Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

OIT and Xolair® (Omalizumab) in Cow's Milk Allergy

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Hugh.Sampson, Mount Sinai School of Medicine Identifier:
First received: July 2, 2010
Last updated: May 2, 2013
Last verified: May 2013

Food allergy affects up to 4% of the U.S. population and is most common in young children. Milk allergy is the most common cause of food allergy in infants and young children, and usually develops in the first year of life. There is no treatment for food allergy and the current standard of care for milk-allergic individuals is the avoidance of milk-containing products. Research is underway to identify potential therapeutic strategies to reduce or eliminate the adverse effects experienced by milk-allergic individuals when they consume milk-containing products.

Several studies have suggested that milk-allergic children who receive milk protein oral immunotherapy (OIT) may become desensitized to milk, resulting in short term protection against accidental ingestion of milk products. However, these children did not develop "tolerance," which is long term protection even after milk immunotherapy is stopped. A potential strategy to induce tolerance to milk uses milk in combination with Xolair® (omalizumab). Xolair consists of anti-IgE molecules that attach to IgE, the major antibody involved in allergic reactions. The goal of this clinical trial is to see whether Xolair® in combination with milk protein OIT is safer and more effective than OIT alone in inducing tolerance to milk and milk products. Participants will be administered a double blind, placebo controlled milk challenge at various time points in the study. If desensitization is achieved participants will be tested for tolerance at a certain time point after stopping treatment.

Condition Intervention Phase
Milk Allergy
Biological: Xolair® (omalizumab) placebo/milk OIT
Biological: Xolair® (omalizumab)/milk OIT
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Oral Immunotherapy Combined With Humanized Monoclonal Anti-IgE Antibody Xolair® (Omalizumab)in the Treatment of Cow's Milk Allergy

Resource links provided by NLM:

Further study details as provided by Mount Sinai School of Medicine:

Primary Outcome Measures:
  • Percentage of subjects in the Xolair® (omalizumab)group vs. placebo group developing clinical tolerance to milk [ Time Frame: 8 weeks following the discontinuation of milk-OIT and 4 months after discontinuing the Xolair (omalizumab) / placebo for Xolair ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Incidence of dosing reactions to milk OIT [ Time Frame: During the Escalation and Maintenance Phases ] [ Designated as safety issue: Yes ]
  • Incidence of severe hypersensitivity reactions to milk OIT [ Time Frame: Throughout the study ] [ Designated as safety issue: Yes ]
  • Maximum tolerated dose of milk oral immunotherapy and the time required to achieve it [ Time Frame: 22-40 weeks ] [ Designated as safety issue: Yes ]
  • Percentage of subjects developing desensitization to milk [ Time Frame: at month 28 ] [ Designated as safety issue: No ]
  • Change in endpoint milk skin test titration and mechanistic assays to evaluate biologic responses [ Time Frame: at months 32 ] [ Designated as safety issue: No ]

Estimated Enrollment: 76
Study Start Date: August 2010
Estimated Study Completion Date: July 2015
Estimated Primary Completion Date: January 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Xolair® (omalizumab)/milk OIT
Xolair for 4 months followed by 12 months of milk OIT. After unblinding, will continue receiving Xolair along with milk OIT for an additional 12 months.
Biological: Xolair® (omalizumab)/milk OIT
Xolair for 4 months followed by 12 months of milk OIT. After unblinding, will continue receiving Xolair along with milk OIT for an additional 12 months.
Placebo Comparator: Xolair® (omalizumab) placebo/milk OIT
Placebo for Xolair for 4 months followed by 12 months of milk OIT. after unblinding, will discontinue placebo for Xolair and continue milk OIT for an additional 12 months.
Biological: Xolair® (omalizumab) placebo/milk OIT
Placebo for Xolair for 4 months followed by 12 months of milk OIT. after unblinding, will discontinue placebo for Xolair and continue milk OIT for an additional 12 months.
Other Name: Xolair® (omalizumab) placebo/milk OIT
No Intervention: Untreated control
no intervention.


