A Clinical Safety Study of AT-100 (rhSP-D) in Preterm Neonates at High Risk for Bronchopulmonary Dysplasia (BPD)

• ClinicalTrials.gov Identifier: NCT04662151
• Recruitment Status: Recruiting
• First Posted: December 10, 2020
• Last Update Posted: February 21, 2023
• Sponsor: Airway Therapeutics, Inc.
• Information provided by (Responsible Party): Airway Therapeutics, Inc.

Study Description

Brief Summary:

The purpose of this study is to determine if an investigational drug, AT-100, can reduce the occurrence of Bronchopulmonary Dysplasia (BPD) in babies born premature, as compared to babies born premature who receive an air-sham alone.

Condition or disease	Intervention/treatment	Phase
Bronchopulmonary Dysplasia	Biological: AT-100; Procedure: Air-sham	Phase 1

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 36 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Intervention Model Description: Phase 1b portion is a randomized, dual-armed, dose escalation study to establish the safest & most tolerated AT-100 dose tested as compared to air-sham alone.
• Masking: Single (Participant)
• Primary Purpose: Prevention
• Official Title: A Phase 1b, Randomized, Blinded, Dose-Determined Study Evaluating the Safety and Tolerability Profile of Intervention With AT-100 (rhSP-D) in Preterm Neonates at High Risk for the Development of Bronchopulmonary Dysplasia (BPD)
• Actual Study Start Date: September 1, 2021
• Estimated Primary Completion Date: March 2023
• Estimated Study Completion Date: March 2023

Arms and Interventions

Arm	Intervention/treatment
Experimental: Phase 1b open-label AT-100; Once daily AT-100 via intratracheal administration for up to 2 doses (initial Phase 1b dose-escalation portion) or 7 doses (latter Phase 1b highest tolerated & safety dose level tested portion).	Biological: AT-100; reconstituted AT-100 for intratracheal administration; Other Name: (rhSP-D)
Sham Comparator: Phase 1b open-label air-sham; Once daily air-sham via intratracheal administration for up to 2 doses (initial Phase 1b dose-escalation portion) or 7 doses (latter Phase 1b highest tolerated & safety dose level tested portion).	Procedure: Air-sham; room air for intratracheal administration

Outcome Measures

Primary Outcome Measures :

1. Number of participants with treatment-related adverse events [ Time Frame: Adverse events will be followed up to Day 28 of life ]
   Incidence and severity of adverse events between the two treatment groups will be compared
2. Incidence of BPD or death [ Time Frame: Week 36 PMA ]

Eligibility Criteria

• Ages Eligible for Study: 0 Minutes to 96 Hours (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria

1. Preterm neonates born between Gestional Age (GA):

   1. 25 0/7 weeks to 28 6/7 weeks in the initial dose escalation cohorts.
   2. 23 0/7 weeks to 28 6/7 weeks in the latter cohort.
2. Intubated and on mechanical ventilation.
3. Receiving at least 1 dose of standard-of-care-indicated surfactant treatment (Curosurf®) after birth, and able to receive the first dose of AT-100 or air-sham within 96 hours of birth given at any time point after 15 minutes following any of the subject's Curosurf® dose(s).
4. Parent or legal guardian is able to provide informed consent.

Exclusion Criteria:

1. Weight at time of birth < 400 g or > 1,800 g.
2. Major apparent congenital abnormalities impacting cardio and pulmonary function.
3. Active DNR (Do Not Resuscitate) order in place.
4. Known pulmonary air leaks (e.g. pneumothorax and pneumomediastinum) at the time of AT-100 or air-sham administration.
5. History of allergy or sensitivity to any surfactant or any component of the Investigational Product (AT-100).
6. AT-100 or air-sham dosing was set to occur before Data Safety Monitoring Committee recommendation to proceed to the next dose-escalation cohort.

7. Use of minimally invasive surfactant techniques (e.g., LISA, MIST) or INSURE or if, in the opinion of the care team, the infant is very likely too be extubated shortly after receiving Curosurf®.

   a. Subjects extubated and re-intubated after their Curosurf® dose(s) are eligible, so long as the subject meets Inclusion #3.

8. Birth mother:

   1. Has known active Hepatitis B, C, or E diagnosis.
   2. Has a known illness or exposure that, in the judgement of the Investigator, is serious enough to induce an immune deficiency such as Human Immunodeficiency Virus (HIV) and/or is receiving chemotherapy.
   3. Has known active Sexually Transmitted Infection (STI).
   4. Has known Cytomegalovirus (CMV) active infection.
   5. Has known history or evidence of alcohol or drug abuse, wit the exception of marijuana/marijuana-based products/THC, based on a positive maternal or infant drug screen as evidenced by the institution's standard-of-care practice.
9. Concurrent enrollment in an investigational drug, device, or treatment modulation trial that utilizes treatments outside of standard-of-care.
10. Any condition or situation which, in the Investigator's judgement, puts the mother or the neonate at significant risk, could confound the trial results, or may interfere significantly with the mother's or neonate's participation in the trial.
11. Symptomatic and confirmed COVID-19 infection of the mother around the time of birth.

