept that treating to a target is associated with a better outcome than standard of care treatment. This has led to recommendations by experts to use T2T strategies in clinical practice.
- Conditions
- Rheumatoid Arthritis
- Being tested
- VX-509
- Medical subject headings
- Arthritis, Rheumatoid
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VX-509Drug
VX-509 dose may be increased every 8 weeks in a stepwise fashion from 100 to 150 mg and from 150 to 200 mg, as needed (determined by ongoing disease activity by CDAI)
| Study group | Role | What they receive |
|---|---|---|
| Single Arm VX-509 | Experimental | Not described Receives: Drug: VX-509 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Long-term safety and tolerability of VX-509 treatment
Measured by clinical laboratory tests
Measured: Baseline through 104 weeks
Long-term safety and tolerability of VX-509 treatment
Measured by adverse events (AEs)
Measured: Baseline through 104 weeks
Long-term safety and tolerability of VX-509 treatment
Measured by electrocardiograms (ECGs)
Measured: Baseline through 104 weeks
Long-term safety and tolerability of VX-509 treatment
Measured by vital signs
Measured: Baseline through 104 weeks
Secondary outcomes (12)
Proportion of subjects who achieve CDAI LDA (≤10) or CDAI remission (≤2.8)
Measured: Baseline through 104 weeks
Proportion of subjects who achieve ≥20% (50%, 70%) improvement in disease severity according to the ACR criteria, using CRP (ACR20 CRP, ACR50 CRP, ACR70 CRP)
Measured: Baseline through 104 weeks
Change from baseline in DAS28 using CRP (4-component) (DAS28 4[CRP])
Measured: Baseline through 104 weeks
Proportion of subjects with DAS28 4(CRP) <2.6 (DAS remission)
Measured: Baseline through 104 weeks
Proportion of subjects who achieve a moderate, good, or no response according to the EULAR response criteria from baseline
Measured: Baseline through 104 weeks
Percentage of subjects with decreased dose of DMARD and/or corticosteroid (if receiving), including the subsets with 50% withdrawal and with full withdrawal (dose = 0)
Measured: Baseline through 104 weeks
ACR hybrid scores
Measured: Baseline through 104 weeks
Proportion of subjects who achieve ACR20/50/70 with erythrocyte sedimentation rate (ESR) and DAS28 4(ESR) response from baseline
Measured: Baseline through 104 weeks
Proportion of subjects with DAS28 4(CRP) <3.2 (DAS LDA) from baseline
Measured: Baseline through 104 weeks
Proportion of subjects achieving a clinical remission (2011 ACR/EULAR criteria), including subsets achieving either the low joint count or simplified disease activity index (SDAI) score remission options (or both) from baseline
Measured: Baseline through week 104
Change from baseline in Health Assessment Questionnaire - Disability Index (HAQ-DI)
Measured: Baseline through 104 weeks
Change from baseline in health-related quality of life assessed by 36-Item Short Form (SF 36) Physical Component Summary score and Physical Function (PF) subscale
Measured: Baseline through 104 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Apr 2013 (estimated)
- Study startApr 2013
- Primary completionJul 2014
- Study completionJul 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rheumatoid ArthritisStudies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- 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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bradley Bloom, MD, FACR, FAAP, Study chair, Vertex Pharmaceuticals Incorporated
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01830985
- Sponsor's protocol ID
- VX12-509-104
- Other IDs
- 2012-004342-14
- Status last verified
- Oct 2015
- First submitted
- 10 Apr 2013
- Last updated
- 20 Nov 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2/3 Open-label Extension Study to Evaluate Long-Term Safety and Efficacy With VX-509 in a Treat to Target Setting in Subjects With Rheumatoid Arthritis on Disease-Modifying Antirheumatic Drugs
- ClinicalTrials.gov ID
- NCT01830985
- Acronym / sponsor ID
- VX12-509-104
- Conditions
- Rheumatoid Arthritis
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- Apr 2013
- Primary completion
- Jul 2014 (actual)
- Study completion
- Jul 2014 (actual)
- First posted
- 12 Apr 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is designed to evaluate the long-term safety and tolerability of VX-509 in subjects with active rheumatoid arthritis (RA) on DMARD therapy. This study will enroll subjects who completed a previous designated study with VX-509 (e.g., Study VX12-509-103).
Detailed description
VX-509 is an oral, selective Janus kinase 3 (JAK3) inhibitor being developed by Vertex. In autoimmune diseases, JAK3 is an essential component of the immune signaling cascade. This cascade ultimately contributes to abnormal immune response that results in chronic inflammation and, in the case of rheumatoid arthritis (RA), irreversible damage to cartilage and bones. Selective inhibition of JAK3 offers a new disease modifying approach to the treatment of RA.
