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

NCT02528318 | Terminated Phase 2 Results DMC

• 1 other identifier: 03-CL-1401
• Study Type: interventional
• Target: 48
• Locations: 4 countries
• Sites: 20

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.

Conditions

Respiratory Distress Syndrome

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Peri-Dosing Adverse Events - Initial Dose

  Number of Participants with adverse events that were experienced during the initial study treatment

  Randomization to 24 Hours Post Randomization

• Number of Participants With Air Leak

  Number of participants with air leak (includes pneumothorax, pulmonary interstitial emphysema (PIE), pneumomediastinum, pneumopericardium, subcutaneous emphysema)

  7 days

Secondary Outcomes (6)

• Number of Participants With Worsening of Respiratory Status Criteria

  Randomization to 72 Hours Post Randomization

• Bronchopulmonary Dysplasia

  Randomization to 36 weeks PMA

• Number of Participants With Nasal Continuous Positive Airway Pressure (nCPAP) Failure

  Randomization to 72 Hours Post Randomization

• Death

  Randomization to 36 weeks PMA

• FiO2

  Randomization to 72 hours post randomization

Other Outcomes (1)

• nCPAP Failure Without Treatment Interruptions

  Randomization to 72 Hours Post Randomization

Study Arms (5)

50 mg/kg

EXPERIMENTAL

Lucinactant for inhalation 50 mg total phospholipids (TPL)/kg with nCPAP 1 repeat dose allowed if repeat dosing criteria are met.

Combination Product: Lucinactant for inhalation

75 mg/kg

EXPERIMENTAL

Lucinactant for inhalation 75 mg TPL/kg with nCPAP 1 repeat dose allowed if repeat dosing criteria are met.

Combination Product: Lucinactant for inhalation

100 mg/kg

EXPERIMENTAL

Lucinactant for inhalation 100 mg TPL/kg with nCPAP 1 repeat dose allowed if repeat dosing criteria are met.

Combination Product: Lucinactant for inhalation

150 mg/kg

EXPERIMENTAL

Lucinactant for inhalation 150 mg TPL/kg with nCPAP 1 repeat dose will be allowed if repeat dosing criteria are met.

Combination Product: Lucinactant for inhalation

nCPAP alone

ACTIVE COMPARATOR

nCPAP therapy alone

Device: nCPAP alone

Interventions

Lucinactant for inhalation COMBINATION_PRODUCT

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

Also known as: AEROSURF®

100 mg/kg; 150 mg/kg; 50 mg/kg; 75 mg/kg

nCPAP alone DEVICE

nCPAP therapy

Also known as: nasal continuous positive airway pressure

nCPAP alone

Eligibility Criteria

• Age: 26 Weeks - 28 Weeks
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Informed consent from a legally authorized representative.
• Gestational age 26 to 28 completed weeks post menstrual age (PMA).
• Successful implementation of controlled nCPAP within 90 minutes after birth.
• Spontaneous breathing.
• Chest radiograph consistent with RDS.
• 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.

You may not qualify if:

• Heart rate that cannot be stabilized above 100 beats/minute within 5 minutes of birth.
• 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.
• A 5 minute Apgar score \< 5.
• Major congenital malformation(s) and cranial/facial abnormalities that preclude nCPAP, diagnosed antenatally or immediately after birth.
• Other diseases or conditions potentially interfering with cardiopulmonary function (eg, hydrops fetalis or congenital infection such as TORCH).
• Known or suspected chromosomal abnormality or syndrome.
• Premature rupture of membranes (PROM) \> 2 weeks.
• Evidence of hemodynamic instability requiring vasopressors or steroids for hemodynamic support and/or presumed clinical sepsis.
• Need for endotracheal intubation and mechanical ventilation.
• Has been administered: another investigational agent or investigational medical device, any other surfactant agent, steroid treatment after birth.

Sponsors & Collaborators

• Windtree Therapeutics: lead

Study Sites (20)

Loma Linda University Medical Center

Loma Linda, California, 92354, United States

Location

Sharp Mary Birch Hospital for Women and Newborns

San Diego, California, 92123, United States

Location

Christiana Care Health System

Newark, Delaware, 19713, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

University of Louisville

Louisville, Kentucky, 40202, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68105, United States

Location

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

New York, New York, 10032, United States

Location

New Hanover Regional Medical Center

Wilmington, North Carolina, 28401, United States

Location

Providence St. Vincent Medical Center

Portland, Oregon, 97225, United States

Location

Women and Infants Hospital

Providence, Rhode Island, 02905, United States

Location

Foothills Medical Centre

Calgary, Alberta, T2N 2T9, Canada

Location

Royal Alexandria Hospital

Edmonton, Alberta, T5H 3V9, Canada

Location

Sunnybrook Health Sciences Centre

Toronto, Ontario, M4N 3M5, Canada

Location

Montreal Children's Hospital

Montreal, Quebec, H4A 3J1, Canada

Location

Hospital Dr Sotero Del Rio

Santiago, 8207257, Chile

Location

Hospital San Juan de Dios

Santiago, 8350488, Chile

Location

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

Poznan, Greater Poland Voivodeship, 60-535, Poland

Location

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

Bydgoszcz, Kujawsko-pomorksie, 85-168, Poland

Location

Szpital Kliniczny im. Ks, Anny Mazowieckiej Klinika Neonatologii

Warsaw, Masovian Voivodeship, 00-315, Poland

Location

Instytut Centrum Zdrowja Matki Polki Klinika Neonatologii

Lodz, Łódź Voivodeship, 93-338, Poland

Location

MeSH Terms

Conditions

Respiratory Distress Syndrome

Interventions

lucinactant; Inhalation; Continuous Positive Airway Pressure

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Respiration Disorders

Intervention Hierarchy (Ancestors)

Respiratory Mechanics; Respiration; Respiratory Physiological Phenomena; Circulatory and Respiratory Physiological Phenomena; Positive-Pressure Respiration; Respiration, Artificial; Airway Management; Therapeutics; Respiratory Therapy

Limitations and Caveats

The study was terminated prior to subject enrollment for the 150 mg TPL/kg dose by the sponsor for administrative reasons. The overall results may have been impacted by treatment interruptions caused by clogging of study drug.

Results Point of Contact

• Title: Robert Segal, MD, FACP
• Organization: Windtree Therapeutics, Inc.

rsegal@windtreetx.com

215-488-9300

Study Officials

• Steve Simonson, MD

  Windtree Therapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 17, 2015
• First Posted: August 19, 2015
• Study Start: August 1, 2015
• Primary Completion: May 31, 2017
• Study Completion: August 11, 2017
• Last Updated: July 23, 2019
• Results First Posted: July 23, 2019

Record last verified: 2019-07

Data Sharing

• IPD Sharing: Will not share

Locations

PL (4); US (10); CA (4); CL (2)