Contacts and Locations

Contacts

Contact: Janet Elkins, RN; 470-486-4687; elkins@airwaytherapeutics.com

Locations

United States, Arizona

Airway Therapeutics Investigational Site; Recruiting

Tucson, Arizona, United States, 85724

United States, Arkansas

Airway Therapeutics Investigational Site; Recruiting

Little Rock, Arkansas, United States, 72202

United States, California

Airway Therapeutics Investigational Site; Recruiting

Los Angeles, California, United States, 90017

United States, Florida

Airway Therapeutics Investigational Site; Recruiting

Miami, Florida, United States, 33143

United States, Georgia

Airway Therapeutics Investigational Site; Recruiting

Atlanta, Georgia, United States, 30308

United States, Indiana

Airway Therapeutics Investigational Site; Recruiting

Indianapolis, Indiana, United States, 46202

United States, Massachusetts

Airway Therapeutics Investigational Site; Recruiting

Boston, Massachusetts, United States, 02215

United States, North Carolina

Airway Therapeutics Investigational Site; Recruiting

Durham, North Carolina, United States, 27705

United States, Virginia

Airway Therapeutics Investigational Site; Recruiting

Norfolk, Virginia, United States, 23507

Spain

Airway Therapeutics Investigational Site; Recruiting

Cadiz, Andalucia, Spain, 11009

Airway Therapeutics Investigational Site; Recruiting

Barcelona, Cataluña, Spain, 08041

Airway Therapeutics Investigational Site; Recruiting

Madrid, Comunidad De Madrid, Spain, 28007

Airway Therapeutics Investigational Site; Recruiting

Madrid, Comunidad De Madrid, Spain, 28046

Airway Therapeutics Investigational Site; Recruiting

Alicante, Comunidad Valenciana, Spain, 03010

Airway Therapeutics Investigational Site; Recruiting

Valencia, Comunidad Valenciana, Spain, 46026

Airway Therapeutics Investigational Site; Recruiting

Santiago De Compostela, Galicia, Spain, 15706

Airway Therapeutics Investigational Site; Recruiting

A Coruña, Spain, 15006

Airway Therapeutics Investigational Site; Recruiting

Lleida, Spain, 25198

Airway Therapeutics Investigational Site; Recruiting

Málaga, Spain, 29010

Sponsors and Collaborators

Airway Therapeutics, Inc.

Investigators

• Study Chair: Marc O. Salzberg, MD; Airway Therapeutics, Inc.

More Information

Additional Information:

Airway Therapeutics' corporate website 

Publications:

Thebaud B, Goss KN, Laughon M, Whitsett JA, Abman SH, Steinhorn RH, Aschner JL, Davis PG, McGrath-Morrow SA, Soll RF, Jobe AH. Bronchopulmonary dysplasia. Nat Rev Dis Primers. 2019 Nov 14;5(1):78. doi: 10.1038/s41572-019-0127-7.

Jensen EA, Dysart K, Gantz MG, McDonald S, Bamat NA, Keszler M, Kirpalani H, Laughon MM, Poindexter BB, Duncan AF, Yoder BA, Eichenwald EC, DeMauro SB. The Diagnosis of Bronchopulmonary Dysplasia in Very Preterm Infants. An Evidence-based Approach. Am J Respir Crit Care Med. 2019 Sep 15;200(6):751-759. doi: 10.1164/rccm.201812-2348OC.

Sorensen GL. Surfactant Protein D in Respiratory and Non-Respiratory Diseases. Front Med (Lausanne). 2018 Feb 8;5:18. doi: 10.3389/fmed.2018.00018. eCollection 2018.

Sato A, Whitsett JA, Scheule RK, Ikegami M. Surfactant protein-d inhibits lung inflammation caused by ventilation in premature newborn lambs. Am J Respir Crit Care Med. 2010 May 15;181(10):1098-105. doi: 10.1164/rccm.200912-1818OC. Epub 2010 Feb 4.

Ikegami M, Carter K, Bishop K, Yadav A, Masterjohn E, Brondyk W, Scheule RK, Whitsett JA. Intratracheal recombinant surfactant protein d prevents endotoxin shock in the newborn preterm lamb. Am J Respir Crit Care Med. 2006 Jun 15;173(12):1342-7. doi: 10.1164/rccm.200509-1485OC. Epub 2006 Mar 23.

• Responsible Party: Airway Therapeutics, Inc.
• ClinicalTrials.gov Identifier: NCT04662151
• Other Study ID Numbers: AT-100/001
• First Posted: December 10, 2020
• Last Update Posted: February 21, 2023
• Last Verified: October 2022

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Airway Therapeutics, Inc.:

Bronchopulmonary Dysplasia; Preterm; Neonate; Mechanical ventilation; Respiratory support; recombinant human Surfactant Protein-D (rhSP-D); intratracheal; air-sham

Additional relevant MeSH terms:

Bronchopulmonary Dysplasia; Hyperplasia; Pathologic Processes; Ventilator-Induced Lung Injury; Lung Injury; Lung Diseases; Respiratory Tract Diseases; Infant, Premature, Diseases; Infant, Newborn, Diseases