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Single Patient Use of Tocilizumab in Systemic Onset Juvenile Idiopathic Arthritis

9. mai 2017 oppdatert av: Tufts Medical Center

Single Patient Use of Tocilizumab for Treatment of Steroid Dependent, Active Systemic Onset Juvenile Idiopathic Arthritis

The purpose of this study is to see if tocilizumab is safe and effective for treating systemic onset Juvenile Idiopathic Arthritis (soJIA). Another purpose is to see if tocilizumab helps reduce the amount of steroids (prednisone) needed to control symptoms of soJIA.

Studieoversikt

Status

Avsluttet

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

Systemic onset Juvenile Idiopathic Arthritis (soJIA) is a type of arthritis (inflammation of the joints) that occurs with other symptoms including fever, swollen lymph nodes (glands), rash, and body aches. Because soJIA can be difficult to treat, children with soJIA can have severe problems from long-term use of steroids (prednisone). These problems include low bone density (weak bones), fractures, failure to grow properly, and large weight gain. The arthritis that occurs in soJIA often causes damage to many joints. This can make it hard to move around or do basic tasks like dressing. Also, a life-threatening illness called Macrophage Activation Syndrome (MAS) can occur when starting, stopping, or changing drugs that are used to treat soJIA.

SoJIA can be hard to treat and many children with soJIA do not respond to drugs that work for other kinds of arthritis. Research doctors have studied a chemical signal called IL-6 that the body uses to manage inflammation. This signal has been found to be very high in patients with active soJIA. A drug called tocilizumab (TCZ) has been designed to block IL-6. For about 6 years, TCZ has been tested in Japan for treating soJIA. It is now being tested in studies in the United States. These studies can have very strict rules for enrolling patients. This trial is a single-patient research study for a subject who otherwise does not meet the rules for enrollment in ongoing trials.

Studietype

Intervensjonell

Registrering (Faktiske)

1

Fase

  • Ikke aktuelt

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

  • Forente stater
    • Massachusetts
      • Boston, Massachusetts, Forente stater, 02111
        • Tufts Medical Center/Floating Hospital for Children

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

2 år til 16 år (Barn)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

Inclusion Criteria:

  • Systemic Juvenile Idiopathic Arthritis according to ILAR criteria (2001)
  • Duration of disease ≥ 6 months since onset
  • Presence of active disease as determined by the presence of at least 5 active joints, OR at least 2 active joints if receiving prednisone at a dose > 0.2 mg/kg/day or > 10 mg/day (whichever is less)
  • Incomplete prior response to methotrexate treatment for at least 3 months at a minimum dose of 15 mg/M2/week, or intolerance to methotrexate
  • Discontinued treatment with other biologics prior to first tocilizumab infusion, for approximately two pharmacokinetic half-lives as per specific biologic (e.g. 48 hours for anakinra, 7 days for etanercept)
  • Not receiving corticosteroids, OR taking oral corticosteroids and the dose has remained stable for 1 week prior to the first tocilizumab infusion at ≤ 2 mg/kg/day prednisone or prednisolone and no more than 80 mg/day

Exclusion Criteria:

  • Concomitant administration of biologic therapies
  • Serum creatinine >1.5 ULN (upper limits normal)
  • AST or ALT > 1.5 ULN
  • Total bilirubin > 1.3 mg/dL
  • Platelet count < LLN (lower limits normal)
  • Hemoglobin < 6.0 g/dL
  • WBC count < 5,000/mm3
  • Neutrophil count < 2,000/ mm3
  • Fibrinogen < LLN

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Tocilizumab
Single arm study - treatment only
Biologisk: tocilizumab

Initial therapy: Tocilizumab dosed by body weight (8mg/kg based on body weight ≥ 30kg) given by intravenous infusion every two weeks for 12 weeks.

Extension of therapy: Continuation of treatment with tocilizumab at 8mg/kg by body weight given by intravenous infusion every 2 weeks based upon achievement of Primary Objective by week 12, OR continuation of treatment with escalation of tocilizumab dose to 12mg/kg by body weight, given by intravenous infusion every two weeks, for failure to achieve ACR JIA30 at 12 weeks or ACR JIA50 response at any time after week 16.

Andre navn:
  • Actemra, RoACTEMRA, MRA

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Efficacy of Tocilizumab as Defined by Presence of an Equal to or Greater Than 30% Improvement in JIA Core Set (i.e. ACR JIA30 Response)
Tidsramme: At week 12 of treatment versus week 0 (pretreatment)
At week 12 of treatment versus week 0 (pretreatment)
Efficacy of Tocilizumab as Defined by Reduction of Oral Prednisone Dose by at Least 20%, or to Less Than 0.5mg/kg/Day, Whichever is of Lesser Daily Dose, While Maintaining an ACR JIA30 Response
Tidsramme: At weeks 12 and 16 of treatment versus week 0 (pretreatment)
At weeks 12 and 16 of treatment versus week 0 (pretreatment)
Number of Participants With at Least One Adverse Event
Tidsramme: Ongoing, throughout 24 month study period
To evaluate the safety of tocilizumab administration in this subject
Ongoing, throughout 24 month study period

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Measurement of Laboratory Parameters of Active Disease, Specifically C-reactive Protein, Hemoglobin, Platelets, White Blood Cell Count, Ferritin, Immunoglobulins.
Tidsramme: At weeks 8, 12, and 16 of treatment, and every 8-12 weeks thereafter
To assess normalization of laboratory parameters of active disease, specifically C-reactive protein, hemoglobin, platelets, white blood cell
At weeks 8, 12, and 16 of treatment, and every 8-12 weeks thereafter
Measurement of Sustained Clinical Response to Tocilizumab, Including Active Joint Count, Joints With Limited Range of Motion, and Absence of Fever or Rash.
Tidsramme: At weeks 8, 12, 16 of treatment, and every 8 weeks thereafter
To assess sustained clinical response to tocilizumab, including active joint count, joints with limited range of motion, and absence of fever
At weeks 8, 12, 16 of treatment, and every 8 weeks thereafter

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Tufts Medical Center

Samarbeidspartnere

Hoffmann-La Roche

Etterforskere

  • Hovedetterforsker: Marc D Natter, MD, Tufts Medical Center

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Generelle publikasjoner

Hjelpsomme linker

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart

1. mars 2009

Primær fullføring (Faktiske)

1. november 2009

Studiet fullført (Faktiske)

1. juni 2010

Datoer for studieregistrering

Først innsendt

24. mars 2009

Først innsendt som oppfylte QC-kriteriene

24. mars 2009

Først lagt ut (Anslag)

25. mars 2009

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

15. mai 2017

Siste oppdatering sendt inn som oppfylte QC-kriteriene

9. mai 2017

Sist bekreftet

1. mai 2017

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • TMC-PRHEU-TCZ-01

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

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