A Dose-Ranging Study to Determine the Efficacy, Safety and Tolerability of AeroFact

A Partially Blinded, Randomized, Controlled, Parallel-Group, Dose-Ranging Study to Determine the Efficacy, Safety and Tolerability of AeroFact in Preterm Infants at Risk of Worsening Respiratory Distress Syndrome

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03969992

Enrollment

261

Registered

2019-05-31

Start date

2020-03-04

Completion date

2024-08-05

Last updated

2025-08-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Respiratory Distress Syndrome in Premature Infant

Keywords

Respiratory Distress Syndrome, BPD, surfactant, Preterm Infant

Brief summary

The purpose of this two-part Phase 2 study is to assess the safety, tolerability and efficacy of aerosolized SF-RI 1 (AeroFact) when delivered via nCPAP at two different doses.

Detailed description

Part I Primary Objective To determine an optimal dose of AeroFact administered by nasal continuous positive airway pressure (nCPAP) versus stand of care in reducing the rate of intubation/cannulation and bolus surfactant instillation in the first 7 days after birth. Part II Primary Objective To evaluate pulmonary outcomes and respiratory utilization at 3, 6, 9, and 12 months post-menstrual age (PMA)

Interventions

DRUGAeroFact

Aerosolized SF-RI 1

OTHERnCPAP

nCPAP (nasal continuous positive airway pressure) alone

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Masking description

Masking within active arms

Intervention model description

Partially Blinded, Randomized, Parallel-Group Dose Ranging

Eligibility

Sex/Gender

ALL

Age

26 Weeks to 31 Weeks

Healthy volunteers

Inclusion criteria

1. Parental consent obtained prior to study procedures being performed (pre-natal consent is allowed) 2. 26 0/7 to 30 6/7 weeks of gestational age 3. Weight \<2.0 Kg 4. Respiratory Severity Score (RSS) 1.4-2.0

Exclusion criteria

1. Apgar score less than or equal to 5 at five minutes after birth 2. Need for chest compressions or administration of epinephrine or bicarbonate in the delivery room 3. Premature rupture of membranes (PROM) \> 14 days 4. Need for intubation and/or mechanical ventilation prior to enrollment 5. Active pneumothorax requiring chest tube 6. Significant congenital anomaly, chromosomal abnormality 7. Concomitant treatments with inhaled nitric oxide

Design outcomes

Primary

Measure	Time frame	Description
Intubation/Cannulation and Instilled Surfactant	First 7 days of life	Number of patients who require intubation/cannulation with bolus surfactant instillation

Secondary

Measure	Time frame	Description
Time to First Intubation/Cannulation and Bolus Surfactant Instillation	First 7 days of life	Time from randomization to intubation/cannulation and bolus surfactant instillation in the first 7 days of life
Proportion of Infants Who Received Multiple Doses of Bolus Surfactant	First 7 days of life	—
Number of Days on Invasive Mechanical Ventilation	Assessed daily from birth to 40 weeks post-menstrual age or discharge	Total number of days each participant required support with invasive mechanical ventilation
Proportion of Patients Survived Without Bronchopulmonary Dysplasia (BPD) at 36 Weeks Post-menstrual Age (PMA)	Birth to 36 weeks post-menstrual age	Total number of patients per treatment group who were alive and did not experience BPD at 36 weeks PMA
Proportion of Patients Survived Without Bronchopulmonary Dysplasia (BPD) at 40 Weeks Post-menstrual Age (PMA)	Birth to 40 weeks post-menstrual age	Total number of patients per treatment group who were alive and did not experience BPD at 40 weeks PMA

Countries

Canada, United States

Participant flow

Participants by arm

Arm	Count
Control: nCPAP Alone nCPAP (nasal continuous positive airway pressure) alone	92
Drug: Low Dose AeroFact AeroFact-low dose SF-RI 1	83
Drug: High Dose AeroFact AeroFact-high dose SF-RI 1	86
Total	261

