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Omalizumab to Treat Hyper-IgE (Job's) Syndrome

Primary Purpose

Hyper-IgE Syndrome, Job's Syndrome

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Omalizumab (Xolair)
Sponsored by
National Institute of Allergy and Infectious Diseases (NIAID)
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hyper-IgE Syndrome focused on measuring Phenotype, Monoclonal, Immunoglobulin, Immunodeficiency, Dermatitis, Job's Syndrome, Hyper-IgE Syndrome, HIES

Eligibility Criteria

6 Years - 76 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

-INCLUSION CRITERIA: All Subjects must be at least 6 years of age and no older than 76 years of age (as per original safety studies). All subjects must meet the established diagnostic criteria for HIES as determined by the NIH scoring system, with a score greater than 40, or have a mutation in the STAT3 gene. Baseline values within the following laboratory ranges: White blood cell count greater than or equal to 3,300 cells/microliter. Absolute neutrophil count greater than or equal to 1,000 cells/microliter. Hemoglobin greater than or equal to 10 g/dL. Platelet count greater than or equal to 100,000 platelets/micoliter. Women of childbearing potential only: negative urine pregnancy test. Both men and women and their partners must agree to practice abstinence or effective contraception from initiation of the protocol and for 2 months following the last dose of the study drug (effective contraception methods include abstinence, surgical sterilization of either partner, barrier methods such as diaphragm, condom, cap, or sponge, or hormonal contraception). Weight (Kg) times serum IgE (IU/mL) less than 3,000,000, then as above greater than 63,000, less than 3,000,000. Patients in tier II must be 16 years or older. Preference will be given to patients with dermatitis. Subjects (guardians for younger patients) must be able to give informed consent (or assent as appropriate). Subjects must already be signed to other NIH studies on HIES. Subjects must be willing to maintain their current regimens for skin care, prophylactic antibiotics (if applicable), and any asthma related oral or inhaled medications. Albuterol rescue medications may be used as needed. Patients participating in protocols at the National Institutes of Health are expected to have a primary physician outside of the NIH. EXCLUSION CRITERIA: Pregnant or nursing women. HIV positive diagnosis. Use of any other investigational agent within 30 days of the study. Any condition that, in the investigator's opinion, places the patient at undue risk by participating in the study. Previous anaphylaxis to the study medication.

Sites / Locations

  • National Institutes of Health Clinical Center, 9000 Rockville Pike

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
December 1, 2005
Last Updated
June 30, 2017
Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
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1. Study Identification

Unique Protocol Identification Number
NCT00260702
Brief Title
Omalizumab to Treat Hyper-IgE (Job's) Syndrome
Official Title
Pilot Study of Omalizumab (Xolair) in Hyper IgE (Job's) Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
March 15, 2010
Overall Recruitment Status
Completed
Study Start Date
November 24, 2005 (undefined)
Primary Completion Date
March 19, 2010 (Actual)
Study Completion Date
March 19, 2010 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)

