Efficacy and Safety of Zelpultide Alfa in Preterm Neonates at High Risk of Developing Bronchopulmonary Dysplasia (BPD)

NCT06897839 | Recruiting Phase 2 DMC

• 2 other identifiers: ZEL-0032024-513420-41-00
• Study Type: interventional
• Target: 366
• Locations: 3 countries
• Sites: 20

Brief Summary

This is a randomized, parallel-group, double-blind, placebo-controlled multicenter phase 2b/3 study with an adaptive seamless design. The goal fo this study is to determine if an investigational drug, Zelpultide Alfa, can reduce the occurrence of Bronchopulmonary Dysplasia (BPD) in extremely premature babies. The study comprises 2 parts:

• Part 1: Phase 2b, dose selection and exploratory efficacy and safety.
• Part 2: Phase 3, confirmatory efficacy and safety. In Part 1, the study subjects will be randomized with a 1:1:1 allocation ratio to either :
• Standard of care + zelpultide alfa 4 mg/kg or,
• Standard of care + zelpultide alfa 6 mg/kg or,
• Standard of care + placebo (air-sham).
• Standard of care + zelpultide alfa (selected dose from Part 1), or
• Standard of care + placebo (air-sham). The main objective in part 2 is to compare the efficacy of zelpultide alfa added to standard of care versus standard of care plus placebo (air-sham) in terms of incidence of grade 2 and grade 3 bronchopulmonary dysplasia (BPD) and death in neonates at high risk for developing BPD.

Conditions

Bronchopulmonary Dysplasia

Keywords

BPD; Zelpultide alfa; AT-100

Outcome Measures

Primary Outcomes (1)

• Incidence of grade 2 or grade 3 Bronchopulmonary Dysplasia (BPD) or death

  BPD is defined and graded based on the classification according to Jensen et al., 2019 (2019 PMID: 30995069)

  Week 36 Post Menstrual Age (PMA)

Secondary Outcomes (23)

• Ventilator-free days

  From birth to 36 weeks Post Menstrual Age (PMA)

• Incidence of grade 2 or grade 3 Bronchopulmonary Dysplasia (BPD)

  Week 36 Post Menstrual Age (PMA)

• Incidence of death

  Week 36 Post Menstrual Age (PMA)

• Incidence of grade 2 or grade 3 Bronchopulmonary Dysplasia (BPD) or death assessed on 3 consecutive days at week 36 PMA

  Week 36 Post Menstrual Age (PMA)

• The proportion of subjects with no Bronchopulmonary Dysplasia (BPD), grade 1, grade 2, or grade 3 BPD

  Week 36 Post Menstrual Age (PMA)

Study Arms (3)

Zelpultide alfa 4 mg/kg plus standard of care

EXPERIMENTAL

In addition to the preterm neonate standard of care, the subjects randomized to this treatment arm will receive up to 7 administrations of zelpultide alfa at 4 mg/kg (birth weight) in 24 h intervals while the subjects are still intubated.

Drug: Zelpultide alfa

Placebo (air-sham) plus standard of care

SHAM COMPARATOR

In addition to the preterm neonate standard of care, approximately 1 mL of air drawn into a dosing syringe will be administered up to 7 times to neonate subjects randomized to the control (placebo) arm, following the same means and intervals as in the treatment arm.

Other: Air-sham

Zelpultide alfa 6 mg/kg plus standard of care

EXPERIMENTAL

In addition to the preterm neonate standard of care, the subjects randomized to this treatment arm will receive up to 7 administrations of zelpultide alfa at 6 mg/kg (birth weight) in 24 h intervals while the subjects are still intubated.

