Infasurf Aero™ for Neonatal Respiratory Distress Syndrome

(Aero-05 Trial)

Recruiting at 12 trial locations

Overseen By Dan Swartz, PhD

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: ONY

Disqualifiers: Congenital anomaly, Airway abnormality, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

Duration: Single dose

220 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of InfasurfAero™, a new method for delivering treatment to babies with Neonatal Respiratory Distress Syndrome (RDS). The treatment uses a nebulizer, a device that turns liquid medicine into a mist, allowing easier breathing without a tube. One group of infants will receive the actual treatment, while the other group will receive a placebo for comparison. Babies a few hours old, born between 29 to 36 weeks, and diagnosed with breathing problems may be suitable for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering a chance to contribute to a potentially groundbreaking treatment for infants.

Will I have to stop taking my current medications?

The trial information does not specify whether participants must stop taking their current medications.

What prior data suggests that the InfasurfAero™ device is safe for neonatal use?

Research shows that InfasurfAero™, which delivers calfactant to address breathing problems, has been studied for safety in newborns with respiratory distress syndrome. Previous studies have found that calfactant is generally well-tolerated in infants and has successfully reduced the need for more invasive procedures, such as inserting a tube into the airway.

In large studies where calfactant was administered as a mist, reports of serious side effects were few. Most infants handled the treatment well, and no unexpected safety concerns arose. Using calfactant as a mist, as in InfasurfAero™, aims to provide effective treatment without requiring a breathing tube, offering a less invasive and potentially safer option for these tiny patients.

In summary, current research supports the safety of InfasurfAero™ for treating infants with respiratory distress syndrome. This treatment is already being tested in advanced stages, indicating it has passed previous safety checks. While any medical treatment may carry risks, the data so far is reassuring. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike traditional treatments for Neonatal Respiratory Distress Syndrome, which often involve invasive procedures like intubation and direct surfactant administration, Infasurf Aero™ offers a non-invasive delivery method. This treatment uses a nebulizer to administer Calfactant, a surfactant, directly into the baby's lungs, potentially reducing the need for more invasive respiratory support. Researchers are excited about Infasurf Aero™ because it could make treatment safer and more comfortable for newborns, while still effectively delivering the medicine they need.

What evidence suggests that InfasurfAero™ is effective for Neonatal Respiratory Distress Syndrome?

Research has shown that InfasurfAero™ could help treat breathing problems in newborns. In this trial, participants in the treatment arm will receive a single dose of Calfactant at 6ml/kg, administered via the Infasurf Aero™ Nebulizer. Studies have found that the main ingredient, calfactant, when turned into a mist, can reduce the need for intubation in newborns by 50%. This mist delivery method is non-invasive and easier to use. Additionally, growing evidence suggests it helps premature babies breathe better. Although some studies have shown mixed results, the potential benefits make it an exciting option for those seeking simpler treatments. ^{2 3 6 7 8}

Who Is on the Research Team?

Scott Guthrie, MD

Principal Investigator

Vanderbilt Jackson-Madison, Jackson TN

Jim Cummings, MD

Principal Investigator

Ony Biotech

Dinushan Kaluarachchi, MD

Principal Investigator

Unity Point Meriter, Madison WI

Are You a Good Fit for This Trial?

This trial is for preterm newborns with a clinical diagnosis of surfactant-deficient RDS, needing non-invasive respiratory support, and weighing between 1,000 to 3,500 grams. They should be born at a gestational age of 29 to almost 37 weeks and are between 1 to 6 hours old. Newborns with airway abnormalities, prior mechanical ventilation or surfactant treatment, severe birth complications or major congenital anomalies cannot participate.

Inclusion Criteria

I am a newborn, less than 6 hours old.

My baby was born between 29 and 36 weeks of pregnancy.

I have been diagnosed with a severe lung condition needing treatment.

Exclusion Criteria

I have received surfactant treatment before being assigned to a study group.

Any condition that, in the opinion of the Investigator, would place the neonate at undue risk

My breathing problems are not due to newborn lung disease but another condition.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

A single dose of Calfactant at 6ml/kg administered via the Infasurf Aero™ Nebulizer or sham treatment with respiratory air alone

Single dose

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessment of CPAP failure and other respiratory outcomes

36 weeks post menstrual age

What Are the Treatments Tested in This Trial?

Interventions

• InfasurfAero™

Trial Overview The study tests the effectiveness and safety of Infasurf® delivered through InfasurfAero™ without the need for intubation in preterm newborns with RDS. It compares this novel delivery method against a sham treatment (placebo) while babies continue receiving nasal respiratory support.

How Is the Trial Designed?