4. Oversight

5. Study Description

Brief Summary
This study will evaluate the safety and effectiveness of a laboratory-made antibody called omalizumab in patients with Job's syndrome, or hyper-IgE syndrome (HIES). Patients with HIES have very high levels of IgE antibody, a protein made by white blood cells. IgE plays an important role in starting allergic reactions in the body and may be related to some HIES symptoms, such as skin rashes and asthma. Patients also have frequent lung infections, easily broken bones and other symptoms. Omalizumab, which is approved to treat allergic asthma, is directed against IgE. This study will see if blocking IgE with omalizumab in HIES patients is safe and if it can reduce patients' IgE count. It will also look at how the body handles omalizumab and how it affects patients' symptoms. Patients 6 years of age or older with HIES may be eligible for this study. Each candidate is screened with a medical history, physical examination, skin examination and blood test. Participants receive an injection of omalizumab under the skin once every 2 weeks for 6 doses. At the time of each injection, patients are examined by a doctor, answer questions about their symptoms and have a blood sample drawn. After the sixth dose, patients have a physical examination, blood tests, skin examination and lung function tests. At follow-up visits scheduled 2, 4 and 6 months after the last dose of omalizumab, patients have a physical examination, answer questions about their symptoms, and have a blood sample drawn. Patients who show a significant response to omalizumab stay off the drug for 3 months after the last dose and then discuss with their study doctor and referring doctor about continuing the medicine.
Detailed Description
The hyper-IgE syndromes (HIES) are disorder characterized by markedly elevated serum IgE levels, eosinophilia, dermatitis, and recurrent skin and lung infections. The autosomal dominant form of HIES, Job's syndrome, is also characterized by skeletal abnormalities and lung cysts, and is caused primarily by a mutation in the STAT3 gene. The most common allergic or atopic symptoms seen in HIES include a characteristic rash and bronchial hyper-responsiveness. The rash tends to respond to systemic antimicrobials, topical antiseptics, mad steroids. It is unclear what the role, if any, IgE plays in the pathogenesis of these allergic (and perhaps non-allergic) phenotypes. Omalizumab is a humanized monoclonal antibody specific for the FcepsilonRI portion of IgE that does not cross-link IgE. It has been used for amelioration of severe asthma and food allergies, and is being studied in a number of other allergic diseases. This study aims to determine whether administration of omalizumab is safe in patients with HIES, effective in inducing FcepsilonRI downregulation, and in reducing some of the cutaneous and/or respiratory symptoms associated with HIES. It also aims to determine whether the present maximum indicated dose has any effect on patients with HIES who have greater than indicated IgE levels. Finally, it aims to assess the safety and pharmacodynamic profile of increased doses of omalizumab likely to be required in most patients with HIES.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hyper-IgE Syndrome, Job's Syndrome
Keywords
Phenotype, Monoclonal, Immunoglobulin, Immunodeficiency, Dermatitis, Job's Syndrome, Hyper-IgE Syndrome, HIES

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Enrollment
1 (Actual)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Omalizumab (Xolair)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
76 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
-INCLUSION CRITERIA: All Subjects must be at least 6 years of age and no older than 76 years of age (as per original safety studies). All subjects must meet the established diagnostic criteria for HIES as determined by the NIH scoring system, with a score greater than 40, or have a mutation in the STAT3 gene. Baseline values within the following laboratory ranges: White blood cell count greater than or equal to 3,300 cells/microliter. Absolute neutrophil count greater than or equal to 1,000 cells/microliter. Hemoglobin greater than or equal to 10 g/dL. Platelet count greater than or equal to 100,000 platelets/micoliter. Women of childbearing potential only: negative urine pregnancy test. Both men and women and their partners must agree to practice abstinence or effective contraception from initiation of the protocol and for 2 months following the last dose of the study drug (effective contraception methods include abstinence, surgical sterilization of either partner, barrier methods such as diaphragm, condom, cap, or sponge, or hormonal contraception). Weight (Kg) times serum IgE (IU/mL) less than 3,000,000, then as above greater than 63,000, less than 3,000,000. Patients in tier II must be 16 years or older. Preference will be given to patients with dermatitis. Subjects (guardians for younger patients) must be able to give informed consent (or assent as appropriate). Subjects must already be signed to other NIH studies on HIES. Subjects must be willing to maintain their current regimens for skin care, prophylactic antibiotics (if applicable), and any asthma related oral or inhaled medications. Albuterol rescue medications may be used as needed. Patients participating in protocols at the National Institutes of Health are expected to have a primary physician outside of the NIH. EXCLUSION CRITERIA: Pregnant or nursing women. HIV positive diagnosis. Use of any other investigational agent within 30 days of the study. Any condition that, in the investigator's opinion, places the patient at undue risk by participating in the study. Previous anaphylaxis to the study medication.
Facility Information:
Facility Name
National Institutes of Health Clinical Center, 9000 Rockville Pike
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
8578348
Citation
Lee M, Hodges WG, Huggins TL, Lee EL. Eosinophilic gastroenteritis. South Med J. 1996 Feb;89(2):189-94. doi: 10.1097/00007611-199602000-00006.
Results Reference
background
PubMed Identifier
10634296
Citation
Kelly KJ. Eosinophilic gastroenteritis. J Pediatr Gastroenterol Nutr. 2000;30 Suppl:S28-35. doi: 10.1097/00005176-200001001-00005.
Results Reference
background
PubMed Identifier
8527050
Citation
Park HS, Kim HS, Jang HJ. Eosinophilic gastroenteritis associated with food allergy and bronchial asthma. J Korean Med Sci. 1995 Jun;10(3):216-9. doi: 10.3346/jkms.1995.10.3.216.
Results Reference
background

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Omalizumab to Treat Hyper-IgE (Job's) Syndrome

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