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

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by University of Zurich
Sponsor:
Information provided by (Responsible Party):
University of Zurich
ClinicalTrials.gov Identifier:
NCT01333293
First received: July 20, 2009
Last updated: March 3, 2014
Last verified: February 2014
  Purpose

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

Condition Intervention Phase
Mastocytosis
Drug: injections
Phase 2
Phase 3

Study Type: Interventional
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

Resource links provided by NLM:


Further study details as provided by University of Zurich:

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. [ Time Frame: 10 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 40
Study Start Date: September 2011
Estimated Study Completion Date: January 2016
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Omalizumab Drug: injections
subcutaneous injections
Placebo Comparator: Placebo Drug: injections
subcutaneous 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.

  Eligibility

Ages Eligible for Study:   18 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01333293

Contacts
Contact: 01 Studienregister MasterAdmins +41 (0)44 255 11 11

Locations
Switzerland
University Hospital Berne (Insel) and Zieglerspital Berne Not yet recruiting
Berne, Switzerland
Contact: Arthur Helbling, MD       Arthur.helbling@insel.ch   
Geneva University Hospitals and Medical Faculty of the University of Geneva Not yet recruiting
Geneva, Switzerland
Contact: Jörg Seebach, MD       joerg.seebach@hcuge.ch   
Allergy Unit, Department of Dermatology, University Hospital Zurich Recruiting
Zurich, Switzerland
Contact: Peter Schmid-Grendelmeier, MD    +41442551111    peter.schmid@usz.ch   
Sponsors and Collaborators
University of Zurich
Investigators
Study Director: 01 Studienregister MasterAdmins UniversitaetsSpital Zuerich
  More Information

No publications provided

Responsible Party: University of Zurich
ClinicalTrials.gov Identifier: NCT01333293     History of Changes
Other Study ID Numbers: CIGE025ACH03T V1
Study First Received: July 20, 2009
Last Updated: March 3, 2014
Health Authority: Switzerland: Swissmedic

Keywords provided by University of Zurich:
mastocytosis
omalizumab

Additional relevant MeSH terms:
Mastocytosis
Urticaria Pigmentosa
Mastocytosis, Cutaneous
Neoplasms
Neoplasms by Histologic Type
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Pigmentation Disorders
Skin Diseases
Omalizumab
Anti-Allergic Agents
Anti-Asthmatic Agents
Pharmacologic Actions
Respiratory System Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on November 25, 2014