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| ID | Type | Description | Link |
|---|---|---|---|
| 268200700015C-2-0-0 | U.S. NIH Grant/Contract | View source | |
| HHSN2682007000015C |
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| Name | Class |
|---|---|
| National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) | NIH |
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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.
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:
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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 |
|
| 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 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Rilonacept | Biological | 2.2 mg/kg subcutaneously |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 | At Week 12 | |
| Number of Serious Adverse Events,Adverse Events, Infections, Development of MAS | At Weeks 0- 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Response as Determined by JIA ACR50 and JIA ACR70 | At Week 4 and week 12 | |
| Pediatric Quality of Life Inventory | Visual Analog Score (0-100 mm) 0 very well , 100 very poor |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Norman T. Ilowite, MD | Montefiore Medical Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Montefiore Medical Center | The Bronx | New York | 10467 | United States |
71 participants enrolled. 1 participant was erroneously randomized and was not included in the analysis. This patient was not exposed to study drug
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| ID | Title | Description |
|---|---|---|
| FG000 | Rilonacept | Loading dose of rilonacept (4.4mg/kg) at Week 0, followed by rilonacept 2.2 mg/kg/week followed by a placebo loading dose and then rilonacept in the long term extension phase Rilonacept: 2.2 mg/kg subcutaneously |
| FG001 | Placebo | Placebo loading dose followed by maintenance dose for 4 weeks, followed by rilonacept loading dose (4.4mg/kg), followed by rilonacept 2.2 mg/kg/week for the long term extension Rilonacept: 2.2 mg/kg subcutaneously |
| FG002 | Long Term Extension All Participants | All participants who benefited from rilonacept were eligible to enroll this phase and receive rilonacept 2.2mg/kg weekly |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Double Blind Placebo Phase Week 0-4 |
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| All Active Treatment Phase Week 4-24 |
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| Long Term Ext. Phase Week 24-month 21 |
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| ID | Title | Description |
|---|---|---|
| BG000 | Rilonacept | Loading dose of rilonacept (4.4mg/kg) at Week 0, followed by rilonacept 2.2 mg/kg/week for the remainder of the study Rilonacept: 2.2 mg/kg subcutaneously |
| BG001 | Placebo |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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 | Posted | Median | Inter-Quartile Range | weeks | At Week 12 |
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Rilonacept Week (0-4) | Loading dose of rilonacept (4.4mg/kg) at Week 0, followed by rilonacept 2.2 mg/kg/week for the remainder of the study Rilonacept: 2.2 mg/kg subcutaneously |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Juvenile Arthritis | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain ,upper | Gastrointestinal disorders | Systematic Assessment | Adverse events |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr.Norman T.Ilowite | Children's Hospital at Montefiore | 718-696-2602 | NILOWITE@montefiore.org |
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| ID | Term |
|---|---|
| D001171 | Arthritis, Juvenile |
| C565798 | Rheumatoid Arthritis, Systemic Juvenile |
| ID | Term |
|---|---|
| D001168 | Arthritis |
| D007592 | Joint Diseases |
| D009140 | Musculoskeletal Diseases |
| D012216 | Rheumatic Diseases |
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| ID | Term |
|---|---|
| C531377 | rilonacept |
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| 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 | At Weeks 12 and 24 |
| Number of Participants With Presence of Systemic Features ( Fever, Rash) | At Weeks 4, 12 and 24 |
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| NOT COMPLETED |
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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
Rilonacept: 2.2 mg/kg subcutaneously
| BG002 | Total | Total of all reporting groups |
| years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Black ,white and Other are races Hispanic and Non-Hispanic are ethnicities. All patients were counted twice. | Number | Participants |
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| Disease Duration | Mean | Standard Deviation | years |
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| No.of joints with active disease | Mean | Standard Deviation | joints |
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| Prior medications | Number | participants |
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| Disease characteristics in the past | Number | participants |
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| Units | Counts |
|---|---|
| Participants |
|
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| Secondary | Number of Participants With Response as Determined by JIA ACR50 and JIA ACR70 | Participants in the study at week 4 (rilonacept 35 and placebo 33).Participants in the study at week 12 ( Rilonacept 33 and placebo 29). | Posted | Number | participants | At Week 4 and week 12 |
|
|
|
| Secondary | Pediatric Quality of Life Inventory | Visual Analog Score (0-100 mm) 0 very well , 100 very poor | At Week 4 ,36 Rilonacept and 34 Placebo patient. At week 12, Rilonacept 33 patients and Placebo 29.At baseline Rilonacept 36 and Placebo 35 participants. | Posted | Median | Inter-Quartile Range | units on a scale | At Weeks 4, 12 and 24 |
|
|
|
| Secondary | 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 | 36 Rilonacept and 35 placebo at baseline , 36 Rilonacept and 34 placebo at week 4, 33 Rilonacept and 29 placebo at week 12, and 57 combined at week 24. | Posted | Median | Inter-Quartile Range | units on a scale | At Weeks 12 and 24 |
|
|
