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Tocilizumab Treatment in Graves´ Ophthalmopathy (Graves´ Orbitopathy or Thyroid Eye Disease)

Phase 3
Completed
Conditions
Thyroid Associated Ophthalmopathies
Graves´ Ophthalmopathy
Thyroid Eye Disease
Interventions
Drug: Sterile 0.9% Sodium Chloride
Registration Number
NCT01297699
Lead Sponsor
Hospital Clinico Universitario de Santiago
Brief Summary

The purpose is to investigate tocilizumab administration in patients with moderately to severely or sight-threatening GO (Graves' ophthalmopathy) without response to treatment with corticoid intravenous pulses. Currently, these patients only have surgery as therapeutic alternative.

The principal aim of this study is to evaluate efficacy and safety of tocilizumab treatment in order to provide a better alternative to surgery for this patients.

Detailed Description

Thyroid orbitopathy or Graves´orbitopathy (GO) is manifested for inflammation of the orbital and periorbital soft tissues. Ten percent of patients with GO will have long-term serious eye problems.

The cause of the GO is complex. Autoreactive T cells recognize an autoantigen, the TSH receptor present in the orbit and thyroid follicular cell, secrete cytokines (IL-6), which stimulate the fibroblasts to synthesize glycosaminoglycan, which draws liquid produce periorbital edema and muscle, causing exophthalmos.

This approach suggests that inhibition of IL-6 can be an effective treatment for this problem on the basis that tocilizumab specifically binds to the receptors of the IL-6, both soluble and membrane-bound. Tocilizumab has been shown to inhibit signaling mediated for IL-6Rm IL-6Rs.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
32
Inclusion Criteria

Patients diagnosed GO with CAS ≥ 4 and an index of severity, according EUGOGO guidelines, 1. moderately to severely or 2. sight-threatening GO, after receiving corticosteroid pulse treatment due to:

  • Poor response to intravenous corticosteroid pulses Or
  • Recurrence of the GO, after treatment with intravenous corticosteroids
Exclusion Criteria
  • Orbital decompression surgery needed immediately
  • Active smoker
  • Patients who could need treatment with radioactive iodine or thyroidectomy during the study
  • Pregnant patient or patient who is planning to become pregnant during the study
  • History of chronic recurrent or active infection
  • History of intestinal ulceration or diverticulitis
  • Patients with a history of chronic liver disease or liver disorders: Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) above 5 times upper limit of normal (ULN)
  • History of HIV, hepatitis C or hepatitis B Positive
  • Neutrophil count < 0.5 × 109/L or a platelet count < 50×103/μL
  • Simultaneous use or contraindications to the use of immunosuppressive agents
  • A treatment with another investigational drug within four weeks of selection or five half-lives of study drug
  • Cardiovascular or cerebrovascular disease clinically significant
  • Uncontrolled diabetes mellitus
  • Use of corticosteroids during four weeks before to inclusion period
  • History of reactions or anaphylactic allergic severe human monoclonal antibodies, humanized or murine
  • Uncontrolled pathologies, whose exacerbations are treated with corticosteroids

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
TocilizumabTocilizumab (RoActemra®)-
Sterile 0.9% Sodium ChlorideSterile 0.9% Sodium Chloride-
Primary Outcome Measures
NameTimeMethod
Drug efficacy will be established as a decrease in CAS (clinical activity score) by 2 or more points in a scale of 10.It will be measured before treatment (week -4 / 0), after treatment (week 16) and after the end of the follow-up (week 40).
Secondary Outcome Measures
NameTimeMethod
Clinical response of patients who respond to treatment with tocilizumabIt will be analyzed before treatment (week -4 / 0), after treatment (week 16) and once to follow up (week 40).
Evaluate the patients quality of life associated with tocilizumab treatment, using SF-36It will be analyzed before treatment (week -4 / 0), after treatment (week 16) and once to follow up (week 40).
Determine time to recurrence during follow-up periodIt is measured on a weeks-time scale assessed by the physician during patient follow-up visits.
Safety as adverse events reported12 months

Trial Locations

Locations (9)

Hospital Universitario Virgen del Rocío

🇪🇸

Sevilla, Spain

Hospital Clínico de Santiago

🇪🇸

Santiago de Compostela, A Coruña, Spain

Hospital Clínico San Carlos

🇪🇸

Madrid, Spain

Hospital Santa Creu i Sant Pau

🇪🇸

Barcelona, Spain

Hospital Universitario Ramón y Cajal

🇪🇸

Madrid, Spain

Hospital Clínico Universitario Lozano Blesa

🇪🇸

Zaragoza, Spain

Hospital Universitario La Fe

🇪🇸

Valencia, Spain

Centro Oftalmológico Moreiras

🇪🇸

Santiago de Compostela, Spain

Hospital Universitario Virgen Macarena

🇪🇸

Sevilla, Spain

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