Active Juvenile Idiopathic Arthritis (JIA) Compassionate Use

NCT00775437 | Completed Phase 3 Results

• 2 other identifiers: M10-4442009-013091-40
• Study Type: interventional
• Target: 32
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Juvenile Idiopathic Arthritis

Keywords

juvenile idiopathic arthritis; compassionate use; open label

Outcome Measures

Primary Outcomes (1)

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

  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).

  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).

Secondary Outcomes (46)

• Mean Serum Adalimumab Trough Concentrations at Week 0, Week 12, and Week 24

  Weeks 0, 12, and 24

• Hemoglobin: Mean Change From Baseline to Each Visit

  Baseline and Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120

• Hematocrit: Mean Change From Baseline to Each Visit

  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

Study Arms (1)

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.

Drug: Adalimumab

Interventions

Adalimumab DRUG

Adalimumab solution for injection for subcutaneous use.

Also known as: Humira, ABT-D2E7

Adalimumab

Eligibility Criteria

• Age: 2 Years - 4 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• AbbVie (prior sponsor, Abbott): lead

Related Publications (2)

• Horneff G, Seyger MMB, Arikan D, Kalabic J, Anderson JK, Lazar A, Williams DA, Wang C, Tarzynski-Potempa R, Hyams JS. Safety of Adalimumab in Pediatric Patients with Polyarticular Juvenile Idiopathic Arthritis, Enthesitis-Related Arthritis, Psoriasis, and Crohn's Disease. J Pediatr. 2018 Oct;201:166-175.e3. doi: 10.1016/j.jpeds.2018.05.042. Epub 2018 Jul 25.

  PMID: 30054164 DERIVED

• Kingsbury DJ, Bader-Meunier B, Patel G, Arora V, Kalabic J, Kupper H. Safety, effectiveness, and pharmacokinetics of adalimumab in children with polyarticular juvenile idiopathic arthritis aged 2 to 4 years. Clin Rheumatol. 2014;33(10):1433-41. doi: 10.1007/s10067-014-2498-1. Epub 2014 Feb 2.

  PMID: 24487484 DERIVED

Related Links

• Related Info

MeSH Terms

Conditions

Arthritis, Juvenile

Interventions

Adalimumab

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Results Point of Contact

• Title: Global Medical Information
• Organization: AbbVie

800-633-9110

Study Officials

• Aileen L. Pangan, MD

  AbbVie

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 17, 2008
• First Posted: October 20, 2008
• Study Start: March 1, 2009
• Primary Completion: March 1, 2013
• Study Completion: March 1, 2013
• Last Updated: January 26, 2016
• Results First Posted: January 26, 2016

Record last verified: 2015-12