Clinical trial • Phase II/III • Respiratory|Rare Disease

RECOMBINANT HUMAN SURFACTANT PROTEIN-D for Bronchopulmonary dysplasia (BPD)|Prematurity

Phase II/III trial of RECOMBINANT HUMAN SURFACTANT PROTEIN-D for Bronchopulmonary dysplasia (BPD)|Prematurity.

Overview

Trial Therapeutic Area
Respiratory|Rare Disease
Trial Disease
Bronchopulmonary dysplasia (BPD)|Prematurity
Trial Stage
Phase II/III
Drug Modality
Peptide/protein/enzyme
Paediatric Trial
Yes
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
13-06-2024
First CTIS Authorization Date
30-09-2024

Trial design

Randomised, zelpultide alfa 4 mg/kg and 6 mg/kg added to standard of care (soc) versus placebo (air-sham; endotracheopulmonary instillation of room air) added to soc. doses evaluated in part 1: 4 mg/kg and 6 mg/kg (dose selection). max daily dose reported 6 mg/kg; max total dose reported 42 mg/kg; max treatment period 7 (time unit per protocol).-controlled, adaptive Phase II/III trial across 46 sites in Belgium, France, Poland and others.

Randomised
Yes
Comparator
Zelpultide alfa 4 mg/kg and 6 mg/kg added to standard of care (SOC) versus placebo (air-sham; endotracheopulmonary instillation of room air) added to SOC. Doses evaluated in Part 1: 4 mg/kg and 6 mg/kg (dose selection). Max daily dose reported 6 mg/kg; max total dose reported 42 mg/kg; max treatment period 7 (time unit per protocol).
Adaptive
True, Part 1 is an adaptive dose-selection phase comparing two dose levels (4 mg/kg and 6 mg/kg) versus placebo with a pre-specified futility assessment to support selection of dose for Part 2 (Phase 3).
Single Multiple Or Escalation Dose Combined
Yes
Target Sample Size
241

Eligibility

Recruits 241 paediatric patients.

Vulnerable Population
The trial enrols high-risk preterm neonates (vulnerable population). Informed consent must be provided and signed by the subject’s parent(s) or legal guardian(s). Study-specific subject information and informed consent forms (SIS/ICF) and family brochures are provided for parents/guardians in local languages per country.

Inclusion criteria

  • {"criterion_text":"- Born between gestational age (GA) 22 0/7 to 27 6/7 weeks, inclusive."}
  • {"criterion_text":"- Received at least 1 dose of SOC-indicated animal-derived pulmonary surfactant treatment after birt"}
  • {"criterion_text":"- Intubated and on invasive mechanical ventilation per SOC."}
  • {"criterion_text":"- Able to receive the first dose of zelpultide alfa or air-sham at least 15 min after the surfactant administration but within 96 h of birth and within 48 h from the start of invasive mechanical ventilation. • Subjects extubated and re-intubated after their pulmonary surfactant dose(s) are eligible as long as the inclusion criteria are met."}
  • {"criterion_text":"- Informed consent and personal information authorization form signed by the subject’s parent(s) or legal guardian(s)."}

