Omalizumab for Childhood Asthma

(OBOE Trial)

Recruiting at 1 trial location

Overseen By William Sheehan, MD

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Children's National Research Institute

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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether a single dose of the medication omalizumab can reduce asthma flare-ups in children during the fall, particularly when they catch a cold. The study includes children aged 6-17 with persistent asthma who have experienced at least one severe asthma attack requiring medication in the past year. Participants will receive either omalizumab or a placebo (a substance with no active medication) when they develop cold symptoms like a runny or congested nose. The goal is to determine if omalizumab can improve asthma symptoms during colds. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group of participants.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that omalizumab, a treatment for asthma, is generally safe for children. In studies, children taking this treatment did not experience serious side effects. Long-term use in everyday situations also supports its safety, with many children achieving better asthma control. Although all medications can have some side effects, evidence suggests that omalizumab is a safe option for managing asthma in children. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for asthma?

Unlike standard asthma treatments that often require daily use, like inhaled corticosteroids or leukotriene modifiers, Omalizumab offers a unique approach by targeting specific immune system components. It’s an antibody that blocks immunoglobulin E (IgE), a key player in allergic reactions that can worsen asthma symptoms. Researchers are particularly excited because Omalizumab is administered as a single dose at the start of a viral upper respiratory infection, potentially providing rapid relief during critical times like the fall when children return to school and are more susceptible to colds. This targeted approach could offer a new way to manage asthma exacerbations with greater precision and convenience.

What evidence suggests that omalizumab might be an effective treatment for childhood asthma?

Research has shown that omalizumab works well for children with asthma. One study greatly reduced asthma attacks in kids aged 6 to 11. Another study found that omalizumab lowered the number of asthma flare-ups compared to a placebo, leading to better asthma control. In this trial, some participants will receive a single dose of omalizumab, while others will receive a placebo. Long-term use of omalizumab has helped many children keep their asthma in check. Overall, omalizumab has a strong history of helping manage asthma symptoms in kids. ^{1 3 5 6 7}

Who Is on the Research Team?

William Sheehan, MD

Principal Investigator

Children's National Research Institute

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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?

Children's National Research Institute

Lead Sponsor

Trials

227

Recruited

258,000+

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+

Published Research Related to This Trial

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]

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]

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 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]

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]

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]

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]

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]

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 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 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]

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]

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]

Citations

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Long-term effectiveness and safety of omalizumab in ... In severe allergic asthmatic children who had received omalizumab for 24 months, 76.7% of 30 children were controlled who were still on omalizumab ...

2. xolair.com xolair.com/allergic-asthma/results/clinical-trial-results.html

Clinical Trials | XOLAIR® (omalizumab) for Allergic Asthma In children ages 6 to less than 12 years old, XOLAIR significantly reduced asthma attacks. In a 52-week study in children with moderate to severe persistent ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10020639/

Severe pediatric asthma therapy: Omalizumab—A ... Omalizumab therapy was effective in decreasing the rate of asthma exacerbations compared to placebo [OR 0.44; 95% CI(0.35, 0.56), P < 0.001] (Figure 2). 3.1.2.

4. jacionline.org jacionline.org/article/S0091-6749(24)01228-4/fulltext

Updated review of omalizumab to treat uncontrolled ... 001) after 2 years of treatment; asthma was controlled in 58% of children, partially controlled in 38%, and uncontrolled in 4% (compared with 90% uncontrolled ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S2213219821000672

Real-World Effectiveness of Omalizumab in Severe Allergic ... ACT score significantly improved after omalizumab treatment at 16 weeks (MD [95% CI]: 4.44 [3.55-5.34]; I2 = 71%), 6 months (MD [95% CI]: 3.98 [2.74-5.23]; I2 = ...

6. xolairhcp.com xolairhcp.com/allergic-asthma/efficacy/clinical-trial-results.html

XOLAIR Clinical Results | Exacerbations | HCP 15.9% of patients taking XOLAIR + ICS (n=126) experienced ≥1 exacerbation vs 15% of those taking placebo + ICS (n=120).

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10381149/

Long-Term Safety of Omalizumab in Children with Asthma and ... Conclusions: Long-term “real-life” treatment with omalizumab in children appears well tolerated. Its safety and efficacy profile makes ...