The Effect of Xolair ® (Omalizumab) in Mastocytosis Patients

Phase 2

Completed

• Conditions: Mastocytosis
• Interventions: Drug: injections

• Registration Number: NCT01333293
• Lead Sponsor: University of Zurich

Brief Summary

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

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 Timeline

• Study First Submitted: 2009-07-20
• Study First Submitted QC: 2011-04-08
• Study First Posted: 2011-04-11
• Study Start: 2011-09
• Primary Completion: 2015-10
• Status Verified: 2016-01
• Study Completion: 2016-01
• Last Update Submitted: 2016-01-15
• Last Update Posted: 2016-01-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 17

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	injections	-
Omalizumab	injections	-

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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
Effect on the consumption or possibility to reduce mast-cell related drugs	10 months

Trial Locations

Locations (3)

University Hospital Berne (Insel) and Zieglerspital Berne; 🇨🇭 Berne, Switzerland

Geneva University Hospitals and Medical Faculty of the University of Geneva; 🇨🇭 Geneva, Switzerland

Allergy Unit, Department of Dermatology, University Hospital Zurich; 🇨🇭 Zurich, Switzerland