CompletedPhase 2Phase 3ACTRN12613001096741

A randomized, placebo-controlled, double blind, parallel design study of Xolair (omalizumab) for the management of treatment-resistant systemic and cutaneous mastocytosis.

In patients with systemic and/or cutaneous mastocytosis, is omalizumab (Xolair) in addition to standard care more effective in the management of symptoms than standard care alone? A randomized, placebo-controlled, double blind, parallel design study


Sponsor

Novartis Pharmaceuticals

Enrollment

30 participants

Start Date

Nov 5, 2014

Study Type

Interventional

Conditions

Summary

The study aims to assess the effect of omalizumab (Xolair) on systemic and cutaneous mastocytosis


Eligibility

Sex: Both males and femalesMin Age: 18 Yearss

Inclusion Criteria6

  • Over 18 years of age;
  • A diagnosis of Indolent Systemic Mastocytosis, OR
  • Smouldering Systemic Mastocytosis, OR
  • Aggressive Systemic Mastocytosis, OR
  • Cutaneous Mastocytosis as per WHO current guidelines, with satisfaction of Valent 2007 criteria for Mastocytosis in the Skin (Valent P, Akin C, Escribano L et. al. Standards and Standardization in Mastocytosis: Consensus Statements on Diagnosis, Treatment Recommendations and Response Criteria. Eur J Clin Invest 2007, 37 (6); 435 – 453.);
  • Breakthrough symptoms of disease despite maximal tolerated conventional therapy.

Exclusion Criteria8

  • Pregnancy (a beta-HCG and pregnancy counseling will be performed for all female patients of child-bearing age).
  • Terminal illness
  • Associated haematological clonal non-mast cell lineage disease or mast cell leukaemia
  • Beta blocker use
  • Unable to give consent
  • Known sensitivity to study drug(s) or class of study drug(s)
  • Patients with severe medical condition(s) that in the view of the investigator prohibits participation in the study (specify as required)
  • Use of any other investigational agent in the last 30 days

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Interventions

Omalizumab (Xolair) 375mg subcutaneously every 4 weeks for 6 months Trial medication with be administered in hospital

Omalizumab (Xolair) 375mg subcutaneously every 4 weeks for 6 months Trial medication with be administered in hospital


Locations(1)

Royal Melbourne Hospital - City campus - Parkville

VIC, Australia

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ACTRN12613001096741