The Effect of Xolair ® (Omalizumab) in Mastocytosis Patients (XOLMA)
Primary Purpose
Mastocytosis
Status
Completed
Phase
Phase 2
Locations
Switzerland
Study Type
Interventional
Intervention
injections
Sponsored by
About this trial
This is an interventional treatment trial for Mastocytosis focused on measuring mastocytosis, omalizumab
Eligibility Criteria
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.
Sites / Locations
- 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
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Omalizumab
Placebo
Arm Description
Outcomes
Primary Outcome Measures
Reduction of mast-cell induced adverse events and symptoms as summarized and calculated from patient's main complaint score and AFIRM score.
Secondary Outcome Measures
Effect on the consumption or possibility to reduce mast-cell related drugs
Effect on: - Lung function (FEV1), analysed by standard lung function measurements - blood pressure, - quantitative measurement of pressure-induced wheal and flare.
Effect on in-vitro parameters (Tryptase levels, density of Fc-IgE-R expression on basophils, Platelet-Activation-Factor (PAF) and cysteinyl leukotriene LTC4)
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01333293
Brief Title
The Effect of Xolair ® (Omalizumab) in Mastocytosis Patients
Acronym
XOLMA
Official Title
The Effect of Xolair ® (Omalizumab) in Mastocytosis Patients Prospective Double-blind, Placebo-controlled, Multicentre Study, XOLMA-Study
Study Type
Interventional
2. Study Status
Record Verification Date
January 2016
Overall Recruitment Status
Completed
Study Start Date
September 2011 (undefined)
Primary Completion Date
October 2015 (Actual)
Study Completion Date
January 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Zurich
4. Oversight
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mastocytosis
Keywords
mastocytosis, omalizumab
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
17 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Omalizumab
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
injections
Intervention Description
subcutaneous injections
Primary Outcome Measure Information:
Title
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
Secondary Outcome Measure Information:
Title
Effect on the consumption or possibility to reduce mast-cell related drugs
Time Frame
10 months
Title
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
Title
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
01 Studienregister MasterAdmins
Organizational Affiliation
UniversitaetsSpital Zuerich
Official's Role
Study Director
Facility Information:
Facility Name
University Hospital Berne (Insel) and Zieglerspital Berne
City
Berne
Country
Switzerland
Facility Name
Geneva University Hospitals and Medical Faculty of the University of Geneva
City
Geneva
Country
Switzerland
Facility Name
Allergy Unit, Department of Dermatology, University Hospital Zurich
City
Zurich
Country
Switzerland
12. IPD Sharing Statement
Learn more about this trial
The Effect of Xolair ® (Omalizumab) in Mastocytosis Patients
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