Exclusion criteria

  • {"criterion_text":"- Birth weight < 400 g or > 1,500 g."}
  • {"criterion_text":"- Major apparent congenital abnormalities impacting cardio and pulmonary function identified before randomization, such as, but not limited to: • Clinically relevant Potter-like syndrome and any pulmonary congenital anomalies, • Clinically relevant congenital hernia, • Omphalocele or diaphragmaticgastroschisis, esophageal atresia,• Known or suspected cyanotic congenital heart disease (ie, tetralogy of fallot, transposition of the great arteries, etc)."}
  • {"criterion_text":"- Active do no resuscitate (DNR) order in place."}
  • {"criterion_text":"- History of allergy or sensitivity to any surfactant or any component of zelpultide alfa."}
  • {"criterion_text":"- Concurrent enrollment in any clinical study that utilizes treatments (investigational medical products or devices) outside of SOC or participation in studies within the last 30 days (or 5 half-lives of an IMP) prior to birth (for the mother) or up to week 36 PMA."}
  • {"criterion_text":"- Any condition or situation that, in the Investigator’s judgement, puts the neonate at significant risk, could confound the study results, or may interfere significantly with the neonate’s participation in the study."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Part 1 (Phase 2b / Dose Selection): Incidence of grade 2 or grade 3 BPD or death at week 36 PMA, analyzed descriptively and used for the pre-specified futility assessment.","definition_or_measurement_approach":"Incidence of grade 2 or grade 3 bronchopulmonary dysplasia or death assessed at week 36 post-menstrual age (PMA); analyzed descriptively and used for a pre-specified futility assessment to support dose selection in Part 1."}
  • {"endpoint_text":"- Part 2 (Phase 3 / Confirmatory Phase): Incidence of grade 2 or grade 3 BPD or death at week 36 PMA.","definition_or_measurement_approach":"Incidence of grade 2 or grade 3 bronchopulmonary dysplasia or death assessed at week 36 post-menstrual age (PMA) (confirmatory analysis in Phase 3)."}

Secondary endpoints

  • {"endpoint_text":"- Part 1 (Phase 2b / Dose Selection): Key Secondary Endpoints: Ventilator-free days from birth to week 36 PMA; Incidence of grade 2 or grade 3 BPD or death assessed at 3 consecutive days at week 36 PMA","definition_or_measurement_approach":"Ventilator-free days from birth to week 36 PMA; incidence of grade 2/3 BPD or death assessed over 3 consecutive days at week 36 PMA."}
  • {"endpoint_text":"- Additional Efficacy Secondary Endpoints: Incidence of grade 2 or grade 3 BPD at week 36 PMA; Incidence of death at week 36 PMA; The proportion of subjects with no BPD, grade 1, grade 2, or grade 3 BPD at week 36 PMA; The proportion of subjects with no BPD, grade 1, grade 2, or grade 3 BPD assessed at 3 consecutive days at week 36 PMA.","definition_or_measurement_approach":"Incidence and categorical distribution of BPD severity and death at week 36 PMA; assessment includes snapshot at week 36 and evaluation over 3 consecutive days where specified."}
  • {"endpoint_text":"- Part 2 (Phase 3 / Confirmatory Phase): Key Efficacy Secondary Endpoints: Ventilator-free days from birth to week 36 PMA; Incidence of grade 2 or grade 3 BPD at week 36 PMA; Incidence of death at week 36 PMA; Incidence of grade 2 or grade 3 BPD or death assessed at 3 consecutive days at week 36 PMA.","definition_or_measurement_approach":"Ventilator-free days from birth to week 36 PMA; incidence of grade 2/3 BPD and death at week 36 PMA; incidence assessed over 3 consecutive days at week 36 PMA."}

Recruitment

Planned Sample Size
241
Recruitment Window Months
29
Consent Approach
Informed consent must be signed by the subject’s parent(s) or legal guardian(s). Subject information sheets and informed consent forms (SIS/ICF) and family brochures are provided; country-specific ICF/SIS documents are available (examples in French, Dutch (NL-BE), Polish, Italian, English and other local languages as per country submissions). Assent is not applicable for neonates; consent is obtained from parents/legal guardians.

