Omalizumab for Preventing Asthma in Kids

(PARK Trial)

Recruiting at 12 trial locations

Overseen By Gabriel Federo, MD, MPH

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Boston Children's Hospital

Must not be taking: Corticosteroids, Biologics, Immunosuppressants, others

Disqualifiers: Prematurity, Cystic fibrosis, Immunodeficiency, 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: Unknown

Duration: 104 weeks

200 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is a randomized, double-blind, placebo controlled trial designed to test whether two years treatment of preschool children aged 2-3 years of age at high risk for asthma with omalizumab (anti-IgE) for two years will prevent the progression to childhood asthma, as reflected by a reduction in the prevalence of active asthma in the Final 12 months during 2 year observation period off study drug.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it excludes those using certain asthma therapies or who have had recent hospitalizations for respiratory symptoms. It's best to discuss your specific medications with the study team.

What data supports the effectiveness of the drug Omalizumab for preventing asthma in kids?

Research shows that Omalizumab helps improve asthma control in children with moderate to severe allergic asthma, especially when other treatments aren't enough. It has been used successfully as an additional therapy to reduce the need for oral corticosteroids (a type of medication that reduces inflammation) and improve asthma symptoms. ^{1 2 3 4 5}

Is omalizumab safe for children with asthma?

Omalizumab is generally considered safe for children with moderate-to-severe allergic asthma, with most side effects being mild to moderate. Studies have shown it is well-tolerated in children aged 6 and older. ^{4 5 6 7 8}

How is the drug omalizumab different from other asthma treatments for kids?

Omalizumab is unique because it is a monoclonal antibody that targets and inhibits immunoglobulin E (IgE), which plays a key role in allergic asthma. It is used as an add-on therapy for children aged 6 and older with moderate-to-severe allergic asthma, especially when other treatments like inhaled corticosteroids and long-acting β2-agonists are not enough. ^{3 6 8 9 10}

Research Team

Wanda Phipatanakul, MD. MSc.

Principal Investigator

Boston Children's Hospital

Eligibility Criteria

This trial is for preschool children aged 2-3 at high risk for asthma, who've had 2-4 wheezing episodes in the past year and a family history of asthma or allergy. They must not have used strong asthma medications recently, had frequent corticosteroid treatments, or have other significant medical conditions.

Inclusion Criteria

I have had 2-4 wheezing episodes in the last year.

A close family member has or had asthma or allergies.

I have been on a stable food allergy treatment not part of a trial for at least 2 months.

Exclusion Criteria

I have had more than 4 wheezing episodes in the last year.

I have previously received allergy shots or biologic treatments.

I am currently using a high-level asthma treatment.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive omalizumab or placebo for two years to prevent asthma progression

104 weeks

Observation

Participants are observed off study drug to assess asthma progression

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Omalizumab

Trial Overview The study tests if treating these kids with Omalizumab (anti-IgE) can prevent them from developing childhood asthma compared to a placebo. It's randomized and double-blind, meaning neither the researchers nor participants know who gets the real treatment.

Participant Groups

Group I: Active Active Control 1 Intervention

omalizumab 0.016 mg/kg/IU total IgE

Group II: Placebo Placebo Group 1 Intervention

looks like active drug

Who Is Running the Clinical Trial?

Boston Children's Hospital

Lead Sponsor

Trials

801

Recruited

5,584,000+

National Institute of Allergy and Infectious Diseases (NIAID)

Collaborator

Trials

3,361

Recruited

5,516,000+

Findings from Research

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]

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 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]

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]

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]

References

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

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

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

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

3. Poland pubmed.ncbi.nlm.nih.gov

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

4. 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]

5. United Arab Emirates pubmed.ncbi.nlm.nih.gov

Omalizumab in Children with Severe Allergic Asthma: The Italian Real-Life Experience. [2022]

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

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

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

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

8. England pubmed.ncbi.nlm.nih.gov

Efficacy and effectiveness of omalizumab in the treatment of childhood asthma. [2019]

9. 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]

10. England pubmed.ncbi.nlm.nih.gov

Omalizumab for pediatric asthma. [2021]