The Effect of Xolair ® (Omalizumab) in Mastocytosis Patients (XOLMA)
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 Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
|Official Title:||The Effect of Xolair ® (Omalizumab) in Mastocytosis Patients Prospective Double-blind, Placebo-controlled, Multicentre Study, XOLMA-Study|
- Reduction of mast-cell induced adverse events and symptoms as summarized and calculated from patient's main complaint score and AFIRM score. [ Time Frame: 10 months ] [ Designated as safety issue: No ]
- Effect on the consumption or possibility to reduce mast-cell related drugs [ Time Frame: 10 months ] [ Designated as safety issue: No ]
- Effect on: - Lung function (FEV1), analysed by standard lung function measurements - blood pressure, - quantitative measurement of pressure-induced wheal and flare. [ Time Frame: 10 months ] [ Designated as safety issue: No ]
- Effect on in-vitro parameters (Tryptase levels, density of Fc-IgE-R expression on basophils, Platelet-Activation-Factor (PAF) and cysteinyl leukotriene LTC4) [ Time Frame: 10 months ] [ Designated as safety issue: No ]
|Study Start Date:||September 2011|
|Estimated Study Completion Date:||February 2016|
|Estimated Primary Completion Date:||December 2015 (Final data collection date for primary outcome measure)|
|Placebo Comparator: Placebo||
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01333293
|University Hospital Berne (Insel) and Zieglerspital Berne|
|Geneva University Hospitals and Medical Faculty of the University of Geneva|
|Allergy Unit, Department of Dermatology, University Hospital Zurich|
|Study Director:||01 Studienregister MasterAdmins||UniversitaetsSpital Zuerich|