Withdrawals & dropouts

Period	Reason	FG000	FG001	FG002
Part 1: Birth to Discharge/40 Weeks PMA	Death	2	3	2
Part 1: Birth to Discharge/40 Weeks PMA	Withdrawal by Subject	1	0	3
Part 2: Discharge/40 Wks PMA to 12 Mos	Lost to Follow-up	12	8	9
Part 2: Discharge/40 Wks PMA to 12 Mos	Physician Decision	0	0	1
Part 2: Discharge/40 Wks PMA to 12 Mos	Withdrawal by Subject	3	1	0

Baseline characteristics

Characteristic	Total	Drug: High Dose AeroFact	Drug: Low Dose AeroFact	Control: nCPAP Alone
Age at Randomization	9.1 hours STANDARD_DEVIATION 6.9	8.9 hours STANDARD_DEVIATION 6.9	8.2 hours STANDARD_DEVIATION 6.5	10.0 hours STANDARD_DEVIATION 7.2
Age, Continuous	29.2 weeks gestational age STANDARD_DEVIATION 1.4	29.1 weeks gestational age STANDARD_DEVIATION 1.5	29.1 weeks gestational age STANDARD_DEVIATION 1.4	29.2 weeks gestational age STANDARD_DEVIATION 1.3
Birth weight	1228.7 grams STANDARD_DEVIATION 300.9	1248.7 grams STANDARD_DEVIATION 303.9	1245.1 grams STANDARD_DEVIATION 301.5	1195.1 grams STANDARD_DEVIATION 297.9
Ethnicity (NIH/OMB) Hispanic or Latino	47 Participants	12 Participants	22 Participants	13 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	207 Participants	72 Participants	58 Participants	77 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	7 Participants	2 Participants	3 Participants	2 Participants
FiO2 at Randomization	0.27 Percentage of inspired oxygen STANDARD_DEVIATION 0.05	0.27 Percentage of inspired oxygen STANDARD_DEVIATION 0.05	0.27 Percentage of inspired oxygen STANDARD_DEVIATION 0.04	0.27 Percentage of inspired oxygen STANDARD_DEVIATION 0.05
Multiple gestation	85 Participants	26 Participants	29 Participants	30 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants	1 Participants	0 Participants	0 Participants
Race (NIH/OMB) Asian	10 Participants	6 Participants	1 Participants	3 Participants
Race (NIH/OMB) Black or African American	43 Participants	15 Participants	12 Participants	16 Participants
Race (NIH/OMB) More than one race	2 Participants	2 Participants	0 Participants	0 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Unknown or Not Reported	50 Participants	14 Participants	20 Participants	16 Participants
Race (NIH/OMB) White	155 Participants	48 Participants	50 Participants	57 Participants
Respiratory Severity Score at Randomization	1.7 units on a scale STANDARD_DEVIATION 0.2	1.7 units on a scale STANDARD_DEVIATION 0.3	1.7 units on a scale STANDARD_DEVIATION 0.2	1.7 units on a scale STANDARD_DEVIATION 0.2
Sex: Female, Male Female	122 Participants	37 Participants	38 Participants	47 Participants
Sex: Female, Male Male	139 Participants	49 Participants	45 Participants	45 Participants
Vaginal delivery	58 Participants	19 Participants	25 Participants	14 Participants

Adverse events

Event type	EG000 affected / at risk	EG001 affected / at risk	EG002 affected / at risk
deaths Total, all-cause mortality	2 / 92	3 / 83	2 / 86
other Total, other adverse events	43 / 92	45 / 83	42 / 86
serious Total, serious adverse events	7 / 92	9 / 83	5 / 86

Outcome results

Primary

Intubation/Cannulation and Instilled Surfactant

Number of patients who require intubation/cannulation with bolus surfactant instillation

Time frame: First 7 days of life

Population: Per Protocol population

Arm	Measure	Value (COUNT_OF_PARTICIPANTS)
Control: nCPAP Alone	Intubation/Cannulation and Instilled Surfactant	45 Participants
Drug: Low Dose AeroFact	Intubation/Cannulation and Instilled Surfactant	21 Participants
Drug: High Dose AeroFact	Intubation/Cannulation and Instilled Surfactant	26 Participants

p-value: 0.1924Generalized estimating equation method

Secondary