Juvenile Idiopathic ArthritisPhase 2NCT00534495
Safety and Effectiveness of Rilonacept for Treating Systemic Juvenile Idiopathic Arthritis in Children and Young Adults
A Phase 2 study testing Rilonacept in people with Juvenile Idiopathic Arthritis, run by Montefiore Medical Center at 1 site in United States.
Trial summary
Systemic juvenile idiopathic arthritis (SJIA) is a type of arthritis that typically occurs before 16 years of age. SJIA usually involves heat, pain, swelling, and stiffness in the body's joints. It can also involve fever, rash, anemia, and inflammation in various parts of the body. Read more about this study
- Who is it for?
- Ages 18 months to 19 years
- Conditions the study is for
- Juvenile Idiopathic Arthritis
- What is being tested?
- Rilonacept
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 71 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2008to Jun 2013
- 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 months to 19 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Fulfills International League Against Rheumatism (ILAR) criteria for SJIA
- Duration of SJIA lasting at least 6 weeks since onset
- Active disease as defined by at least two joints with active disease
- Not currently receiving methotrexate OR if taking methotrexate, the dose has remained stable or has been discontinued for 4 weeks prior to screening
- Has never received certain biologics OR if previously received biologics, discontinued etanercept for at least 4 weeks prior to screening and discontinued infliximab or adalimumab for at least 8 weeks prior to screening
- Not currently receiving corticosteroids OR if taking oral corticosteroids, the dose has remained stable between 2 and 60 mg/day for at least 2 weeks prior to screening
Who cannot join
- Past treatment with anakinra, rilonacept, or other biologic IL-1 inhibitor
- Treatment with other disease-modifying antirheumatic drugs (DMARDs) including, but not limited to, azathioprine, sulfasalazine, cyclosporine, and thalidomide within 4 weeks of screening
- Treatment with leflunomide without cholestyramine washout at the end of therapy
- Treatment with cyclophosphamide within 3 months of study entry
- Treatment with tacrolimus or tocilizumab within 4 weeks of study entry
- Treatment with rituximab within 6 months of study entry
Show all 19Show fewer
- Treatment with intravenous immunoglobulin (IVIG) within 4 weeks of screening
- Kidney disease
- AST or ALT levels more than two times the upper limit of normal
- Bilirubin levels higher than 1.5 mg/dl
- Thrombocytopenia, leukopenia, or neutropenia
- Abnormal prothrombin time (PT) and partial thromboplastin time (PTT) tests
- Low levels of plasma fibrinogen
- Evidence of chronic recurrent infection or other significant, non-SJIA illness that might interfere with study participation
- Psychological or cognitive difficulties that might interfere with study participation
- Current drug or alcohol abuse
- Anticipated poor compliance to assigned study regimen
- Participation in another clinical trial within 30 days of study entry
- Major surgical procedure within 3 months of study entry
Trial locations and contacts
1 site in 1 country.
United States1 site
Montefiore Medical Center
The Bronx, New York, 10467
About this study
Official title: Randomized Placebo Phase Study of Rilonacept in the Treatment of Systemic Juvenile Idiopathic Arthritis (RAPPORT)
Systemic juvenile idiopathic arthritis (SJIA) is a type of arthritis that typically occurs before 16 years of age. SJIA usually involves heat, pain, swelling, and stiffness in the body's joints. It can also involve fever, rash, anemia, and inflammation in various parts of the body. Rilonacept is a drug that can reduce inflammation. The purpose of this study is to determine whether a rilonacept drug regimen initiated early is more effective than a similar rilonacept drug regimen initiated 4 weeks later when treating children and young adults with SJIA.