Group I: Treatment Arm Experimental Treatment 1 Intervention

A single dose of Calfactant at 6ml/kg administered via the Infasurf Aero™ Nebulizer until completion.

Group II: Control Arm Placebo Group 1 Intervention

Low flow respiratory air alone through the InfasurfAero™ Nebulizer until completion.

Who Is Running the Clinical Trial?

ONY

Lead Sponsor

Trials

9

Recruited

1,400+

Avania

Industry Sponsor

Trials

59

Recruited

10,000+

Published Research Related to This Trial

In a study of 293 preterm infants with respiratory distress syndrome (RDS), treatment with 200 mg/kg of poractant alfa resulted in a faster reduction in the need for supplemental oxygen compared to beractant, indicating its efficacy in managing RDS.

Infants born at or below 32 weeks gestation who received 200 mg/kg of poractant alfa had significantly lower mortality rates (3%) compared to those treated with beractant (11%) or 100 mg/kg of poractant alfa, highlighting its potential safety and effectiveness in this vulnerable population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A randomized, multicenter masked comparison trial of poractant alfa (Curosurf) versus beractant (Survanta) in the treatment of respiratory distress syndrome in preterm infants. Ramanathan, R., Rasmussen, MR., Gerstmann, DR., et al.[2022]

In a study of 871 premature infants, Infasurf significantly reduced the incidence of neonatal respiratory distress syndrome (RDS) by 62% and RDS-related deaths by 70% compared to Exosurf Neonatal, indicating its superior efficacy in preventing RDS.

While Infasurf was more effective, it was associated with a higher overall risk of total intraventricular hemorrhage (IVH), although severe IVH rates were similar between the two treatments, highlighting a safety consideration in its use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A multicenter randomized masked comparison trial of synthetic surfactant versus calf lung surfactant extract in the prevention of neonatal respiratory distress syndrome. Hudak, ML., Martin, DJ., Egan, EA., et al.[2019]

In a study of 203 premature infants with respiratory distress syndrome (RDS), Survanta surfactant showed a significantly greater improvement in respiratory function compared to Exosurf from 12 to 48 hours after treatment.

Both Survanta and Exosurf were effective in treating RDS, but Survanta provided a faster response, while Exosurf was associated with a higher rate of retinopathy of prematurity in older infants (28 to 32 weeks).

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparative efficacy of exosurf and survanta surfactants on early clinical course of respiratory distress syndrome and complications of prematurity. Modanlou, HD., Beharry, K., Padilla, G., et al.[2018]

Citations

1. publications.aap.org publications.aap.org/pediatrics/article/146/5/e20193967/75345/Aerosolized-Calfactant-for-Newborns-With

Aerosolized Calfactant for Newborns With Respiratory ... This is the largest clinical trial evaluating the efficacy of aerosolized surfactant for preterm infants with respiratory distress syndrome.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8228799/

Aerosolized Surfactant for Preterm Infants with Respiratory ... There is increasing evidence suggesting that supraglottic administration via aerosolization is feasible and effective in preterm infants with RDS.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05960929

InfasurfAero™ Versus Sham Treatment in Preterm ... The purpose of this clinical study is to determine the effectiveness and safety of Infasurf® administered through the InfasurfAero™, a novel oral airway ...

4. onybiotech.com onybiotech.com/news/aerosolized-surfactant-the-trial-results-are-in/

Study Results are in on a Potential Non-Invasive Treatment ... This new method is non-invasive, and study results show it has the potential to reduce the need for intubation by 50 percent in babies.

5. researchgate.net researchgate.net/publication/347129567_Aerosolized_Calfactant_for_Newborns_With_Respiratory_Distress_A_Randomized_Trial

Aerosolized Calfactant for Newborns With Respiratory ... Aerosol delivery offers a safer alternative, but clinical studies have had mixed results. We hypothesized that efficient aerosolization of a ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12562553/

Comparison of Clinical Outcomes Between Calfactant and ... Comparison of Efficacy and Pharmacoeconomic Outcomes Between Calfactant and Poractant Alfa in Preterm Infants With Respiratory Distress Syndrome ...

7. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2024/020521s038lbl.pdf

INFASURF® (calfactant) intratracheal suspension The safety of INFASURF is based on the pooled safety population from three, randomized, active-controlled clinical trials that evaluated INFASURF to reduce the ...

8. onybiotech.com onybiotech.com/news/first-patient-enrolled-in-aero-05-study/

First Patient Enrolled in Aero-05 Study - ONY Biotech Research from earlier studies has shown that aerosolized surfactant can nearly halve the need for intubation in newborns experiencing early, ...