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| Secondary | Number of Participants With Presence of Systemic Features ( Fever, Rash) | At week 4 ,Rilonacept 36 and Placebo 34 participants , at week 12 , Rilonacept 33 and 29 Placebo participants , and at week 24 combined group with 57 participants, at baseline Rilonacept 36 and Placebo 35 participants. | Posted | Number | participants | At Weeks 4, 12 and 24 |
|
|
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| Primary | Number of Serious Adverse Events,Adverse Events, Infections, Development of MAS | Posted | Number | events | At Weeks 0- 24 |
|
|
|
| 1 |
| 36 |
| 9 |
| 36 |
| EG001 | Placebo Week (0-4) | 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 Rilonacept: 2.2 mg/kg subcutaneously | 1 | 35 | 19 | 35 |
| EG002 | Rilonacept Week (4-24) | Loading dose of rilonacept (4.4mg/kg) at Week 0, followed by rilonacept 2.2 mg/kg/week for the remainder of the study Rilonacept: 2.2 mg/kg subcutaneously | 3 | 35 | 27 | 35 |
| EG003 | Placebo Week (4-24) | 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 Rilonacept: 2.2 mg/kg subcutaneously | 1 | 33 | 28 | 33 |
| EG004 | Long Term Extension 24 Weeks to 21 Months | 6 | 40 | 28 | 40 |
| Abnormal Liver Function Test | Gastrointestinal disorders | Systematic Assessment |
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| Pyrexia | Infections and infestations | Systematic Assessment |
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| Variccela | Infections and infestations | Systematic Assessment |
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| Viral uper respiratory tract infestion | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Mental status Changes | Psychiatric disorders | Systematic Assessment |
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| Pericarditis | Cardiac disorders | Systematic Assessment |
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| Pharingitis,Streptoccocal | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Gastroenteritis ,Salmonela | Gastrointestinal disorders | Systematic Assessment |
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| Histiocytosis,hematophagic | Blood and lymphatic system disorders | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | Systematic Assessment | Adverse events |
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| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | Adverse events |
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| Headache | General disorders | Systematic Assessment | Adverse events |
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| Nausea | Gastrointestinal disorders | Systematic Assessment | Adverse events |
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| Pharyngitis,Streptococcal | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | Adverse events |
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| Pyrexia | Infections and infestations | Systematic Assessment | Adverse events |
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| Rash | Immune system disorders | Systematic Assessment | Adverse events |
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| Upper Respiratory tract infection | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | Adverse events |
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| Vomiting | Gastrointestinal disorders | Systematic Assessment | Adverse events |
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| injection site reaction | General disorders | Systematic Assessment |
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| Body temperature increased | Investigations | Systematic Assessment |
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| Urinary tract infections | Infections and infestations | Systematic Assessment |
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| Fatigue | General disorders | Systematic Assessment |
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| Non Cardiac chest pain | General disorders | Systematic Assessment |
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| Pain | General disorders | Systematic Assessment |
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| Rhinorea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| dyspnea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | Systematic Assessment |
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| diarrhoea | Gastrointestinal disorders | Systematic Assessment |
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| Joint swelling | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| dry skin | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Erythema | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Dizziness | Nervous system disorders | Systematic Assessment |
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| Blood fibrinogen dicreased | Investigations | Systematic Assessment |
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| Alanine amino transferase Increased | Investigations | Systematic Assessment |
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| Aspartate Amino transferase increased | Investigations | Systematic Assessment |
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| D003240 |
| Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| week 4 ,ACR 70 |
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| week 12,ACR 70 |
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| week 12 |
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| week 24 |
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| baseline |
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| week 12 |
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| week 24 |
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| baseline |
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| Fever at week12 |
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| Rash at week 12 |
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| week 24 Fever |
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| week 24 Rash |
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| baseline Fever |
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| beseline Rash |
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| Title | Measurements |
|---|---|
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| Infections |
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| MAS |
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