A Study to Evaluate Efficacy and Safety of Anakinra in the Treatment of Still's Disease (SJIA and AOSD)

NCT03265132 | Terminated Phase 3 Results DMC FDA Drug

• 1 other identifier: Sobi.ANAKIN-301
• Study Type: interventional
• Target: 13
• Locations: 2 countries
• Sites: 39

Brief Summary

The aim of this study is to demonstrate the efficacy and to evaluate the safety, pharmacokinetics (PK) and immunogenicity of anakinra in patients with newly diagnosed Still's disease, including SJIA (Systemic juvenile idiopathic arthritis) and AOSD (Adult-onset Still's disease).

Conditions

Still's Disease, Adult-Onset; Still's Disease, Juvenile-Onset

Keywords

Interleukin 1 receptor antagonist; IL-1 receptor antagonist; Kineret; anakinra; Adult-Onset Still's Disease; Systemic Juvenile Idiopathic Arthritis

Outcome Measures

Primary Outcomes (1)

• Proportion of ACR30 Responders With Absence of Fever Attributable to the Disease During the 7 Days Preceding Week 2.

  ACR30 response is defined as an improvement of ≥ 30% from baseline in at least 3 of any 6 variables listed below. Also no more than 1 of the 6 variables may worsen by \>30% from baseline. (ACR: American College of Rheumatology) 1. Physician global assessment of disease activity - Assessed on a Visual Analogue Scale (VAS) from no disease activity (0 mm) to very severe disease activity (100 mm). 2. Patient/parent global assessment of overall well-being - Assessed on a VAS from very well (0 mm) to very poor (100 mm). 3. Number of joints with active arthritis. 4. Number of joints with limitation of motion. 5. Assessment of physical function - Patient Reported Outcome instruments : Childhood Health Assessment Questionnaire (CHAQ) /Stanford Health Assessment Questionnaire (SHAQ). 6. C-Reactive Protein (CRP) (mg/L).

  Week 2

Secondary Outcomes (49)

• Proportion of ACR30 Responders With Absence of Fever During 24 Hours Preceding Week 1.

  Week 1

• Proportion of ACR50 Responders With Absence of Fever During 24 Hours Preceding Week 1.

  Week 1

• Proportion of ACR70 Responders With Absence of Fever During 24 Hours Preceding Week 1.

  Week 1

• Proportion of ACR90 Responders With Absence of Fever During 24 Hours Preceding Week 1.

  Week 1

• Proportion of ACR50 Responders With Absence of Fever During 7 Days Preceding Week 2.

  Week 2

Study Arms (2)

anakinra

EXPERIMENTAL

2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day)

Biological: anakinra

Placebo

PLACEBO COMPARATOR

Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day

Drug: Placebo

Interventions

anakinra BIOLOGICAL

sub cutaneous injection

Also known as: Kineret

anakinra

Placebo DRUG

sub cutaneous injection

Placebo

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed informed consent.
• Male and female patients with a body weight ≥ 10 kg.
• Diagnosis of Still's disease.
• If currently on glucocorticoid treatment, a stable dose for at least 1 week prior to randomization.
• If currently on methotrexate treatment, a stable dose for at least 8 weeks prior to randomization.
• Active disease.
• Female patients of childbearing potential must use an effective method of contraception during the study (abstinence being a possible option) as well as present a negative pregnancy test prior to randomization.
• Negative interferon-gamma release assay or Purified protein derivative ( PPD) test within 2 months prior to randomization. If not available, a test should be performed at day of randomization.

You may not qualify if:

• Diagnosis of Still's disease more than 6 months prior to randomization.
• Previous randomization into this study.
• Participation in another concurrent clinical interventional study within 30 days of randomization.
• Treatment with an investigational drug within 5 half-lives prior to randomization.
• Previous or current treatment with anakinra, canakinumab or any other IL-1 inhibitor.
• Use of the following therapies prior to randomization:
• Narcotic analgesics within 24 hours prior to randomization.
• Dapsone or etanercept within 3 weeks prior to randomization.
• Intraarticular, intramuscular or intravenous administration of glucocorticoids or intravenous immunoglobulin (Ig) within 4 weeks prior to randomization.
• Intravenous Ig with proven Still's disease modifying effect, leflunomide, infliximab or adalimumab within 8 weeks prior to randomization.
• Thalidomide, cyclosporine, mycophenolate mofetil, 6-mercaptopurine, azathioprine, cyclophosphamide, chlorambucil or any other immunosuppressant within 12 weeks prior to randomization.
• Tocilizumab within 12 weeks prior to randomization or any other immunomodulatory medication within 4 half-lives prior to randomization
• Rituximab within 26 weeks prior to randomization.
• Live vaccines within 1 month prior to randomization.
• Known presence or suspicion of active, chronic or recurrent bacterial, fungal or viral infections, including tuberculosis, HIV infection or hepatitis B or C infection.

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead

Study Sites (39)

University of Alabama Birmingham

Birmingham, Alabama, 35294, United States

Location

Attune Health

Beverly Hills, California, 90211, United States

Location

Rady Children's Hospital & Health Center

San Diego, California, 92123, United States

Location

The Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

University of Florida

Gainesville, Florida, 32611, United States

Location

University of Miami

Miami, Florida, 33124, United States

Location

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

Location

Children's Mercy Hospital and Clinics

Kansas City, Kansas, 67208, United States

Location

University of Louisville School of Medicine Research Foundation

Louisville, Kentucky, 40202, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02115, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Saint Paul Rheumatology

Eagan, Minnesota, 55121, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55454, United States

Location

Saint Louis University

St Louis, Missouri, 63104, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

Institute for Rheumatic and Autoimmune Diseases

Summit, New Jersey, 07901, United States

Location

Hospital for Special Surgery

New York, New York, 10021, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

UNC Hospitals

Chapel Hill, North Carolina, 27514, United States

Location

Duke Children's Hospital and Health Center

Durham, North Carolina, 27710, United States

Location

Wake Forest Baptist Brenner Medical Center

Winston-Salem, North Carolina, 27157, United States

Location

MetroHealth System

Cleveland, Ohio, 44109, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

Monroe Carell Jr. Children's Hospital at Vanderbilt

Nashville, Tennessee, 37232, United States

Location

Baylor Research Institute

Dallas, Texas, 75204, United States

Location

Univ of TX Southwestern Medical Center Dallas - Texas Scottish Rite Hospital for Children

Dallas, Texas, 75219, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

University of Texas Health Science Center at San Antonio

San Antonio, Texas, 78229, United States

Location

University of Utah Hospitals and Clinics

Salt Lake City, Utah, 84113, United States

Location

University of Vermont Medical Center

Burlington, Vermont, 05401, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

University of Washington

Seattle, Washington, 98195, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

University of Calgary - Alberta Children's Hospital

Calgary, Canada

Location

University of Calgary

Calgary, Canada

Location

The Hospital for Sick Children

Toronto, Canada

Location

MeSH Terms

Conditions

Still's Disease, Adult-Onset; Arthritis, Juvenile

Interventions

Interleukin 1 Receptor Antagonist Protein

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Results Point of Contact

• Title: Kineret Clinical Program Leader
• Organization: Swedish Orphan Biovitrum

info@sobi.com

+46(8)6972000

Study Officials

• Sven Ohlman, MD PhD

  Swedish Orphan Biovitrum

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 25, 2017
• First Posted: August 29, 2017
• Study Start: September 26, 2017
• Primary Completion: February 13, 2019
• Study Completion: May 23, 2019
• Last Updated: June 30, 2021
• Results First Posted: April 28, 2020

Record last verified: 2021-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (36); CA (3)