Geography

Total Number Of Sites
46
Total Number Of Participants
241

Belgium

Earliest CTIS Part Ii Submission Date
11-12-2025
Latest Decision Or Authorization Date
04-02-2026
Processing Time Days
55
Number Of Sites
4
Number Of Participants
20

Sites

Site Name
Centre Hospitalier Regional De La Citadelle
Department Name
Neonatology
Principal Investigator Name
Vincent Rigo
Principal Investigator Email
vincent.rigo@chuliege.be
Contact Person Name
Vincent Rigo
Contact Person Email
vincent.rigo@chuliege.be
Site Name
CHC MontLegia
Department Name
Neonatal Intensive Care Unit
Principal Investigator Name
Pierre Maton
Principal Investigator Email
pierre.maton@chc.be
Contact Person Name
Pierre Maton
Contact Person Email
pierre.maton@chc.be
Site Name
UZ Leuven
Department Name
Neonatalogy, Department of Reproduction and Growth
Principal Investigator Name
Anne Debeer
Principal Investigator Email
anne.debeer@uzleuven.be
Contact Person Name
Anne Debeer
Contact Person Email
anne.debeer@uzleuven.be
Site Name
Cliniques Universitaires Saint-Luc
Department Name
Pediatrics
Principal Investigator Name
Olivier Danhaive
Principal Investigator Email
olivier.danhaive@uclouvain.be
Contact Person Name
Olivier Danhaive
Contact Person Email
olivier.danhaive@uclouvain.be

France

Earliest CTIS Part Ii Submission Date
14-01-2026
Latest Decision Or Authorization Date
24-02-2026
Processing Time Days
41
Number Of Sites
9
Number Of Participants
32

Sites

Site Name
Hopital Antoine-Beclere
Department Name
Division of Pediatrics, Transportation and Neonatal Critical Care
Principal Investigator Name
Daniele De Luca
Principal Investigator Email
daniele.deluca@aphp.fr
Contact Person Name
Daniele De Luca
Contact Person Email
daniele.deluca@aphp.fr
Site Name
Robert Debre University Hospital
Department Name
Service reanimation et pédiatrie néonatales
Principal Investigator Name
Valérie Biran
Principal Investigator Email
valerie.biran@aphp.fr
Contact Person Name
Valérie Biran
Contact Person Email
valerie.biran@aphp.fr
Site Name
CHRU De Nancy
Department Name
Neonatology
Principal Investigator Name
Elena Ismael
Principal Investigator Email
e.ismael@chru-nancy.fr
Contact Person Name
Elena Ismael
Contact Person Email
e.ismael@chru-nancy.fr
Site Name
Centre Hospitalier Universitaire De Lille
Department Name
Neonatology
Principal Investigator Name
Mohamed Riadh Boukhris
Principal Investigator Email
riadh.boukhris@chu-lille.fr
Contact Person Name
Mohamed Riadh Boukhris
Contact Person Email
riadh.boukhris@chu-lille.fr
Site Name
Bicetre Hospital
Department Name
Pediatric and Neonatal Intensive Care
Principal Investigator Name
Silvia Foligno
Principal Investigator Email
silvia.foligno@aphp.fr
Contact Person Name
Silvia Foligno
Contact Person Email
silvia.foligno@aphp.fr
Site Name
Centre Hospitalier Intercommunal De Poissy Saint Germain
Department Name
Neonatal Intensive Care Unit
Principal Investigator Name
Emmanuelle Motte-Signoret
Contact Person Name
Emmanuelle Motte-Signoret
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Neonatal Intensive Care Unit
Principal Investigator Name
Olivier Baud
Principal Investigator Email
olivier.baud@aphp.fr
Contact Person Name
Olivier Baud
Contact Person Email
olivier.baud@aphp.fr
Site Name
Centre Hospitalier Universitaire De Nice
Department Name
Médecine et réanimation néonatale
Principal Investigator Name
Sergio Eleni Dit Trolli
Principal Investigator Email
elenidittrolli.s@chu-nice.fr
Contact Person Name
Sergio Eleni Dit Trolli
Contact Person Email
elenidittrolli.s@chu-nice.fr
Site Name
CHRU De Nancy (duplicate listed?)

