Omalizumab for Preventing Asthma in Kids

(PARK Trial)

Not currently recruiting at 14 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether the medication omalizumab, an antibody treatment, can prevent asthma in preschool children aged 2-3 who are at high risk. The study compares children receiving omalizumab to those receiving a placebo to determine if it reduces asthma symptoms over time. Children who have experienced 2-4 wheezing episodes in the past year, are allergic to airborne allergens, and have a close family member with asthma or allergies may qualify for this trial. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group.

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.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that omalizumab is generally safe for children with asthma. In one study, children aged 6 to under 12 experienced fewer asthma attacks while using this treatment. Another review found that omalizumab is safe for children with asthma.

Specifically, past patients demonstrated that omalizumab can greatly improve asthma control and is a safe addition to asthma treatment plans for children. Most children in these studies tolerated the treatment well, with very few serious side effects reported. ^{1 2 3 4 5}

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

Most treatments for asthma in children, like inhaled corticosteroids or bronchodilators, work by reducing inflammation or relaxing the airways. Omalizumab is unique because it targets and neutralizes IgE antibodies, which play a key role in allergic reactions and asthma attacks. This novel mechanism of action can potentially prevent asthma symptoms before they start, rather than just managing them after they occur. Researchers are excited about omalizumab because it offers a proactive approach to asthma management, possibly reducing the frequency and severity of attacks in children.

What evidence suggests that omalizumab might be an effective treatment for preventing asthma in kids?

Studies have shown that omalizumab helps control asthma symptoms. In earlier research, about 25% of patients experienced good asthma control, and nearly 47% had partial control with this treatment. Another study found that the benefits of omalizumab lasted even a year after stopping the treatment. In children aged 6 to under 12, omalizumab significantly reduced asthma attacks over a year. These findings suggest it could help prevent asthma in young children at high risk. Participants in this trial will receive either omalizumab or a placebo to evaluate its effectiveness in preventing asthma in kids. ^{1 2 3 6 7}

Who Is on the Research Team?

Wanda Phipatanakul, MD. MSc.

Principal Investigator

Boston Children's Hospital

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

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]

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 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]

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9519799/

Long-term effectiveness and safety of omalizumab in ... Omalizumab treatment for a median duration of 32 months resulted in good asthma control in 25.2%, partial control in 47.1%, and poor control in 24.5% of ...

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

XOLAIR Clinical Results | Exacerbations | HCP The XOLAIR Effect: XOLAIR significantly improved daytime and nighttime symptom scores. 1.4x overall. Allergic asthma patients aged ≥12 years. ICS-stable phase ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11925863/

Efficacy of omalizumab after discontinuation The study confirmed the efficacy and safety of omalizumab, with its benefits persisting one year after treatment discontinuation.

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

Updated review of omalizumab to treat uncontrolled ... Results at the week 104 assessment (n = 78) showed that treatment with omalizumab significantly improved scores, with 85.9% of results classified as “excellent” ...

5. 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 ...

6. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0091674924012284

Updated review of omalizumab to treat uncontrolled ... Recent systemic reviews and RWSs support omalizumab as an effective and safe addition to asthma management strategies in treating severe AA in pediatric ...

7. publications.ersnet.org publications.ersnet.org/content/erj/42/5/1224

Add-on omalizumab in children with severe allergic asthma Omalizumab improved asthma control in children with severe allergic asthma and was generally well tolerated. The observed benefit was greater than that reported ...