Therapeutic Use of Tadekinig Alfa in Adult-onset Still's Disease

NCT ID: NCT02398435

Last Updated: 2016-12-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

23 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-02-28

Study Completion Date

2016-07-31

Brief Summary

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The objective of this study is to assess safety, tolerability and early signs of efficacy of the investigational drug Tadekinig alfa in Adult-onset Still's disease, a rare polygenic auto-inflammatory disorder for which treatment remains empirical.

This disease is characterized by a daily spiking fever, arthralgia / arthritis, and skin rashes with frequent components of sore throat, lymphadenopathies and neutrophilic leukocytosis. The etiology is unknown. In addition to the above-mentioned clinical features, the diagnosis includes some laboratory components that reflect the systemic inflammation: high erythro-sedimentation rate, C-reactive protein, high serum ferritin and high levels of interleukin 18 (IL-18).

Tadekinig alfa is the drug name for recombinant human interleukin-18 binding protein (IL-18BP). This investigational drug was tested in healthy volunteers, psoriasis and rheumatoid arthritis patients in phase I studies. It demonstrated good safety and tolerability profile with only mild adverse events in the injection site.

Detailed Description

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The hypothesis of this study considers high levels of IL-18 during active Adult-onset still's disease as the therapeutic target. Treatment with IL-18BP will permit to inhibit the pro-inflammatory cascade triggered by IL-18 and may help to manage the different components of the disease.

This study is an open label, dose-finding study involving multiple centers in Europe. Two dose cohorts (80mg and 160mg) were treated during twelve weeks and followed-up for four more weeks.

Conditions

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Still's Disease, Adult-Onset

Keywords

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Auto-inflammatory disease Still's disease Arthritis Rheumatoid IL-18 interleukin-18 IL-18 binding protein

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 80 mg

Cohort 1 included 10 patients. Patients received Tadekinig alfa s.c with a dosage of 80mg. Safety assessments were conducted by data safety monitoring board. Non-responder patients were upscaled to next dose (160mg) after 3 weeks of treatment.

Group Type EXPERIMENTAL

Tadekinig alfa (recombinant human IL-18 binding protein)

Intervention Type BIOLOGICAL

Patients received the study treatment three times a week subcutaneously.

Cohort 160 mg

Cohort 2 included 13 patients. all patients were treated with Tadekinig alfa s.c with a dosage of 160mg. Safety was evaluated by data safety monitoring board.

Group Type EXPERIMENTAL

Tadekinig alfa (recombinant human IL-18 binding protein)

Intervention Type BIOLOGICAL

Patients received the study treatment three times a week subcutaneously.

Interventions

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Tadekinig alfa (recombinant human IL-18 binding protein)

Patients received the study treatment three times a week subcutaneously.

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Patients aged 18 years and older, diagnosed as AoSD based on the presence of the Yamaguchi criteria with active disease (see appendix 2), irrespective of the continuation of the permitted treatment mentioned below
* Patients with active disease will be considered if they exhibit at least two of the Yamaguchi's major criteria (see appendix 2) at the screening visit plus at least either fever or elevation of markers of inflammation (CRP ≥ 10 mg/L).
* Patients that have been exposed to NSAIDS, Prednisone (at least 5mg/day for ≥1 month) and/or synthetic sDMARDs (methotrexate at a dose of at least 10mg/day for ≥ 3 months) without response to treatment or with incomplete response to treatment
* Women of childbearing potential with negative pregnancy test at screening, V3, V4, V5 and V6 and that agree to follow highly effective birth control recommendations during the study and until 1 month after the end of the treatment. Birth control methods that are considered as highly effective are either: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation, progestogen-only hormonal contraception associated with inhibition of ovulation, intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomized partner or sexual abstinence.

In each case of delayed menstrual period (over one month between menstruations, confirmation of absence of pregnancy is strongly recommended. This recommendation also applies to women of child bearing potential with infrequent or irregular menstrual cycles.

As regards the duration of contraception after the study, taking into account the median half-life of Tadekinig alfa of almost 40h, 5 half-lives represent duration of 200 hours. In order to be on the safe side, a post-study contraception duration of - Patients can maintain treatment with stable doses of Non-Steroidal anti-inflammatory Drugs (NSAIDs), Prednisone (stable dose of Prednisone of at least 5mg/day), and sDMARDs during Tadekinig alfa treatment (methotrexate at a dose of at least 10mg/week). Specifically baseline levels of prednisone treatment can be maintained or tapered (due to patient improvement), any requirement for prednisone increase during treatment will be considered a treatment failure.

* Ability to understand and willingness to sign a written informed consent
* Previous treatments with biologicals are allowed if the following wash-out periods are respected: one week for anakinra, two weeks for etanercept, and 6 weeks for, adalimumab, certolizumab, golimumab, tocilizumab, abatacept and 8 weeks for infliximab. Previous rituximab administration will require 6 months of washout and normal B-cell counts and previous treatment with canakinumab will require 6 months of washout.

* Inability to understand and unwilling to sign a written informed consent
* Any acute or chronic life-threatening disease: Such as cancer, and irreversible organ failures of heart, liver, lung and kidney (creatinine not higher than 1.5 X upper limit of normal).
* Patients having received adalimumab, certolizumab, golimumab, tocilizumab, abatacept within 6 weeks, infliximab within 8 weeks, canakinumab within 6 months, etanercept within 2 weeks, or anakinra 1 week prior to the start of Tadekinig alfa will not be enrolled into the study. Patients that have received rituximab within 6 months and/or have persistent low B-cell counts will not be eligible for enrolment.
* Subject who cannot be expected to comply with the study procedures
* Currently participating or having participated in another clinical trial during the last 4 weeks prior to the beginning of this study.
* Patients with no social security coverage
* Patients with a history of severe hypersensitivity reactions

Exclusion Criteria

* Patients with a first episode of AoSD with less than one month of therapy with Prednisone or sDMARDs
* Patients with active or chronic infections (i.e. Tuberculosis (TB), HIV, HBV \& HCV)
* Patients suffering from inherited immunodefinciency diseases
* Patients suffering from immune-mediated inflammatory diseases, including RA, SLE, etc. or spondylarthropathies, or inflammatory bowel disease.
* Patients with white blood cell counts below 2'500 cells/mm3
* Concomitantly treated with biologicals
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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AB2 Bio Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Cem Gabay, Prof.