Read the detailed description
The current standard treatment for SJIA includes nonsteroidal anti-inflammatory drugs (NSAIDS) and corticosteroids. However, in most people, NSAIDS do not completely control the disease. Also, no studies have been done to prove which medication or combination of medications is best to treat children and adolescents with SJIA. Interleukin-1 (IL-1), a protein secreted by certain cells in the body, assists in regulating immune and inflammatory responses. Too much IL-1 can be harmful and has been shown to play a role in the inflammation associated with a variety of diseases, including SJIA. Rilonacept is a drug that inhibits IL-1 activity. The purpose of this study is to determine whether a rilonacept drug regimen initiated early is more effective than a similar rilonacept drug regimen initiated 4 weeks later when treating children and young adults with SJIA. This study will also evaluate the safety of rilonacept, and various tissue samples will be collected from participants for future genetic studies.
This study will last 6 months. Participants will be randomly assigned to one of two groups:
- Group 1 participants will receive rilonacept injections at a dose of 4.4mg/kg at study entry (loading dose), then 2.2 mg/kg weekly until Week 4. At Week 4, they will receive a loading dose of placebo, followed by weekly rilonacept injections at 2.2 mg/kg for the duration of the study.
- Group 2 participants will receive placebo at study entry and then during the first 4 weeks of treatment. At Week 4, they will receive a loading dose of rilonacept injections of 4.4 mg/kg, followed by weekly rilonacept injections at a dose of 2.2 mg/kg for the duration of the study.
Participants will continue any previous corticosteroid therapy, but in tapering doses. All participants will attend study visits at Weeks 0, 2, 4, 6, 8, 10, 12, 14 and 24. Study visits will include a physical exam, joint exam, blood collection, interview, and questionnaires. Urine collection may occur for some female participants. Other evaluations may be performed by the participant's regular doctor. Throughout the study, participants will maintain at-home diaries to record fever, morning stiffness and pain, when rilonacept or placebo was taken, any side effects experienced from treatment, and any additional medications that were taken.
- Conditions
- Juvenile Idiopathic Arthritis
- Being tested
- Rilonacept
- Keywords
- Systemic Juvenile Idiopathic Arthritis, Juvenile Rheumatoid Arthritis, Systemic Juvenile Rheumatoid Arthritis
- Medical subject headings
- Arthritis, Juvenile, Rheumatoid Arthritis, Systemic Juvenile
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RilonaceptBiological
Also called IL-1 Trap, Arcalyst
2.2 mg/kg subcutaneously
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Experimental | Loading dose of rilonacept (4.4mg/kg) at Week 0, followed by rilonacept 2.2 mg/kg/week for the remainder of the study Receives: Biological: Rilonacept |
| Group 2 | Placebo comparator | Placebo for 4 weeks, followed by rilonacept loading dose (4.4mg/kg), followed by rilonacept 2.2 mg/kg/week for the remainder of the study Receives: Biological: Rilonacept |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to Response to Treatment, as Determined by a Modified JIA ACR30 Requiring no Fever, Coupled With a Requirement for Corticosteroid Taper in Participants Who Are Taking Corticosteroids
Measured: At Week 12
Number of Serious Adverse Events,Adverse Events, Infections, Development of MAS
Measured: At Weeks 0- 24
Secondary outcomes (4)
Number of Participants With Response as Determined by JIA ACR50 and JIA ACR70
Measured: At Week 4 and week 12
Pediatric Quality of Life Inventory
Visual Analog Score (0-100 mm) 0 very well , 100 very poor
Measured: At Weeks 4, 12 and 24
Physical Function as Determined by Childhood Health Assessment Questionnaire ( CHAQ)
Childhood Health Assesment Questionairre dissability index (C-HAQ)-DI, Disability Index Calculation:
The index is calculated by adding the scores for each of the categories and dividing by the number of categories answered. This gives a score in the 0 to 3.0 range. lower is better
Measured: At Weeks 12 and 24
Number of Participants With Presence of Systemic Features ( Fever, Rash)
Measured: At Weeks 4, 12 and 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Sep 2007 (estimated)
- Study startNov 2008
- Primary completionJun 2013
- Study completionJun 2013
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 Juvenile Idiopathic ArthritisStudies of the same condition, open and under way.
