The Effect of Xolair ® (Omalizumab) in Mastocytosis Patients

Mastocytosis Clinical Trial

— XOLMA  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01333293
• Other study ID #: CIGE025ACH03T V1
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: July 20, 2009
• Last updated: January 15, 2016
• Start date: September 2011
• Est. completion date: January 2016

Study information

• Verified date: January 2016
• Source: University of Zurich
• Contact: n/a
• Is FDA regulated: No
• Health authority: Switzerland: Swissmedic
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 17
• Est. completion date: January 2016
• Est. primary completion date: October 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 70 Years

Eligibility

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 Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Mastocytosis

Intervention

Drug:
• injections
subcutaneous injections

Locations

Country	Name	City	State
Switzerland	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

Sponsors (1)

Lead Sponsor	Collaborator
University of Zurich

Country where clinical trial is conducted

Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Reduction of mast-cell induced adverse events and symptoms as summarized and calculated from patient's main complaint score and AFIRM score.		10 months	No
Secondary	Effect on the consumption or possibility to reduce mast-cell related drugs		10 months	No
Secondary	Effect on: - Lung function (FEV1), analysed by standard lung function measurements - blood pressure, - quantitative measurement of pressure-induced wheal and flare.		10 months	No
Secondary	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	No