Role: PRINCIPAL_INVESTIGATOR

Hospital University of Geneva

Locations

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Hôpital Pellegrin

Bordeaux, , France

Site Status

CHRU de Lille - Hôpital Claude Huriez

Lille, , France

Site Status

Hôpital de la Croix Rousse

Lyon, , France

Site Status

CHRU de Montpellier

Montpellier, , France

Site Status

CHU de Nantes - Hôtel Dieu

Nantes, , France

Site Status

CHU Paris-GH La Pitié Salpêtrière-Charles Foix - Hôpital Pitié-Salpêtrière

Paris, , France

Site Status

Strasbourg University Hospital

Strasbourg, , France

Site Status

Innere Medizin II - Rheumatologie Schlosspark-Klinik

Berlin, , Germany

Site Status

Medizinische Klinik - Rheumatologie und Klinische

Berlin, , Germany

Site Status

Universitätsklinikum Erlangen

Erlangen, , Germany

Site Status

Asklepios Klinik Altona

Hamburg, , Germany

Site Status

St. Elisabeth Gruppe GmbH Katholische Kliniken Rhein-Ruhr Rheumazentrum Ruhrgebiet

Herne, , Germany

Site Status

Universitätsklinikum Jena Klinik für Innere Medizin III Rheumatologie/Osteologie

Jena, , Germany

Site Status

Klinik Kirchheim

Kirchheim unter Teck, , Germany

Site Status

Universitätsklinikum Schleswig-Holstein - Campus Lübeck

Lübeck, , Germany

Site Status

UNIVERSITÄTSMEDIZIN der Johannes-Gutenberg-Universität Mainz I. Medizinische Klinik und Poliklinik Rheumatologie

Mainz, , Germany

Site Status

LMU München

München, , Germany

Site Status

Hôpitaux Universitaires de Genève - HUG

Geneva, , Switzerland

Site Status

CHUV hospital

Lausanne, , Switzerland

Site Status

Immunologie-Zentrum de Zürich

Zurich, , Switzerland

Site Status

Countries

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France Germany Switzerland

References

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Bagnari V, Colina M, Ciancio G, Govoni M, Trotta F. Adult-onset Still's disease. Rheumatol Int. 2010 May;30(7):855-62. doi: 10.1007/s00296-009-1291-y. Epub 2009 Dec 18.

Reference Type BACKGROUND
PMID: 20020138 (View on PubMed)

Bywaters EG. Still's disease in the adult. Ann Rheum Dis. 1971 Mar;30(2):121-33. doi: 10.1136/ard.30.2.121. No abstract available.

Reference Type BACKGROUND
PMID: 5315135 (View on PubMed)

Efthimiou P, Kontzias A, Ward CM, Ogden NS. Adult-onset Still's disease: can recent advances in our understanding of its pathogenesis lead to targeted therapy? Nat Clin Pract Rheumatol. 2007 Jun;3(6):328-35. doi: 10.1038/ncprheum0510.

Reference Type BACKGROUND
PMID: 17538564 (View on PubMed)

Fautrel B. Adult-onset Still disease. Best Pract Res Clin Rheumatol. 2008 Oct;22(5):773-92. doi: 10.1016/j.berh.2008.08.006.

Reference Type BACKGROUND
PMID: 19028363 (View on PubMed)

Kawashima M, Yamamura M, Taniai M, Yamauchi H, Tanimoto T, Kurimoto M, Miyawaki S, Amano T, Takeuchi T, Makino H. Levels of interleukin-18 and its binding inhibitors in the blood circulation of patients with adult-onset Still's disease. Arthritis Rheum. 2001 Mar;44(3):550-60. doi: 10.1002/1529-0131(200103)44:33.0.CO;2-5.

Reference Type BACKGROUND
PMID: 11263769 (View on PubMed)

Yamaguchi M, Ohta A, Tsunematsu T, Kasukawa R, Mizushima Y, Kashiwagi H, Kashiwazaki S, Tanimoto K, Matsumoto Y, Ota T, et al. Preliminary criteria for classification of adult Still's disease. J Rheumatol. 1992 Mar;19(3):424-30.

Reference Type BACKGROUND
PMID: 1578458 (View on PubMed)

Gabay C, Fautrel B, Rech J, Spertini F, Feist E, Kotter I, Hachulla E, Morel J, Schaeverbeke T, Hamidou MA, Martin T, Hellmich B, Lamprecht P, Schulze-Koops H, Courvoisier DS, Sleight A, Schiffrin EJ. Open-label, multicentre, dose-escalating phase II clinical trial on the safety and efficacy of tadekinig alfa (IL-18BP) in adult-onset Still's disease. Ann Rheum Dis. 2018 Jun;77(6):840-847. doi: 10.1136/annrheumdis-2017-212608. Epub 2018 Feb 22.

Reference Type DERIVED
PMID: 29472362 (View on PubMed)

Other Identifiers

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AOSD-2014-001

Identifier Type: -

Identifier Source: org_study_id