- More trials run by Montefiore Medical CenterThe 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 71 participants (actual)
Sponsor and investigators
- Lead sponsor
- Montefiore Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- Responsible party
- Principal investigator, Norman Ilowite, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- Investigators
- Norman T. Ilowite, MD, Principal investigator, Montefiore Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00534495
- Sponsor's protocol ID
- N01 AR070015
- Other IDs
- 268200700015C-2-0-0, HHSN2682007000015C
- Status last verified
- Nov 2015
- First submitted
- 24 Sep 2007
- Last updated
- 11 Dec 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized Placebo Phase Study of Rilonacept in the Treatment of Systemic Juvenile Idiopathic Arthritis (RAPPORT)
- ClinicalTrials.gov ID
- NCT00534495
- Acronym / sponsor ID
- N01 AR070015
- Conditions
- Juvenile Idiopathic Arthritis
- Keywords
- Systemic Juvenile Idiopathic Arthritis, Juvenile Rheumatoid Arthritis, Systemic Juvenile Rheumatoid Arthritis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 71 participants (actual)
- Lead sponsor
- Montefiore Medical CenterUniversity, hospital or other
- Collaborators
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- Study start
- Nov 2008
- Primary completion
- Jun 2013 (actual)
- Study completion
- Jun 2013 (actual)
- First posted
- 26 Sep 2007 (estimated)
- Results
- Posted 11 Dec 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Systemic juvenile idiopathic arthritis (SJIA) is a type of arthritis that typically occurs before 16 years of age. SJIA usually involves heat, pain, swelling, and stiffness in the body's joints. It can also involve fever, rash, anemia, and inflammation in various parts of the body. Rilonacept is a drug that can reduce inflammation. The purpose of this study is to determine whether a rilonacept drug regimen initiated early is more effective than a similar rilonacept drug regimen initiated 4 weeks later when treating children and young adults with SJIA.
Detailed description
The current standard treatment for SJIA includes nonsteroidal anti-inflammatory drugs (NSAIDS) and corticosteroids. However, in most people, NSAIDS do not completely control the disease. Also, no studies have been done to prove which medication or combination of medications is best to treat children and adolescents with SJIA. Interleukin-1 (IL-1), a protein secreted by certain cells in the body, assists in regulating immune and inflammatory responses. Too much IL-1 can be harmful and has been shown to play a role in the inflammation associated with a variety of diseases, including SJIA. Rilonacept is a drug that inhibits IL-1 activity. The purpose of this study is to determine whether a rilonacept drug regimen initiated early is more effective than a similar rilonacept drug regimen initiated 4 weeks later when treating children and young adults with SJIA. This study will also evaluate the safety of rilonacept, and various tissue samples will be collected from participants for future genetic studies.
This study will last 6 months. Participants will be randomly assigned to one of two groups:
- Group 1 participants will receive rilonacept injections at a dose of 4.4mg/kg at study entry (loading dose), then 2.2 mg/kg weekly until Week 4. At Week 4, they will receive a loading dose of placebo, followed by weekly rilonacept injections at 2.2 mg/kg for the duration of the study.
- Group 2 participants will receive placebo at study entry and then during the first 4 weeks of treatment. At Week 4, they will receive a loading dose of rilonacept injections of 4.4 mg/kg, followed by weekly rilonacept injections at a dose of 2.2 mg/kg for the duration of the study.
