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

• Sponsor: Airway Therapeutics, Inc. (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT04662151
• Collaborator: (none)
• Enrollment: 36
• Locations: 18
• Arms: 2
• Anticipated Duration (Months): 17.9
• Patients Per Site: 2
• Patients Per Site Per Month: 0.1

Study Details

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
• Anticipated 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.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: Sep 1, 2021
• Anticipated Primary Completion Date: Mar 1, 2023
• Anticipated Study Completion Date: Mar 1, 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 Names: (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 adverse events experienced by participants [From time of initial AT-100 or Air-Sham administration through Day 28 of Life]

   Comparison of the number of adverse events experienced by participants in the group receiving AT-100 versus the group receiving Air-Sham.

2. Severity of adverse events experienced by participants [From time of initial AT-100 or Air-Sham administration through Day 28 of Life]

   Comparison of the severity of adverse events (categorized as Mild, Moderate, Severe, Life-Threatening, or Death) experienced by participants in the group receiving AT-100 versus the group receiving Air-Sham.

Secondary Outcome Measures

1. Incidence of BPD or death [From birth to Week 36 PMA]

   Number of participants that are diagnosed with BPD at the Week 36 PMA timepoint or that experience death at or before the Week 36 PMA timepoint.

2. Time on mechanical ventilation [From birth to 36 Weeks PMA]

   Time (days) on mechanical ventilation.

Eligibility Criteria

Criteria

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

Inclusion Criteria

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

2. 25 0/7 weeks to 28 6/7 weeks in the initial dose escalation cohorts.

3. 23 0/7 weeks to 28 6/7 weeks in the latter cohort.

4. Intubated and on mechanical ventilation.

5. 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).

6. 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®.

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

1. Birth mother:

2. Has known active Hepatitis B, C, or E diagnosis.

3. 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.

4. Has known active Sexually Transmitted Infection (STI).

5. Has known Cytomegalovirus (CMV) active infection.

6. 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.

7. Concurrent enrollment in an investigational drug, device, or treatment modulation trial that utilizes treatments outside of standard-of-care.

8. 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.

9. Symptomatic and confirmed COVID-19 infection of the mother around the time of birth.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Airway Therapeutics Investigational Site	Tucson	Arizona	United States	85724
2	Airway Therapeutics Investigational Site	Little Rock	Arkansas	United States	72202
3	Airway Therapeutics Investigational Site	Los Angeles	California	United States	90017
4	Airway Therapeutics Investigational Site	Orange	California	United States	92868
5	Airway Therapeutics Investigational Site	Miami	Florida	United States	33143
6	Airway Therapeutics Investigational Site	Atlanta	Georgia	United States	30308
7	Airway Therapeutics Investigational Site	Indianapolis	Indiana	United States	46202
8	Airway Therapeutics Investigational Site	Boston	Massachusetts	United States	02215
9	Airway Therapeutics Investigational Site	Durham	North Carolina	United States	27705
10	Airway Therapeutics Investigational Site	Nashville	Tennessee	United States	37232
11	Airway Therapeutics Investigational Site	Norfolk	Virginia	United States	23507
12	Airway Therapeutics Investigational Site	Cadiz	Andalucia	Spain	11009
13	Airway Therapeutics Investigational Site	Barcelona	Cataluña	Spain	08041
14	Airway Therapeutics Investigational Site	Madrid	Comunidad De Madrid	Spain	28007
15	Airway Therapeutics Investigational Site	Madrid	Comunidad De Madrid	Spain	28046
16	Airway Therapeutics Investigational Site	Alicante	Comunidad Valenciana	Spain	03010
17	Airway Therapeutics Investigational Site	Valencia	Comunidad Valenciana	Spain	46026
18	Airway Therapeutics Investigational Site	Santiago De Compostela	Galicia	Spain	15706

Sponsors and Collaborators

• Airway Therapeutics, Inc.

Investigators

• Study Chair: Marc O. Salzberg, MD, Airway Therapeutics, Inc.
• Study Director: Michael DePietro, MD, Airway Therapeutics, Inc.

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• Airway Therapeutics' corporate website

Publications

• 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. Epub 2006 Mar 23.
• 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.
• 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.
• 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. Review.
• Thébaud 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. Review.

• Responsible Party: Airway Therapeutics, Inc.
• ClinicalTrials.gov Identifier: NCT04662151
• Other Study ID Numbers: AT-100/001
• First Posted: Dec 10, 2020
• Last Update Posted: May 2, 2022
• Last Verified: Apr 1, 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

Study Results

No Results Posted as of May 2, 2022