Omalizumab for Childhood Asthma

(OBOE Trial)

Overseen By Alicia Mathis

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Stephen J. Teach, MD, MPH

Must not be taking: Monoclonal antibodies

Disqualifiers: Chronic conditions, Pregnancy, Latex allergy, others

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: 2

Duration: 3-6 days

300 Participants Needed

Trial Summary

Do I need to stop my current medications for the trial?

The trial does not specify if you need to stop your current medications, but it does mention that you should not use nasal corticosteroids or nasal vaccinations within 14 days before a specific visit. It's best to discuss your current medications with the study team.

What data supports the effectiveness of the drug Omalizumab for childhood asthma?

Research shows that Omalizumab can help children with moderate-to-severe asthma by reducing the need for oral corticosteroids (a type of medication that reduces inflammation) and improving lung function. It has been found to be safe and effective in managing asthma symptoms over the long term. ^{1 2 3 4 5}

Is omalizumab safe for children with asthma?

Omalizumab is generally considered safe for children with moderate-to-severe asthma, with most adverse events being mild to moderate. Studies show that it does not increase the frequency of serious adverse events compared to a placebo. ^{6 7 8 9 10}

How is the drug omalizumab different from other asthma treatments?

Omalizumab is unique because it is a monoclonal antibody that targets and reduces IgE (a protein involved in allergic reactions), helping to control severe allergic asthma in children who do not respond well to standard treatments like inhaled steroids. It can also reduce the need for oral corticosteroids and decrease asthma attacks and hospital visits. ^{2 11 12 13 14}

What is the purpose of this trial?

OBOE is a prospective, pilot, parallel group RCT with the overall aim of examining the effect of a single dose of anti-IgE (omalizumab) vs. placebo administered at the onset of URIs in the fall season among highly exacerbation-prone, urban, and atopic youth aged 6-17 years with persistent asthma. OBOE will recruit and randomize participants over 3 years (3 annual cohorts of participants). Recruitment for each of the yearly cohorts of OBOE will begin in February. Each cohort will be followed for a 2-6-month run-in period with the objective to gain control of each participant's asthma and to stabilize the required controller medication step level. Participants will receive routine asthma care every 1-2 months (a total of 2-4 times) during run-in using a previously described algorithm developed by the Inner-city Asthma Consortium and successfully employed in the PROSE study. The primary outcome is the change in the amount of nasal IFN-α recovered by nasal fluid absorption between two time points, within 72 hours of onset of a URI as defined by onset of (or substantial worsening of) rhinorrhea, nasal congestion or sneezing (single or multiple symptoms) and 3-6 days after study drug injection.

Research Team

Stephen Teach, MD. MPH.

Principal Investigator

Children's National Research Institute

Eligibility Criteria

This trial is for urban youth aged 6-17 with persistent asthma, who have had at least one severe asthma attack in the past six months. They must be sensitive to certain allergens, meet specific IgE levels and eosinophil counts, and not currently need steroids for respiratory symptoms. Participants can't join if they're pregnant or lactating, have a latex allergy, plan to homeschool during the study period, or have been treated with omalizumab recently.

Inclusion Criteria

I haven't used nasal sprays or had nasal vaccines in the last 2 weeks.

I don't need steroids for any lung problems right now.

My insurance covers medications that are commonly used for my condition.

Exclusion Criteria

You have had an allergic reaction to latex in the past.

I haven't had omalizumab, other monoclonal antibody, or allergy shots in the last 6 months.

Plan for home schooling during the 90-day outcome period

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Run-in

Participants are followed for a 2-6 month run-in period to gain control of asthma and stabilize medication levels

8-24 weeks

2-4 visits (in-person)

Treatment

Single dose of omalizumab or placebo administered at the onset of a viral upper respiratory infection

3-6 days

Follow-up

Participants are monitored for safety and effectiveness after treatment, including unscheduled healthcare utilization and asthma control

60-150 days

Treatment Details

Interventions

• Omalizumab
• Placebo

Trial Overview The OBOE trial tests whether a single dose of Omalizumab (anti-IgE) given at the start of upper respiratory infections can help prevent asthma exacerbations in high-risk children during fall. It's compared against a placebo over three years with participants monitored closely for changes in nasal interferon-alpha after treatment.

Participant Groups

Group I: Omalizumab Experimental Treatment 1 Intervention

Single dose of omalizumab at the start of a viral upper respiratory infection as defined by onset of (or substantial worsening of) rhinorrhea, nasal congestion or sneezing (single or multiple symptoms) during the fall outcome season (defined as the 90-day period beginning on each child's return to school)

Group II: Placebo for omalizumab Placebo Group 1 Intervention

Single dose of placebo for omalizumab at the start of a viral upper respiratory infection as defined by onset of (or substantial worsening of) rhinorrhea, nasal congestion or sneezing (single or multiple symptoms) during the fall outcome season (defined as the 90-day period beginning on each child's return to school)

Who Is Running the Clinical Trial?

