Trial To Assess The Safety And Tolerability Of Lucinactant For Inhalation In Preterm Neonates 26 to 28 Weeks PMA

NCT ID: NCT02528318

Last Updated: 2019-07-23

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 48 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-08-31
• Study Completion Date: 2017-08-11

Brief Summary

This study is to evaluate the safety and tolerability of lucinactant for inhalation, administered as an aerosol in up to four escalating doses to preterm neonates 26 to 28 weeks gestational age who are receiving nCPAP for RDS compared to neonates receiving nCPAP alone.

Detailed Description

This study was a multicenter, randomized, controlled, open-label, dose-escalation study, conducted to evaluate the safety and tolerability of lucinactant for inhalation in conjunction with nasal continuous positive airway pressure (nCPAP) in comparison with nCPAP alone. The study was to evaluate the safety and tolerability of lucinactant for inhalation, administered as an aerosol in 4 escalating doses.

For this study, lucinactant for inhalation refers to the active investigational agent, lyophilized lucinactant, in combination with the prototype investigational delivery device. Reconstituted lyophilized lucinactant was aerosolized by the investigational device and introduced into the nCPAP circuit. Those randomized to the control arm continued to receive nCPAP alone. Dose assignments were unblinded, as the primary objective of this study was safety and tolerability.

Preterm neonates with respiratory distress syndrome (RDS) between 26 and 28 completed weeks PMA who were within the first 20 hours after birth and who had successful implementation of controlled nCPAP within 90 minutes of birth were considered to be potential subjects. Before study enrollment, legal guardians were provided a written informed consent form (ICF) for each potential subject.

Conditions

Respiratory Distress Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

50 mg/kg

Lucinactant for inhalation 50 mg total phospholipids (TPL)/kg with nCPAP

1 repeat dose allowed if repeat dosing criteria are met.

Group Type: EXPERIMENTAL

Lucinactant for inhalation

Intervention Type: COMBINATION_PRODUCT

Lucinactant for inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product)

75 mg/kg

Lucinactant for inhalation 75 mg TPL/kg with nCPAP

1 repeat dose allowed if repeat dosing criteria are met.

Group Type: EXPERIMENTAL

Lucinactant for inhalation

Intervention Type: COMBINATION_PRODUCT

Lucinactant for inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product)

100 mg/kg

Lucinactant for inhalation 100 mg TPL/kg with nCPAP

1 repeat dose allowed if repeat dosing criteria are met.

Group Type: EXPERIMENTAL

Lucinactant for inhalation

Intervention Type: COMBINATION_PRODUCT

Lucinactant for inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product)

150 mg/kg

Lucinactant for inhalation 150 mg TPL/kg with nCPAP

1 repeat dose will be allowed if repeat dosing criteria are met.

Group Type: EXPERIMENTAL

Lucinactant for inhalation

Intervention Type: COMBINATION_PRODUCT

Lucinactant for inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product)

nCPAP alone

nCPAP therapy alone

Group Type: ACTIVE_COMPARATOR

nCPAP alone

Intervention Type: DEVICE

nCPAP therapy

Interventions

Lucinactant for inhalation

Lucinactant for inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product)

Intervention Type: COMBINATION_PRODUCT

nCPAP alone

nCPAP therapy

Intervention Type: DEVICE

Other Intervention Names

AEROSURF®; nasal continuous positive airway pressure

Eligibility Criteria

Inclusion Criteria

1. Informed consent from a legally authorized representative.

2. Gestational age 26 to 28 completed weeks post menstrual age (PMA).

3. Successful implementation of controlled nCPAP within 90 minutes after birth.

4. Spontaneous breathing.

5. Chest radiograph consistent with RDS.

6. Within the first 20 hours after birth, have an nCPAP of 5 to 6 cm H2O to maintain oxygen saturation measured by pulse oximetry (SpO2) of 88% to 95% with a fraction of inspired oxygen (FiO2) of 0.25 to 0.50 that is clinically indicated for at least 30 minutes. Transient (<10 minutes) FiO2 excursions below 0.25 or above 0.50 did not reset the 30 minute requirement.

Exclusion Criteria

1. Heart rate that cannot be stabilized above 100 beats/minute within 5 minutes of birth.

2. Recurrent episodes of apnea occurring after the initial newborn resuscitation period (ie, 10 minutes after birth) requiring intermittent positive pressure breaths using inflating pressures above the set CPAP pressure administered manually or mechanically through any patient interface.

3. A 5 minute Apgar score < 5.

4. Major congenital malformation(s) and cranial/facial abnormalities that preclude nCPAP, diagnosed antenatally or immediately after birth.

5. Other diseases or conditions potentially interfering with cardiopulmonary function (eg, hydrops fetalis or congenital infection such as TORCH).

6. Known or suspected chromosomal abnormality or syndrome.

7. Premature rupture of membranes (PROM) > 2 weeks.

8. Evidence of hemodynamic instability requiring vasopressors or steroids for hemodynamic support and/or presumed clinical sepsis.

9. Need for endotracheal intubation and mechanical ventilation.

10. Has been administered: another investigational agent or investigational medical device, any other surfactant agent, steroid treatment after birth.

• Minimum Eligible Age: 26 Weeks
• Maximum Eligible Age: 28 Weeks
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Windtree Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Steve Simonson, MD

Role: STUDY_DIRECTOR

Windtree Therapeutics

Locations

Loma Linda University Medical Center

Loma Linda, California, United States

Sharp Mary Birch Hospital for Women and Newborns

San Diego, California, United States

Christiana Care Health System

Newark, Delaware, United States

University of Miami

Miami, Florida, United States

University of Louisville

Louisville, Kentucky, United States

University of Nebraska Medical Center

Omaha, Nebraska, United States

Columbia University College of Physicians and Surgeons, New York Presbyterian Hospital - Morgan Stanley Children's Hospital

New York, New York, United States

New Hanover Regional Medical Center

Wilmington, North Carolina, United States

Providence St. Vincent Medical Center

Portland, Oregon, United States

Women and Infants Hospital

Providence, Rhode Island, United States

Foothills Medical Centre

Calgary, Alberta, Canada

Royal Alexandria Hospital

Edmonton, Alberta, Canada

Sunnybrook Health Sciences Centre

Toronto, Ontario, Canada

Montreal Children's Hospital

Montreal, Quebec, Canada

Hospital Dr Sotero Del Rio

Santiago, , Chile

Hospital San Juan de Dios

Santiago, , Chile

Ginekologiczno-Polozniczy Szpital Klinicznym UM im. Karola Marcinkowskiego w Poznan i u Katedra Neonatologii

Poznan, Greater Poland Voivodeship, Poland

S.U. nr2im. Dr. Jana Biziela Oddzial Kliniczny N. W. Z. Intensywna Terapia Noworodka wraz z Wgjazdowy m Zespolem N

Bydgoszcz, Kujawsko-pomorksie, Poland

Szpital Kliniczny im. Ks, Anny Mazowieckiej Klinika Neonatologii

Warsaw, Masovian Voivodeship, Poland

Instytut Centrum Zdrowja Matki Polki Klinika Neonatologii

Lodz, Łódź Voivodeship, Poland

Countries

United States; Canada; Chile; Poland

Provided Documents

Document Type: Statistical Analysis Plan

View Document

Document Type: Study Protocol

View Document

Other Identifiers

03-CL-1401

Identifier Type: -

Identifier Source: org_study_id