Clinical trial to assess the safety of the drug named AT-100 in preterm neonates at high risk for development of bronchopulmonary dysplasia
- Conditions
- Bronchopulmonary Dysplasia (BPD)Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]MedDRA version: 21.1Level: PTClassification code 10006475Term: Bronchopulmonary dysplasiaSystem Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
- Registration Number
- EUCTR2021-005752-10-IT
- Lead Sponsor
- Airway Therapeutics, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- All
- Target Recruitment
- 36
1. Inpatient preterm neonates born between GA 25 to 28 6/7 weeks, inclusive, for the dose escalation cohorts; GA 23 to 28 6/7 weeks, inclusive, for the latter cohort receiving the highest-tolerated and safest tested dose.
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 point after 15 minutes following any of the the subject's Curosurf® dose(s).
4. Parent or legal guardian (as per local laws governing birth & neonate decision rights) is able to understand and sign a written informed consent form (ICF), which must be obtained prior to treatment and any study-related procedures.
5. Parent or legal guardian is able to understand and sign a consent or authorization form, which shall permit the use, disclosure, and transfer of the subject's personal health information (e.g., in the USA, Health Information Portability and Accountability Act [HIPAA] Authorization form).
6. Parent or legal guardian is willing and able to have neonate complete all trial procedures and successfully complete the trial.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Weight at time of birth < 400 g or > 1,800 g.
2. Major apparent congenital abnormalities impacting cardio and pulmonary function such as, but not limited to:
a. congenital diaphragmatic hernia,
b. congenital pulmonary airway malformation,
c. omphalocele, or
d. known or suspected cyanotic congenital heart disease (i.e., tetralogy
of fallot, transposition of the great arteries, etc.).
3. Active DNR (Do Not Resuscitate) order in place.
4. Known pulmonary air leaks (e.g., pneumothorax or 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 DSMC recommendation to proceed to the next dose-escalation cohort.
7. Use of minimally invasive surfactant administration techniques (e.g., LISA, MIST) or INSURE or if, in the opinion of the care team, the infant is very likely to 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:
a. Has known active Hepatitis B, C, or E diagnosis.
b. Has 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.
c. Has known active Sexually Transmitted Infection (STI) (i.e., Herpes Simplex Virus [HSV] with known active lesions; HIV; current Syphilis, Gonococcal, or Chlamydial infection that has not been treated)
d. Has known Cytomegalovirus (CMV) active infection (not including CMV-carrier).
e. Has known history or evidence of alcohol or drug abuse, with 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. For the neonate, concurrent enrollment in an investigational drug, device, or treatment modulation trial that utilizes treatments outside of standard-of-care or participation in such a trial within the last 30 days (or 5 half-lives of an investigational product) prior to birth or up to Week 36 PMA.
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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To determine the recommended highest-tolerated and safest-tested dose that maximizes benefit-to-risk of AT-100 in preterm neonates born initially between GA 25 weeks and 28 6/7 weeks, then between GA 23 weeks and 28 6/7 weeks, who are at high risk for the development of BPD.;Secondary Objective: To evaluate preliminary efficacy parameters.;Primary end point(s): Safety and tolerability of AT-100;Timepoint(s) of evaluation of this end point: From time of initial AT-100 dosing until end of the study participation up to 36 Weeks Postmenstrual Age
- Secondary Outcome Measures
Name Time Method