Poland

Earliest CTIS Part Ii Submission Date
19-02-2026
Latest Decision Or Authorization Date
23-03-2026
Processing Time Days
32
Number Of Sites
7
Number Of Participants
36

Sites

Site Name
Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu
Department Name
Neonatology
Principal Investigator Name
Barbara Królak-Olejnik
Principal Investigator Email
kne@usk.wroc.pl
Contact Person Name
Barbara Królak-Olejnik
Contact Person Email
kne@usk.wroc.pl
Site Name
Uniwersytecki Szpital Kliniczny Nr 2 Pum W Szczecinie
Department Name
Neonatal Pathology Clinics
Principal Investigator Name
Beata Łoniewska
Principal Investigator Email
beata.loniewska@pum.edu.pl
Contact Person Name
Beata Łoniewska
Contact Person Email
beata.loniewska@pum.edu.pl
Site Name
Instytut Centrum Zdrowia Matki Polki
Department Name
Neonatology, Pathology, and Intensive Care Clinics for Newborns
Principal Investigator Name
Ewa Gulczyńska
Principal Investigator Email
e.gulczynska@iczmp.eud.pl
Contact Person Name
Ewa Gulczyńska
Contact Person Email
e.gulczynska@iczmp.eud.pl
Site Name
Ginekologiczno Polozniczy Szpital Kliniczny Uniwersytetu Medycznego Im Karola Marcinkowskiego W Poznaniu
Department Name
Neonatology II
Principal Investigator Name
Tomasz Szczapa
Principal Investigator Email
tszczapa@gmail.com
Contact Person Name
Tomasz Szczapa
Contact Person Email
tszczapa@gmail.com
Site Name
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie
Department Name
Neonatology
Principal Investigator Name
Agnieszka Ochoda-Mazur
Principal Investigator Email
aochoda@su.krakow.pl
Contact Person Name
Agnieszka Ochoda-Mazur
Contact Person Email
aochoda@su.krakow.pl
Site Name
Ginekologiczno Polozniczy Szpital Kliniczny Uniwersytetu Medycznego Im Karola Marcinkowskiego W Poznaniu (second site)
Department Name
Neonatology I
Principal Investigator Name
Jan Mazela
Principal Investigator Email
janco@pol-med.com.pl
Contact Person Name
Jan Mazela
Contact Person Email
janco@pol-med.com.pl
Site Name
Uniwersyteckie Centrum Kliniczne
Department Name
Neonatology
Principal Investigator Name
Paulina Kobiela
Principal Investigator Email
paulina.kobiela@gumed.edu.pl
Contact Person Name
Paulina Kobiela
Contact Person Email
paulina.kobiela@gumed.edu.pl

Spain

Earliest CTIS Part Ii Submission Date
03-09-2024
Latest Decision Or Authorization Date
03-10-2024
Processing Time Days
30
Number Of Sites
13
Number Of Participants
90

