The Effect of Xolair ® (Omalizumab) in Mastocytosis Patients (XOLMA)

The Effect of Xolair ® (Omalizumab) in Mastocytosis Patients Prospective Double-blind, Placebo-controlled, Multicentre Study, XOLMA-Study

Patients with mastocytosis often suffer from associated symptoms such as nausea, vertigo, fatigue, urticaria, abdominal cramps, diarrhea or hypotension due to release of mediators by mast cells. These patients have also an increased frequency of anaphylactic/anaphylactoid reactions due to allergens such as hymenoptera or nonspecific stimuli such as contrast media, local anesthetics or analgesics. In addition, there is increased osteoporosis in mastocytosis patients due to the activity of mast cell mediators on osteoblasts and osteoclasts. Symptoms of mastocytosis respond poorly to treatment with antihistamines or other antiallergic drugs. There is currently no specific treatment for this disease with the exception of rare cases. There are, however, some case reports suggesting that omalizumab might decrease symptoms including hypotensive events.

The aim of the study is to investigate whether patients suffering from mastocytosis benefit from a 6 month course of omalizumab with regard to symptoms and quality of life and whether the applied in vitro and in vivo monitoring tools represent useful surrogate markers for the efficacy of omalizumab in patients with mastocytosis.

• Trial with medicinal product

Study Overview

• Status: Completed
• Conditions: Mastocytosis
• Intervention / Treatment: Drug: injections

Detailed Description

The study will take place as double-blind placebo controlled study. After a first a run-period of 2 months for all participants randomization (1:1) in two group will take place:

Group A: With omalizumab treatment for 6 months; dosage and administration schedule according to body weight and total IgE level (1/2 of the patients). Group B: Placebo (1/2 of the patients). After 4 months of treatment in both groups patients are encouraged to stop all drugs given to reduce mast cell related effects, mainly antihistaminics . In case that disturbing symptoms are reoccurring patients are allowed to restart these drugs. The evaluation will take place after 5 months of treatment.

Finally, a follow up visit 1 and 4 months after the study will take place.

• Study Type: Interventional
• Enrollment (Actual): 17
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• Switzerland
  • Berne, Switzerland
    • University Hospital Berne (Insel) and Zieglerspital Berne
  • Geneva, Switzerland
    • Geneva University Hospitals and Medical Faculty of the University of Geneva
  • Zurich, Switzerland
    • Allergy Unit, Department of Dermatology, University Hospital Zurich

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion criteria:

• Histological proven mastocytosis (cutaneous or systemic);
• Diagnosis made by one marrow unction and/or skin biopsy or other histological work up;
• Age: 18-70 years

Exclusion criteria

• Age <18 years;
• Known hypersensitivity to omalizumab or any of its components;
• History of cancer in previous 5 years;
• Patients with serious infections;
• Patients with active tuberculosis or undergoing anti-TB therapy;
• Patients currently treated with systemic immunosuppressive agents;
• Female patients who are pregnant or breast feeding;Contraception must be performed by a save reliable and accept method such as oral or implanted contraceptives, intravaginal or male preservatives or permanent methods such as tubal ligation.
• Patients with known positivity for human immunodeficiency virus (HIV). HIV screening will be performed by an HIV 1/2 Antibody-detection test.

Note: Specific immunotherapy for insect sting allergy is no exclusion criteria.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: injections; subcutaneous injections
Experimental: Omalizumab	Drug: injections; subcutaneous injections

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Reduction of mast-cell induced adverse events and symptoms as summarized and calculated from patient's main complaint score and AFIRM score.	10 months

Secondary Outcome Measures

Outcome Measure	Time Frame
Effect on the consumption or possibility to reduce mast-cell related drugs	10 months
Effect on: - Lung function (FEV1), analysed by standard lung function measurements - blood pressure, - quantitative measurement of pressure-induced wheal and flare.	10 months
Effect on in-vitro parameters (Tryptase levels, density of Fc-IgE-R expression on basophils, Platelet-Activation-Factor (PAF) and cysteinyl leukotriene LTC4)	10 months

Collaborators and Investigators

• Sponsor: University of Zurich

Study record dates

Study Major Dates

• Study Start: September 1, 2011
• Primary Completion (Actual): October 1, 2015
• Study Completion (Actual): January 1, 2016

Study Registration Dates

• First Submitted: July 20, 2009
• First Submitted That Met QC Criteria: April 8, 2011
• First Posted (Estimate): April 11, 2011

Study Record Updates

• Last Update Posted (Estimate): January 18, 2016
• Last Update Submitted That Met QC Criteria: January 15, 2016
• Last Verified: January 1, 2016

More Information

Terms related to this study

• Keywords: omalizumab; mastocytosis
• Additional Relevant MeSH Terms: Skin Diseases; Immune System Diseases; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Hypersensitivity; Neoplasms, Connective Tissue; Immune Complex Diseases; Mastocytosis
• Other Study ID Numbers: CIGE025ACH03T V1