Stephen J. Teach, MD, MPH

Lead Sponsor

Trials

4

Recruited

1,100+

National Institute of Allergy and Infectious Diseases (NIAID)

Collaborator

Trials

3,361

Recruited

5,516,000+

Findings from Research

In a study of 18 non-atopic asthmatics over 20 weeks, omalizumab therapy significantly reduced bronchial mucosal IgE+ cells, indicating a reduction in inflammation associated with asthma.

Patients receiving omalizumab maintained or improved their lung function after a staged withdrawal of their regular asthma therapy, while those on placebo experienced a decline in lung function.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Omalizumab reduces bronchial mucosal IgE and improves lung function in non-atopic asthma. Pillai, P., Chan, YC., Wu, SY., et al.[2022]

In a 16-week trial involving 34 children with severe asthma, omalizumab significantly reduced the median daily dose of oral prednisolone from 20 mg to 5 mg, with some children completely stopping prednisolone.

Improvements in asthma control and quality of life were observed, as indicated by significant increases in the mini-Asthma Quality of Life Questionnaire and Childhood Asthma Control Test scores, demonstrating the potential of omalizumab as a corticosteroid-sparing treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The oral corticosteroid-sparing effect of omalizumab in children with severe asthma. Brodlie, M., McKean, MC., Moss, S., et al.[2022]

A 52-week study involving 309 children with moderate-to-severe allergic asthma showed that long-term treatment with omalizumab is safe, with adverse events similar to those seen in the placebo group.

The most common side effects were upper respiratory infections and headaches, with no serious adverse events or anaphylactic reactions reported, indicating that omalizumab is well tolerated in this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Evaluation of long-term safety of the anti-IgE antibody, omalizumab, in children with allergic asthma. Berger, W., Gupta, N., McAlary, M., et al.[2015]

Omalizumab significantly reduces the rate of asthma exacerbations in children with moderate-to-severe asthma, with a notable odds ratio of 0.51 based on a review of three studies involving 1380 pediatric patients.

The treatment not only improves asthma control but also lowers the incidence of severe adverse events, indicating it is a safe option for long-term management of asthma in children.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of omalizumab in children with moderate-to-severe asthma: a meta-analysis. Fu, Z., Xu, Y., Cai, C.[2021]

Omalizumab is projected to significantly reduce asthma exacerbations by 30%, emergency room visits by 37%, and hospitalizations by 38% over 5 years for all eligible patients with moderate-to-severe allergic asthma.

For patients who respond to omalizumab, the treatment could lead to a 50% reduction in exacerbations, a 62% decrease in emergency visits, and a 63% drop in hospitalizations, while also allowing 45% of patients to lower their steroid dosage without losing asthma control.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Modeling the effects of omalizumab over 5 years among patients with moderate-to-severe persistent allergic asthma. Grossman, HL., Schlender, A., Alperin, P., et al.[2019]

In a pooled analysis of two studies involving 926 children aged 6 to under 12 with moderate-to-severe allergic asthma, omalizumab demonstrated a safety profile comparable to placebo, with fewer adverse events reported in the omalizumab group (89.7%) compared to the placebo group (91.7%).

Omalizumab may be a beneficial treatment option for children with asthma who do not respond adequately to standard therapies, as it shows an acceptable safety profile and potential efficacy in managing their condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and tolerability of omalizumab in children with allergic (IgE-mediated) asthma. Milgrom, H., Fowler-Taylor, A., Vidaurre, CF., et al.[2022]

Omalizumab significantly reduced the frequency of asthma exacerbations in pediatric patients, with 26.7% of those treated experiencing at least one exacerbation compared to 40.6% in the placebo group, indicating its efficacy as an add-on therapy.

The safety profile of omalizumab was acceptable, with similar rates of serious adverse events (5.2% for omalizumab vs. 5.6% for placebo) and no increased risk of hypersensitivity reactions or malignancies, based on data from three randomized controlled trials involving 1381 participants.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Systematic review on the use of omalizumab for the treatment of asthmatic children and adolescents. Rodrigo, GJ., Neffen, H.[2022]

Omalizumab significantly reduces the rate of asthma exacerbations and the need for inhaled corticosteroids and rescue medications in children with moderate-to-severe allergic asthma, based on both randomized clinical trials and real-world studies.

The treatment is well tolerated with no new safety concerns identified, and it also leads to improved lung function and reduced healthcare resource utilization, such as fewer hospitalizations and emergency visits.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of omalizumab in children and adolescents with moderate-to-severe asthma: A systematic literature review. Corren, J., Kavati, A., Ortiz, B., et al.[2022]

Omalizumab significantly improves asthma control and reduces levels of serum immunoglobulin E in children and adolescents aged 6-20 with moderate-to-severe allergic asthma, based on a meta-analysis of 13 studies.

While omalizumab is generally safe, with mild to moderate adverse events reported, it is effective as an add-on therapy, although it did not show significant improvements in certain lung function measures.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness and Safety Studies of Omalizumab in Children and Adolescents With Moderate-To-Severe Asthma. Cheng, L., Yang, T., Ma, X., et al.[2023]

Omalizumab significantly reduces the risk of asthma exacerbations and emergency visits in patients with refractory allergic asthma, based on a meta-analysis of 15 randomized controlled trials involving 6,316 patients.

