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

Phase 2

Completed

• Conditions: Systemic mastocytosis; Mastocytosis in the skin; Blood - Other blood disorders; Skin - Other skin conditions

• Registration Number: ACTRN12613001096741
• Lead Sponsor: ovartis Pharmaceuticals

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2013-10-01
• Last Update Posted: 13 January 2020

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

1. Over 18 years of age;
2. 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.);
3. Breakthrough symptoms of disease despite maximal tolerated conventional therapy.

Exclusion Criteria

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

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Improvement in AFIRMM score (standardised and validated symptom score for mastocytosis)[<br>4 weeks after final dose (week 24)]

Secondary Outcome Measures

Name	Time	Method
Improvement in VAS (symptom score)[<br>4 weeks after last dose (week 24)];Improvement in standardised clinical assessment for mastocytosis(SCORMA) [<br>4 weeks after last dose (week 24)];Improvement in standardised score for clinical skin photography as assessed by a dermatologist[<br>4 weeks after last dose (week 24)];Improvement in laboratory parameters relating to mastocytosis including tryptase, routine haematology and biochemistry, bone turnover markers, basophil activation and serum cytokines[<br>4 weeks after last dose (week 24)]