Preventing Childhood Asthma Using Prophylactic House Dust Mite Allergen Immunotherapy (PAPA)

To establish efficacy and safety of HDM sublingual Immunotherapy (HDM-SLIT) by comparing Odactra and placebo, when given sublingually for 3 years to high risk infants aged between 6 to 12 months at enrollment in preventing the development of asthma, assessed 1.5 years after discontinuation of treatment.

Study Overview

• Status: Withdrawn
• Conditions: Asthma; Allergy
• Intervention / Treatment: Drug: HDM sublingual Immunotherapy; Drug: Placebo

Detailed Description

Aim to prevent the development of asthma in children by intervening at a time when the developing system in still amenable to immune modulation, by using the only therapy that has proven ability to change the natural history of allergic diseases and induce clinical remission. We hypothesize that three years of HDM Immunotherapy in high-risk infants will prevent the development of asthma assessed over a period of 1.5 years after discontinuation of treatment and HDM immunotherapy will result in (a) a significant reduction in the number and pathogenic features of HDM-reactive TH2 cells, and (b) an increase in the numbers of HDM-reactive TH and TREG cells expressing the interferon-response signature. The study is a Randomized (1:1), double blinded, placebo controlled, parallel arms, multi-site, Phase II study with a 3 year treatment phase and further 1.5 year follow-up phase. The study duration for each participant is 4.5 years. Overall study duration is 84 months (7 years); which include 9 months preparation and regulatory approvals, 15 months accrual, 36 months treatment and 18 months follow-up observation period.

• Study Type: Interventional
• Phase: Phase 2

Contacts and Locations

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 1 year (Child)
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

1. Aged 6 to 12 months of age
2. history of physician diagnosed atopic dermatitis OR
3. allergen sensitization OR
4. family history of asthma or allergy

Exclusion Criteria:

1. Evidence of sensitization to house dust mite (SPT ≥3 mm OR sIgE (> 0.35 kU/L)
2. Prematurity (<37 weeks)
3. Failure to thrive
4. Need for oxygen for more than 5 days in the neonatal period
5. history of intubation or mechanical ventilation

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Experimental arm; HDM sublingual Immunotherapy (HDM-SLIT) with Odactra® for 3 years to high-risk infants aged between 6 to 12 months at enrollment in preventing the development of asthma, assessed 1.5 years after discontinuation of treatment.	Drug: HDM sublingual Immunotherapy; HDM-SLIT tablet (Odactra®/Acarizax®) is a rapidly dissolving, freeze-dried, oral lyophilizate for oromucosal treatment, which contains a 1:1 mixture of two allergen extracts derived for the cultivated house dust mites, D. pteronyssinus and D. farinae. These species are included in SLIT tablet and subcutaneous immunotherapy administered for the treatment of allergic rhinitis and asthma in the pediatric and adult populations. The tablet dissolves within seconds after contact with a wet surface such as the oral mucosa. Participants will be administered the drug sublingually for 3 years.; Other Names: dust mite sublingual immunotherapy
Placebo Comparator: Placebo arm; Placebo administered sublingually for 3 years to high-risk infants aged between 6 to 12 months with outcome of asthma development assessed 1.5 years after discontinuation of treatment.	Drug: Placebo; Placebo sublingual therapy; Other Names: placebo dust mite sublingual immunotherapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants with Asthma	asthma diagnosis	1.5 years after discontinuation of 3 years of treatment

Collaborators and Investigators

• Sponsor: Boston Children's Hospital
• Collaborators: Emory University; Imperial College London; University of Southampton; University of North Carolina, Chapel Hill; George Washington University; Children's Hospital Medical Center, Cincinnati
• Investigators: Principal Investigator: Wanda Phipatanakul, MD, MS, Boston Children's Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): February 1, 2027
• Primary Completion (Estimated): February 1, 2028
• Study Completion (Estimated): August 1, 2029

Study Registration Dates

• First Submitted: October 19, 2021
• First Submitted That Met QC Criteria: October 28, 2021
• First Posted (Actual): November 9, 2021

Study Record Updates

• Last Update Posted (Estimated): November 26, 2024
• Last Update Submitted That Met QC Criteria: November 22, 2024
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Respiratory Tract Diseases; Lung Diseases; Bronchial Diseases; Lung Diseases, Obstructive; Respiratory Hypersensitivity; Hypersensitivity, Immediate; Hypersensitivity; Asthma; Physiological Effects of Drugs; Immunomodulating Agents; Immunologic Factors

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No