The treatment is safe, showing no increase in adverse events compared to placebo, and it also improves asthma-related quality of life while allowing for a reduction in inhaled corticosteroid dosage.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Efficacy and safety of omalizumab in patients with refractory allergic asthma: a meta-analysis]. Chen, XQ., Jia, XY., Wu, JJ., et al.[2022]

Omalizumab is effective in improving asthma control for patients with moderate-to-severe uncontrolled asthma, leading to reduced reliance on inhaled steroids and fewer exacerbations, emergency visits, and hospitalizations.

The drug is also showing promise for treating other IgE-mediated conditions, such as allergic rhinitis and atopic dermatitis, although further research is needed to confirm its safety and efficacy in these areas.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Current status of therapy with omalizumab in children. Baena-Cagnani, CE., Gómez, RM.[2015]

After 2 years of treatment with omalizumab, children with severe asthma showed significant improvements in quality of life, as measured by the Pediatric Asthma Quality of Life Questionnaire (PAQLQ) and the Pediatric Asthma Caregiver's Quality of Life Questionnaire (PACQLQ).

While 63.3% of participants were able to reduce their daily inhaled corticosteroid (ICS) doses, there was no significant improvement in lung function (FEV1), indicating that omalizumab primarily enhances quality of life rather than directly improving lung function.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Quality of life in asthmatic children and their caregivers after two-year treatment with omalizumab, a real-life study. Sztafińska, A., Jerzyńska, J., Stelmach, W., et al.[2022]

In a study of 17 children and adolescents aged 8-16 with uncontrolled allergic asthma, omalizumab therapy significantly improved asthma control, with most patients reporting excellent or good responses to treatment.

After 104 weeks of treatment, there was a notable reduction in the use of oral corticosteroids and inhaled corticosteroids, along with a decrease in asthma exacerbations from an average of 5.59 to 0.53 per year, indicating improved management of asthma symptoms.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness of omalizumab in children and adolescents with uncontrolled allergic asthma: a case series from Poland. Sztafińska, A., Gwardys, M., Podlecka, D., et al.[2022]

In a post-marketing surveillance trial involving 280 patients with severe allergic asthma, omalizumab significantly reduced daily and nocturnal asthma symptoms by 76% and 84%, respectively, and decreased exacerbations by 82% over a 6-month period.

The majority of physicians (82%) and patients (86%) rated the efficacy of omalizumab as excellent or good, with only 7% of patients experiencing adverse events, indicating a favorable safety profile alongside its effectiveness.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Omalizumab in patients with severe persistent allergic asthma in a real-life setting in Germany. Korn, S., Thielen, A., Seyfried, S., et al.[2022]

References

1. England pubmed.ncbi.nlm.nih.gov

Omalizumab reduces bronchial mucosal IgE and improves lung function in non-atopic asthma. [2022]

2. England pubmed.ncbi.nlm.nih.gov

The oral corticosteroid-sparing effect of omalizumab in children with severe asthma. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Evaluation of long-term safety of the anti-IgE antibody, omalizumab, in children with allergic asthma. [2015]

4. England pubmed.ncbi.nlm.nih.gov

Efficacy and safety of omalizumab in children with moderate-to-severe asthma: a meta-analysis. [2021]

5. England pubmed.ncbi.nlm.nih.gov

Modeling the effects of omalizumab over 5 years among patients with moderate-to-severe persistent allergic asthma. [2019]

6. England pubmed.ncbi.nlm.nih.gov

Safety and tolerability of omalizumab in children with allergic (IgE-mediated) asthma. [2022]

7. England pubmed.ncbi.nlm.nih.gov

Systematic review on the use of omalizumab for the treatment of asthmatic children and adolescents. [2022]

8. United States pubmed.ncbi.nlm.nih.gov

Efficacy and safety of omalizumab in children and adolescents with moderate-to-severe asthma: A systematic literature review. [2022]

9. United States pubmed.ncbi.nlm.nih.gov

Effectiveness and Safety Studies of Omalizumab in Children and Adolescents With Moderate-To-Severe Asthma. [2023]

10. China pubmed.ncbi.nlm.nih.gov

[Efficacy and safety of omalizumab in patients with refractory allergic asthma: a meta-analysis]. [2022]

11. United States pubmed.ncbi.nlm.nih.gov

Current status of therapy with omalizumab in children. [2015]

12. Poland pubmed.ncbi.nlm.nih.gov

Quality of life in asthmatic children and their caregivers after two-year treatment with omalizumab, a real-life study. [2022]

13. Poland pubmed.ncbi.nlm.nih.gov

Effectiveness of omalizumab in children and adolescents with uncontrolled allergic asthma: a case series from Poland. [2022]

14. England pubmed.ncbi.nlm.nih.gov

Omalizumab in patients with severe persistent allergic asthma in a real-life setting in Germany. [2022]