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Clinical Trials/NCT00775437
NCT00775437CompletedPhase 3

Compassionate Use Study of Adalimumab in Children 2 to < 4 Years Old or Age 4 and Above Weighing Less Than 15 kg With Active Juvenile Idiopathic Arthritis (JIA)

AbbVie (prior sponsor, Abbott)0 sites32 target enrollmentStarted: March 2009Last updated:
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Conditions
Interventions
Drugs

Trial Snapshot

Phase
Phase 3
Status
Completed
Sponsor
Enrollment
32
Primary Endpoint
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

The main objective of this study is to evaluate the safety of adalimumab in patients 2 to < 4 years of age or ≥ 4 years of age weighing < 15 kg, with moderately to severely active polyarticular juvenile idiopathic arthritis (JIA) or polyarticular course JIA.

Study Design

Study Type
Interventional
Allocation
Na
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
2 Years to 4 Years (Child)
Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • •A parent or guardian has voluntarily signed and dated an informed consent form, approved by an Institutional Review Board (IRB)/Independent Ethics Committee (IEC), after the nature of the study has been explained and the subject's parent or legal guardian has had the opportunity to ask questions. The informed consent must be signed before any study-specific procedures are performed or before any medication is discontinued for the purpose of this study. The parent must also be willing to comply with all the requirements of this study protocol.
  • •Subject has a disease diagnosis of moderately to severely active polyarticular or polyarticular course juvenile idiopathic arthritis (JIA; defined as arthritis affecting > = 5 joints at the time of treatment initiation). This corresponds to the International League of Associations for Rheumatology categories of polyarticular rheumatoid factor positive (RF+), polyarticular Rheumatoid factor negative (RF-) disease, and extended oligoarthritis disease.
  • •Subject must be aged 2 to < 4 years old with moderately to severely active polyarticular JIA or polyarticular course JIA or age 4 or greater weighing < 15 kg with moderately to severely active polyarticular JIA or polyarticular course JIA, per International League of Associations for Rheumatology (ILAR) criteria.
  • •Subject is judged to be in generally good health as determined by the Investigator based upon the results of medical history, laboratory profile, and physical examination performed at Screening and confirmed at Baseline. This includes, but is not limited to, a normal cardiopulmonary and normal neurological exam result.
  • •Parent or legal guardian must be able and willing to administer subcutaneous (SC) injections or have a qualified person available to administer SC injections.
  • •Parent or legal guardian must be willing to actively supervise storage and administration of study drug and to ensure that the time of each dose is accurately recorded in the subject's diary.
  • •Subject must have negative purified protein derivative (PPD) test (or equivalent) at Screening. If subject has a positive (greater than or equal to 5mm induration) PPD test result, a chest x-ray (posterior-anterior [PA] and lateral view) must be performed. If subject has a positive test (or equivalent), has had a past ulcerative reaction to PPD placement, and/or a chest x ray consistent with tuberculosis [TB] exposure, subject may not be enrolled into the study.
  • •For subjects in the European Union [EU], subject must have previously failed, had an insufficient response, or have been intolerant to at least one Disease-Modifying Anti Rheumatic Drug (DMARD).

Exclusion Criteria

  • •Subject has had prior exposure to Tysabri® (natalizumab) or Raptiva® (efalizumab) or any other biologic therapy, such as Orencia® (abatacept), Kineret® (anakinra), Actemra® (tocilizumab), Rituxan® (rituximab). Any previous use of anti-tumor necrosis factor [TNF] agents, including Enbrel® (etanercept), Remicade® (infliximab), Cimzia® (certolizumab pegol), Simponi® (golimumab), and adalimumab is also prohibited.
  • •Infection(s) requiring treatment with intravenous (IV) anti-infectives within 30 days prior to Baseline Visit or oral anti-infectives within 14 days prior to the Baseline Visit.
  • •Subject has undergone joint surgery within the preceding two months of the Screening Visit (at joints to be assessed within the study).
  • •Subject has a previous diagnosis of a condition that could cause arthritis other than polyarticular JIA.
  • •Subject has a history of an allergic reaction or significant sensitivity to constituents of the study drug, adalimumab.
  • •Subject has been treated with any investigational drug of chemical or biologic nature within a minimum of 30 days or 5 half lives (whichever is longer) of the drug prior to Baseline visit. Should these biologics become approved, they would continue to be excluded.
  • •Subject has a poorly controlled medical condition, such as uncontrolled diabetes, unstable heart disease, recent cerebrovascular accidents, seizure disorder, and any other condition which, in the opinion of the Investigator, would put the subject at risk by participation in the study.
  • •Subject has a history of clinically significant hematologic (e.g., severe anemia, leukopenia, thrombocytopenia, a clotting disorder), renal, liver disease (e.g., fibrosis, cirrhosis, hepatitis), or active gastroenteric ulcer.
  • •Subject is considered by the Investigator, for any reason, to be an unsuitable candidate for the study.
  • •Subject has evidence of active TB infection.
  • •Subject has history of moderate to severe congestive heart failure (New York Heart Association [NYHA] class III or IV), recent cerebrovascular accident or thrombotic event and any other condition which, in the opinion of the investigator, would put the subject at risk by participation in the protocol.
  • •Evidence of dysplasia or history of malignancy (including lymphoma and leukemia).
  • •History of demyelinating disease (including myelitis) or neurologic symptoms suggestive of central nervous system [CNS] demyelinating disease.
  • •History of invasive fungal infection (e.g., listeriosis, histoplasmosis), active viral disorders, human immunodeficiency virus (HIV) infection.
  • •Positive Hepatitis B test result.
  • •Chronic recurring infections or active TB.
  • •Screening laboratory and other analyses show any of the following abnormal results:
  • •electrocardiogram [ECG] - with clinically significant abnormalities;
  • •Aspartate transaminases (AST) or alanine transaminase (ALT) > 1.75 the upper limit of the reference range;
  • •Total bilirubin >=3 mg/dL;
  • •Serum creatinine > 1.6 mg/dL (convert to mmol/L).
  • •Evidence of dysplasia or history of malignancy (including lymphoma and leukemia) other than a successfully treated non-metastatic cutaneous squamous cell or basal cell carcinoma.