Ages Eligible for Study:   7 Years to 35 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Subject and/or parent/ legal guardian must be able to understand and provide written informed consent
  • Written or verbal assent from all study subjects less than 18 years (per site IRB regulations)
  • 7 to 35 years of age; any gender; any racial and ethnic origin
  • No known contraindications to therapy using oral immunotherapy with milk protein or Xolair® (omalizumab)
  • All female subjects of childbearing potential must have a negative pregnancy test upon study entry
  • All treated females of childbearing potential must agree to use FDA approved methods of birth control for the duration of the study

Active Treatment Subjects:

  • Cow's milk allergy confirmed by a positive double-blind placebo controlled milk challenge (DBPCMC) to a dose of less than 2 g of milk protein within the past 6 months
  • A skin prick test positive to milk (diameter of wheal >= 3.0 mm) OR detectable serum milk specific IgE level within the previous 12 months (UniCAP > = 0.35 kUA/L)

Control Subjects:

• A skin prick test positive to milk (diameter of wheal >= 10.0 mm) OR detectable serum milk specific IgE level within the previous 12 months (UniCAP >= 15 kUA/L)

Exclusion Criteria:

  • A history of life-threatening anaphylaxis to milk (involving hypotension or requiring mechanical ventilation)
  • Known allergy to any components of the placebo for Xolair®
  • Chronic disease other than asthma, atopic dermatitis, or allergic rhinitis requiring therapy (e.g., heart disease, diabetes)
  • Use of β-blockers (oral), angiotensin-converting enzyme (ACE) inhibitors, angiotensin-receptor blockers (ARB), or calcium channel blockers
  • Severe asthma
  • Mild or moderate asthma with any of the following criteria met:

    • FEV1<80% with or without controller medications
    • ICS dosing of >500 mcg daily fluticasone (or equivalent inhaled corticosteroids based on NHLBI dosing chart)
    • history of daily oral steroid dosing for >1 month during the past year
    • burst oral steroid course in the past 6 months
    • more than one burst oral steroid course in the past year
    • more than one hospitalization in the past year for asthma, or
    • more than one ER visit in the past 6 months for asthma
  • Baseline spirometry (or PFR if unable to perform spirometry) result of FEV1<80%
  • Pregnancy or lactation. All females of child-bearing age will undergo pregnancy testing. All treated females will confirm compliance to appropriate birth control measures throughout the course of the study;
  • Participation in any interventional study for the treatment of food allergy in the past 6 months
  • Subject is on a buildup phase of standard subcutaneous immunotherapy for inhalant allergens (may be enrolled on maintenance dose);
  • Use of Xolair® (omalizumab) or other non-traditional forms of allergen immunotherapy (e.g., oral or sublingual immunotherapy) or immunomodulator therapy (not including corticosteroids) or biologic therapy within the past year
  • Inability to discontinue antihistamines for 5 half-lives prior to routine study tests (DBPCMC or endpoint titration tests)
  • Known sensitivity to Xolair® (omalizumab) or to the class of study drugs
  • Baseline serum total IgE over 1,300 IU/mL or body weight more than 150 kg, or subjects with weight-IgE combination that yields a dose requirement greater than 750 mg (due to limitations of Xolair® (omalizumab) dosing)
  • Mental illness or history of drug or alcohol abuse that, in the opinion of the investigator, would interfere with the subject's ability to comply with study requirements
  • Inability or unwillingness of a subject to give written informed consent or comply with study protocol
  • Use of investigational drugs within 90 days of participation
  • Other contraindications to milk oral immunotherapy or Xolair® (omalizumab)
  • Recipient of any licensed or investigational live attenuated vaccine(s) within 2 months of enrollment
  • Families who do not speak English
  • Systemic steroids oral, IM, or IV for indications other than asthma for greater than 3 weeks in the past 6 months
  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 identifier: NCT01157117

United States, California
Stanford University School of Medicine
Stanford, California, United States, 94305
United States, Maryland
Johns Hopkins University School of Medicine
Baltimore, Maryland, United States, 21287
United States, New York
Icahn School of Medicine at Mount Sinai
New York, New York, United States, 10029
Sponsors and Collaborators
Study Chair: Hugh A. Sampson, M.D. Mount Sinai School of Medicine
  More Information

No publications provided

Responsible Party: Hugh.Sampson, Dean for Translational Biomedical Sciences, Director, Jaffe Food Allergy Institute, Mount Sinai School of Medicine Identifier: NCT01157117     History of Changes
Other Study ID Numbers: DAIT AADCRC-MSSM-01, U19AI044236
Study First Received: July 2, 2010
Last Updated: May 2, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Mount Sinai School of Medicine:
food allergy

Additional relevant MeSH terms:
Milk Hypersensitivity
Food Hypersensitivity
Hypersensitivity, Immediate
Immune System Diseases
Anti-Allergic Agents
Anti-Asthmatic Agents
Pharmacologic Actions
Respiratory System Agents
Therapeutic Uses processed this record on February 27, 2015