Drug: Zelpultide alfa

Interventions

Air-sham OTHER

Room air for intratracheal administration

Placebo (air-sham) plus standard of care

Zelpultide alfa DRUG

Reconstituted Zelpultide alfa for intratracheal administration

Also known as: AT-100, rhSP-D

Zelpultide alfa 4 mg/kg plus standard of care; Zelpultide alfa 6 mg/kg plus standard of care

Eligibility Criteria

• Age: 0 Minutes - 96 Hours
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Born between gestational age (GA) 22 0/7 to 27 6/7 weeks, inclusive.
• Received at least 1 dose of SOC-indicated animal-derived pulmonary surfactant treatment after birth.
• Intubated and on invasive mechanical ventilation per SOC.
• Informed consent and personal information authorization form signed by the subject's parent(s) or legal guardian(s).

You may not qualify if:

• Birth weight \< 400 g or \> 1,500 g.
• Major apparent congenital abnormalities impacting cardio and pulmonary function identified before randomization, such as, but not limited to:
• Clinically relevant Potter-like syndrome and any pulmonary congenital anomalies,
• Clinically relevant congenital diaphragmatic hernia,
• Omphalocele or gastroschisis, esophageal atresia,
• Known or suspected cyanotic congenital heart disease (ie, tetralogy of fallot, transposition of the great arteries, etc).
• Active do no resuscitate (DNR) order in place.
• History of allergy or sensitivity to any surfactant or any component of zelpultide alfa.
• Concurrent enrollment in any clinical study that utilizes treatments (investigational medical products or devices) outside of SOC or participation in studies within the last 30 days (or 5 half-lives of an IMP) prior to birth (for the mother) or up to week 36 PMA.
• Any condition or situation that, in the Investigator's judgement, puts the neonate at significant risk, could confound the study results, or may interfere significantly with the neonate's participation in the study.

Sponsors & Collaborators

• Airway Therapeutics, Inc.: lead

Study Sites (20)

Bnai Zion Medical Center

Haifa, Israel

NOT YET RECRUITING

Shaare-Zedek Medical Center

Jerusalem, Israel

RECRUITING

Ziv Medical Center

Safed, Israel

RECRUITING

IRCCS Azienda Ospedaliero-Universitaria di Bologna - Policlinico di Sant'Orsola

Bologna, Italy

RECRUITING

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, Italy

NOT YET RECRUITING

Ospedale dei Bambini "V. Buzzi"

Milan, Italy

RECRUITING

Azienda Ospedale Università di Padova

Padua, Italy

RECRUITING

Hospital General Universitario de Alicante Dr. Balmis

Alicante, Spain

RECRUITING

Hospital Clínic de Barcelona

Barcelona, Spain

RECRUITING

Hospital de la Santa Creu i Sant Pau

Barcelona, Spain

RECRUITING

Hospital Sant Joan de Déu

Barcelona, Spain

RECRUITING

Hospital Universitario Cruces - OSI Ezkerraldea-Enkarterri-Cruces

Bilbao, Spain

RECRUITING

Hospital Universitario Puerta del Mar

Cadiz, Spain

RECRUITING

Hospital Universitari Arnau de Vilanova de Lleida

Lleida, Spain

RECRUITING

Hospital General Universitario Gregorio Marañón

Madrid, Spain

NOT YET RECRUITING

Hospital Universitario La Paz

Madrid, Spain

RECRUITING

Hospital Regional Universitario de Málaga

Málaga, Spain

RECRUITING

Hospital Clínico Universitario de Santiago

Santiago de Compostela, Spain

RECRUITING

Hospital Universitari i Politècnic La Fe

Valencia, Spain

RECRUITING

Hospital Universitario Miguel Servet

Zaragoza, Spain

RECRUITING

MeSH Terms

Conditions

Bronchopulmonary Dysplasia

Condition Hierarchy (Ancestors)

Ventilator-Induced Lung Injury; Lung Injury; Lung Diseases; Respiratory Tract Diseases; Infant, Premature, Diseases; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Alan Wolk

CONTACT

513-770-9630; info@airwaytherapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 20, 2025
• First Posted: March 27, 2025
• Study Start: February 3, 2025
• Primary Completion (Estimated): March 31, 2028
• Study Completion (Estimated): April 30, 2030
• Last Updated: February 6, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

IL (3); IT (4); ES (13)