This study will follow a "treat to target" (T2T) paradigm. T2T strategies have been followed in non-rheumatologic fields for decades. T2T trials have been conducted for RA from the late 1990's, and have substantiated the concept that treating to a target is associated with a better outcome than standard of care treatment. This has led to recommendations by experts to use T2T strategies in clinical practice.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm VX-509 | Experimental | Drug: VX-509 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Subjects must have completed the assigned study drug treatment phase of a previous VX-509 study (e.g., Study 103).
- Subjects must voluntarily sign and date the Study 104 informed consent document.
- Subject must be willing and able to comply with the scheduled visits, treatment plan, laboratory tests, contraceptive guidelines, and other study procedures.
Key exclusion criteria (6 listed)
- Inflammatory and rheumatological disorders other than RA, where arthritis may be a prominent feature.
- History of any clinically significant illness that might, in the opinion of the investigator, confound the results of the study or pose an additional risk in administering study drug(s) to the subject
- History of tuberculosis (TB), regardless of history of antimycobacterial treatment.
- Planned surgery during the study.
- History of alcohol or drug abuse, or excessive alcohol consumption as determined by the investigator, during the previous 12 months before Day 1.
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 |
|---|---|---|
| VX-509VX-509 dose may be increased every 8 weeks in a stepwise fashion from 100 to 150 mg and from 150 to 200 mg, as needed (determined by ongoing disease activity by CDAI) | Drug | Single Arm VX-509 |
Primary outcome
Long-term safety and tolerability of VX-509 treatment
Time frame: Baseline through 104 weeks
Measured by clinical laboratory tests
Long-term safety and tolerability of VX-509 treatment
Time frame: Baseline through 104 weeks
Measured by adverse events (AEs)
Long-term safety and tolerability of VX-509 treatment
Time frame: Baseline through 104 weeks
Measured by electrocardiograms (ECGs)
Long-term safety and tolerability of VX-509 treatment
Time frame: Baseline through 104 weeks
Measured by vital signs
12 secondary outcomes
Proportion of subjects who achieve CDAI LDA (≤10) or CDAI remission (≤2.8)
Time frame: Baseline through 104 weeks
Proportion of subjects who achieve ≥20% (50%, 70%) improvement in disease severity according to the ACR criteria, using CRP (ACR20 CRP, ACR50 CRP, ACR70 CRP)
Time frame: Baseline through 104 weeks
Change from baseline in DAS28 using CRP (4-component) (DAS28 4[CRP])
Time frame: Baseline through 104 weeks
Proportion of subjects with DAS28 4(CRP) <2.6 (DAS remission)
Time frame: Baseline through 104 weeks
Proportion of subjects who achieve a moderate, good, or no response according to the EULAR response criteria from baseline
Time frame: Baseline through 104 weeks
Percentage of subjects with decreased dose of DMARD and/or corticosteroid (if receiving), including the subsets with 50% withdrawal and with full withdrawal (dose = 0)
Time frame: Baseline through 104 weeks
ACR hybrid scores
Time frame: Baseline through 104 weeks
Proportion of subjects who achieve ACR20/50/70 with erythrocyte sedimentation rate (ESR) and DAS28 4(ESR) response from baseline
Time frame: Baseline through 104 weeks
Proportion of subjects with DAS28 4(CRP) <3.2 (DAS LDA) from baseline
Time frame: Baseline through 104 weeks
Proportion of subjects achieving a clinical remission (2011 ACR/EULAR criteria), including subsets achieving either the low joint count or simplified disease activity index (SDAI) score remission options (or both) from baseline
Time frame: Baseline through week 104
Change from baseline in Health Assessment Questionnaire - Disability Index (HAQ-DI)
Time frame: Baseline through 104 weeks
Change from baseline in health-related quality of life assessed by 36-Item Short Form (SF 36) Physical Component Summary score and Physical Function (PF) subscale
Time frame: Baseline through 104 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 2015.
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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.
Bradley Bloom, MD, FACR, FAAP study chair
Vertex Pharmaceuticals Incorporated
22 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing VX-509 in people with Rheumatoid Arthritis, run by Vertex Pharmaceuticals Incorporated at 22 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 3 inclusion and 6 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 20 Nov 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States, South Africa, Estonia, Lithuania. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Long-term safety and tolerability of VX-509 treatment (time frame: Baseline through 104 weeks). The study lists 4 primary outcomes in all. 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 20 Nov 2015.