Arms & Interventions

Adalimumab

Experimental

Adalimumab 24 mg/m^2 body surface area (BSA) up to a total dose of 20 mg administered every other week (eow) by parent or designee as a single dose via subcutaneous injection at approximately the same time of day, for a minimum of 24 weeks. Participants could continue in the study until age 4 and 15 kg (US and Puerto Rico) or for up to 1 additional year after reaching age 4 and 15 kg (EU). Visits beyond Week 24 occurred every 12 weeks for those participants who continued in the study.

Intervention: Adalimumab (Drug)

Outcomes

Primary Outcomes

Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

Time Frame: TEAEs were collected from first dose of study drug until 70 days after the last dose of study drug and before start of commercial adalimumab or other biologics (32.5 months).

An adverse event (AE) is defined as any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. If an AE meets any of the following criteria, it is considered a serious adverse event (SAE): Results in death, is life-threatening, results in hospitalization or the prolongation of hospitalization, is a congenital anomaly or a persistent or significant disability/incapacity, or is an important medical event requiring medical or surgical intervention to prevent a serious outcome. A treatment-emergent AE (TEAE) is defined as any AE with onset or worsening reported by a participant from the time that the first dose of adalimumab is administered until 5 half-lives (70 days) have elapsed following discontinuation of adalimumab administration (total of 32.5 months).

Secondary Outcomes

  • Mean Serum Adalimumab Trough Concentrations at Week 0, Week 12, and Week 24(Weeks 0, 12, and 24)
  • Hematocrit: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Hemoglobin: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Creatinine: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Percentage of Participants Achieving Pediatric American College of Rheumatology (PedACR) 90% Response (PedACR90)(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Disability Index of Child Health Assessment Questionnaire (DICHAQ): Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Active Joint Counts (AJC73): Mean Change From Baseline to Each Visit(Baseline and Weeks 2, 4, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Neutrophils: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Eosinophils: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Aspartate Aminotransferase (SGOT/AST): Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Total Bilirubin: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Percentage of Participants Achieving Pediatric American College of Rheumatology (PedACR) 30% Response (PedACR30)(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Red Blood Cell (RBC) Count: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Platelets: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • White Blood Cell (WBC) Count: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Lymphocytes: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Parent's Global Assessment of Disease Activity: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Child's Health Questionnaire Parent Form (CHQ-PF50) Physical Functioning Category: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120.)
  • Monocytes: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Basophils: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Alanine Aminotransferase (SGPT/ALT): Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Alkaline Phosphatase (ALP): Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Creatine Phosphokinase: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Percentage of Participants Achieving Pediatric American College of Rheumatology (PedACR) 50% Response (PedACR50)(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Physician's Global Assessment of Disease Activity: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Limitation of Passive Motion (LOM69) Joint Count: Mean Change From Baseline to Each Visit(Baseline and Weeks 2, 4, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Uric Acid: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Swollen Joint Count (SJC66): Mean Change From Baseline to Each Visit(Baseline and Weeks 2, 4, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Child's Health Questionnaire Parent Form (CHQ-PF50) Global Health Category: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Child's Health Questionnaire Parent Form (CHQ-PF50) Bodily Pain/Discomfort Category: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Child's Health Questionnaire Parent Form (CHQ-PF50) Change in Health Category: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Percentage of Participants Achieving Pediatric American College of Rheumatology (PedACR) 70% Response (PedACR70)(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • C-reactive Protein (CRP): Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Tender Joint Count (TJC75): Mean Change From Baseline to Each Visit(Baseline and Weeks 2, 4, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Child's Health Questionnaire Parent Form (CHQ-PF50) Global Behavior Item: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Child's Health Questionnaire Parent Form (CHQ-PF50) Mental Health Category: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Child's Health Questionnaire Parent Form (CHQ-PF50) General Health Perceptions Category: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Pain on Passive Motion (POM75) Joint Count: Mean Change From Baseline to Each Visit(Baseline and Weeks 2, 4, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Child's Health Questionnaire Parent Form (CHQ-PF50) Role/Social Limitations/Emotional/Behavioral Category: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Child's Health Questionnaire Parent Form (CHQ-PF50) Behavior Category: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Child's Health Questionnaire Parent Form (CHQ-PF50) Role/Social Limitations - Physical Category: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Child's Health Questionnaire Parent Form (CHQ-PF50) Parental Impact - Emotional Category: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Child's Health Questionnaire Parent Form (CHQ-PF50) Family Cohesion Category: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Child's Health Questionnaire Parent Form (CHQ-PF50) Self Esteem Category: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Child's Health Questionnaire Parent Form (CHQ-PF50) Parental Impact - Time Category: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)
  • Child's Health Questionnaire Parent Form (CHQ-PF50) Family Activities Category: Mean Change From Baseline to Each Visit(Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120)

Investigators

Sponsor
AbbVie (prior sponsor, Abbott)
Sponsor Class
Industry
Responsible Party
Sponsor

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