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A Phase 2/3 Open-label Extension Study to Evaluate Long-Term Safety and Efficacy With VX-509 in Subjects With Rheumatoid Arthritis

Primary Purpose

Rheumatoid Arthritis

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
VX-509
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Rheumatoid Arthritis

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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

Sites / Locations

  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site
  • Vertex Investigational Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Single Arm VX-509

Arm Description

Outcomes

Primary Outcome Measures

Long-term safety and tolerability of VX-509 treatment
Measured by clinical laboratory tests
Long-term safety and tolerability of VX-509 treatment
Measured by adverse events (AEs)
Long-term safety and tolerability of VX-509 treatment
Measured by electrocardiograms (ECGs)
Long-term safety and tolerability of VX-509 treatment
Measured by vital signs

Secondary Outcome Measures

Proportion of subjects who achieve CDAI LDA (≤10) or CDAI remission (≤2.8)
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)
Change from baseline in DAS28 using CRP (4-component) (DAS28 4[CRP])
Proportion of subjects with DAS28 4(CRP) <2.6 (DAS remission)
Proportion of subjects who achieve a moderate, good, or no response according to the EULAR response criteria from baseline
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)
ACR hybrid scores
Proportion of subjects who achieve ACR20/50/70 with erythrocyte sedimentation rate (ESR) and DAS28 4(ESR) response from baseline
Proportion of subjects with DAS28 4(CRP) <3.2 (DAS LDA) from baseline
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
Change from baseline in Health Assessment Questionnaire - Disability Index (HAQ-DI)
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

Full Information

First Posted
April 10, 2013
Last Updated
October 23, 2015
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT01830985
Brief Title
A Phase 2/3 Open-label Extension Study to Evaluate Long-Term Safety and Efficacy With VX-509 in Subjects With Rheumatoid Arthritis
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
Study Type
Interventional

2. Study Status

Record Verification Date
October 2015
Overall Recruitment Status
Completed
Study Start Date
April 2013 (undefined)
Primary Completion Date
July 2014 (Actual)
Study Completion Date
July 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief 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).
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Rheumatoid Arthritis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
39 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Single Arm VX-509
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
VX-509
Intervention Description
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)
Primary Outcome Measure Information:
Title
Long-term safety and tolerability of VX-509 treatment
Description
Measured by clinical laboratory tests
Time Frame
Baseline through 104 weeks
Title
Long-term safety and tolerability of VX-509 treatment
Description
Measured by adverse events (AEs)
Time Frame
Baseline through 104 weeks
Title
Long-term safety and tolerability of VX-509 treatment
Description
Measured by electrocardiograms (ECGs)
Time Frame
Baseline through 104 weeks
Title
Long-term safety and tolerability of VX-509 treatment
Description
Measured by vital signs
Time Frame
Baseline through 104 weeks
Secondary Outcome Measure Information:
Title
Proportion of subjects who achieve CDAI LDA (≤10) or CDAI remission (≤2.8)
Time Frame
Baseline through 104 weeks
Title
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
Title
Change from baseline in DAS28 using CRP (4-component) (DAS28 4[CRP])
Time Frame
Baseline through 104 weeks
Title
Proportion of subjects with DAS28 4(CRP) <2.6 (DAS remission)
Time Frame
Baseline through 104 weeks
Title
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
Title
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
Title
ACR hybrid scores
Time Frame
Baseline through 104 weeks
Title
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
Title
Proportion of subjects with DAS28 4(CRP) <3.2 (DAS LDA) from baseline
Time Frame
Baseline through 104 weeks
Title
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
Title
Change from baseline in Health Assessment Questionnaire - Disability Index (HAQ-DI)
Time Frame
Baseline through 104 weeks
Title
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
Other Pre-specified Outcome Measures:
Title
Change in Outcome Measures in Rheumatology Clinical Trials (OMERACT) RAMRIS synovitis score, bone marrow edema (osteitis), erosion score, and joint space narrowing score by magnetic resonance imaging (MRI) in the designated hand
Time Frame
Baseline through week 12

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bradley Bloom, MD, FACR, FAAP
Organizational Affiliation
Vertex Pharmaceuticals Incorporated
Official's Role
Study Chair
Facility Information:
Facility Name
Vertex Investigational Site
City
Upland
State/Province
California
Country
United States
Facility Name
Vertex Investigational Site
City
Fort Lauderdale
State/Province
Florida
Country
United States
Facility Name
Vertex Investigational Site
City
Venice
State/Province
Florida
Country
United States
Facility Name
Vertex Investigational Site
City
West Palm Beach
State/Province
Florida
Country
United States
Facility Name
Vertex Investigational Site
City
Canton
State/Province
Georgia
Country
United States
Facility Name
Vertex Investigational Site
City
Decatur
State/Province
Georgia
Country
United States
Facility Name
Vertex Investigational Site
City
Elizabethtown
State/Province
Kentucky
Country
United States
Facility Name
Vertex Investigational Site
City
Fredrick
State/Province
Maryland
Country
United States
Facility Name
Vertex Investigational Site
City
Lincoln
State/Province
Nebraska
Country
United States
Facility Name
Vertex Investigational Site
City
Rochester
State/Province
New York
Country
United States
Facility Name
Vertex Investigational Site
City
Greensboro
State/Province
North Carolina
Country
United States
Facility Name
Vertex Investigational Site
City
Duncansville
State/Province
Pennsylvania
Country
United States
Facility Name
Vertex Investigational Site
City
Charleston
State/Province
South Carolina
Country
United States
Facility Name
Vertex Investigational Site
City
Memphis
State/Province
Tennessee
Country
United States
Facility Name
Vertex Investigational Site
City
Katy
State/Province
Texas
Country
United States
Facility Name
Vertex Investigational Site
City
San Antonio
State/Province
Texas
Country
United States
Facility Name
Vertex Investigational Site
City
Webster
State/Province
Texas
Country
United States
Facility Name
Vertex Investigational Site
City
Spokane
State/Province
Washington
Country
United States
Facility Name
Vertex Investigational Site
City
Tallinn
Country
Estonia
Facility Name
Vertex Investigational Site
City
Vilnius
Country
Lithuania
Facility Name
Vertex Investigational Site
City
Pretoria
Country
South Africa
Facility Name
Vertex Investigational Site
City
Stellenbosch
Country
South Africa

12. IPD Sharing Statement

Learn more about this trial

A Phase 2/3 Open-label Extension Study to Evaluate Long-Term Safety and Efficacy With VX-509 in Subjects With Rheumatoid Arthritis

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