Participants will continue any previous corticosteroid therapy, but in tapering doses. All participants will attend study visits at Weeks 0, 2, 4, 6, 8, 10, 12, 14 and 24. Study visits will include a physical exam, joint exam, blood collection, interview, and questionnaires. Urine collection may occur for some female participants. Other evaluations may be performed by the participant's regular doctor. Throughout the study, participants will maintain at-home diaries to record fever, morning stiffness and pain, when rilonacept or placebo was taken, any side effects experienced from treatment, and any additional medications that were taken.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1 | Experimental | Biological: RilonaceptLoading dose of rilonacept (4.4mg/kg) at Week 0, followed by rilonacept 2.2 mg/kg/week for the remainder of the study |
| Group 2 | Placebo comparator | Biological: RilonaceptPlacebo for 4 weeks, followed by rilonacept loading dose (4.4mg/kg), followed by rilonacept 2.2 mg/kg/week for the remainder of the study |
Who may qualify
- Ages
- 18 months to 19 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Fulfills International League Against Rheumatism (ILAR) criteria for SJIA
- Duration of SJIA lasting at least 6 weeks since onset
- Active disease as defined by at least two joints with active disease
- Not currently receiving methotrexate OR if taking methotrexate, the dose has remained stable or has been discontinued for 4 weeks prior to screening
- Has never received certain biologics OR if previously received biologics, discontinued etanercept for at least 4 weeks prior to screening and discontinued infliximab or adalimumab for at least 8 weeks prior to screening
Key exclusion criteria (19 listed)
- Past treatment with anakinra, rilonacept, or other biologic IL-1 inhibitor
- Treatment with other disease-modifying antirheumatic drugs (DMARDs) including, but not limited to, azathioprine, sulfasalazine, cyclosporine, and thalidomide within 4 weeks of screening
- Treatment with leflunomide without cholestyramine washout at the end of therapy
- Treatment with cyclophosphamide within 3 months of study entry
- Treatment with tacrolimus or tocilizumab within 4 weeks of study 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 |
|---|---|---|
| RilonaceptAlso: IL-1 Trap, Arcalyst2.2 mg/kg subcutaneously | Biological | Group 1, Group 2 |
Primary outcome
Time to Response to Treatment, as Determined by a Modified JIA ACR30 Requiring no Fever, Coupled With a Requirement for Corticosteroid Taper in Participants Who Are Taking Corticosteroids
Time frame: At Week 12
Number of Serious Adverse Events,Adverse Events, Infections, Development of MAS
Time frame: At Weeks 0- 24
4 secondary outcomes
Number of Participants With Response as Determined by JIA ACR50 and JIA ACR70
Time frame: At Week 4 and week 12
Pediatric Quality of Life Inventory
Time frame: At Weeks 4, 12 and 24
Physical Function as Determined by Childhood Health Assessment Questionnaire ( CHAQ)
Time frame: At Weeks 12 and 24
Number of Participants With Presence of Systemic Features ( Fever, Rash)
Time frame: At Weeks 4, 12 and 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Juvenile Idiopathic Arthritis
RecruitingPhase 3
A Study of Baricitinib in Participants From 1 Year to Less Than 18 Years Old With Juvenile Idiopathic Arthritis
Juvenile Idiopathic Arthritis
Recruiting
The Use of Infrared Cameras for Detecting Juvenile Idiopathic Arthritis
Juvenile Idiopathic Arthritis, Infrared Thermography
Not yet recruitingPhase 4
Withdrawal Versus Continued TNF Inhibitor Monotherapy Treatment in Children With Inactive Juvenile Idiopathic Arthritis
Juvenile Idiopathic Arthritis (JIA)
RecruitingObservational study
Tofacitinib in Juvenile Idiopathic Arthritis
Juvenile Idiopathic Arthritis (JIA)
RecruitingPhase 3
An Open-label Study to Evaluate the Pharmacokinetics and Safety of Bimekizumab in Pediatric Study Participants With Active Juvenile Idiopathic Arthritis Subtypes Enthesitis-related Arthritis (Including Juvenile-onset Ankylosing Spondylitis) and Juvenile Psoriatic Arthritis
Enthesitis-related Arthritis, Juvenile Psoriatic Arthritis
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.
Norman T. Ilowite, MD principal investigator
Montefiore Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Rilonacept in people with Juvenile Idiopathic Arthritis, run by Montefiore Medical Center at 1 site in United States.
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 6 inclusion and 19 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 11 Dec 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 11 Dec 2015). 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?
Time to Response to Treatment, as Determined by a Modified JIA ACR30 Requiring no Fever, Coupled With a Requirement for Corticosteroid Taper in Participants Who Are Taking Corticosteroids (time frame: At Week 12). The study lists 2 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 11 Dec 2015.