Sites

Site Name
Hospital General Universitario Gregorio Maranon
Department Name
Neonatology
Principal Investigator Name
Manuel Sánchez Luna
Principal Investigator Email
msluna@salud.madrid.org
Contact Person Name
Manuel Sánchez Luna
Contact Person Email
msluna@salud.madrid.org
Site Name
Hospital Universitario La Paz
Department Name
Neonatology
Principal Investigator Name
Ana María Sanchez Torres
Principal Investigator Email
asancheztorres@salud.madrid.org
Contact Person Name
Ana María Sanchez Torres
Site Name
Hospital Universitario De Cruces
Department Name
Neonatology
Principal Investigator Name
Margarita Ferrer
Principal Investigator Email
margarita.ferrerarriazu@osakidetza.eus
Contact Person Name
Margarita Ferrer
Site Name
Hospital General Universitario Dr. Balmis
Department Name
Neonatology
Principal Investigator Name
Caridad Tapia
Principal Investigator Email
catapiac@gmail.com
Contact Person Name
Caridad Tapia
Contact Person Email
catapiac@gmail.com
Site Name
Hospital Universitario Puerta Del Mar
Department Name
Neonatology
Principal Investigator Name
Almudena Alonso
Principal Investigator Email
aaojemba@gmail.com
Contact Person Name
Almudena Alonso
Contact Person Email
aaojemba@gmail.com
Site Name
Hospital Sant Joan De Deu Barcelona
Department Name
N
Principal Investigator Name
Cristina Carrasco Carrasco
Principal Investigator Email
cristina.carrasco@sjd.es
Contact Person Name
Cristina Carrasco Carrasco
Contact Person Email
cristina.carrasco@sjd.es
Site Name
Hospital Universitari Arnau De Vilanova De La Gerencia Territorial De Lleida
Department Name
Neonatology
Principal Investigator Name
Eduard Solé
Principal Investigator Email
esolem.lleida.ics@gencat.cat
Contact Person Name
Eduard Solé
Contact Person Email
esolem.lleida.ics@gencat.cat
Site Name
Hospital De La Santa Creu I Sant Pau
Department Name
Neonatology
Principal Investigator Name
Elisenda Moliner
Principal Investigator Email
emoliner@santpau.cat
Contact Person Name
Elisenda Moliner
Contact Person Email
emoliner@santpau.cat
Site Name
Complexo Hospitalario Universitario De Santiago
Department Name
Neonatology
Principal Investigator Name
María Luz Couce
Principal Investigator Email
maria.luz.couce.pico@sergas.es
Contact Person Name
María Luz Couce
Contact Person Email
maria.luz.couce.pico@sergas.es
Site Name
Hospital Unviersitario Miguel Servet
Department Name
Neonatology
Principal Investigator Name
Segundo Rite
Principal Investigator Email
segundorite@gmail.com
Contact Person Name
Segundo Rite
Contact Person Email
segundorite@gmail.com
Site Name
Hospital Universitario Regional De Malaga
Department Name
Neonatology
Principal Investigator Name
María del Mar Serrano
Principal Investigator Email
mmarser@live.com
Contact Person Name
María del Mar Serrano
Contact Person Email
mmarser@live.com
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Neonatology
Principal Investigator Name
Marta Aguar
Principal Investigator Email
maraca@alumni.uv.es
Contact Person Name
Marta Aguar
Contact Person Email
maraca@alumni.uv.es
Site Name
Hospital Clinic De Barcelona
Department Name
Neonatology
Principal Investigator Name
Victoria Aldecoa Bilbao
Principal Investigator Email
valdecoa@clinic.cat
Contact Person Name
Victoria Aldecoa Bilbao
Contact Person Email
valdecoa@clinic.cat

Italy

Earliest CTIS Part Ii Submission Date
24-06-2024
Latest Decision Or Authorization Date
30-09-2024
Processing Time Days
98
Number Of Sites
6
Number Of Participants
45

Sites

Site Name
Azienda Ospedaliera di Padova
Department Name
Department of Woman's and Child's health
Principal Investigator Name
Luca Bonadies
Principal Investigator Email
luca.bonadies@unipd.it
Contact Person Name
Luca Bonadies
Contact Person Email
luca.bonadies@unipd.it
Site Name
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Department Name
Dipartimento Area Materno Infantile - Neonatologia e Terapia Intensiva Neonatale
Principal Investigator Name
Roberto Raschetti
Principal Investigator Email
roberto.raschetti@policlinico.mi.it
Contact Person Name
Roberto Raschetti
Site Name
Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
Department Name
Departmen of Woman and Child
Principal Investigator Name
Arianna Aceti
Principal Investigator Email
arianna.aceti2@unibo.it
Contact Person Name
Arianna Aceti
Contact Person Email
arianna.aceti2@unibo.it
Site Name
IRCCS Istituto Giannina Gaslini
Department Name
U.O.C Patologia Neonatale
Principal Investigator Name
Luca Antonio Ramenghi
Principal Investigator Email
lucaramenghi@gaslini.org
Contact Person Name
Luca Antonio Ramenghi
Contact Person Email
lucaramenghi@gaslini.org
Site Name
Azienda Ospedaliera Universitaria Federico II Di Napoli
Department Name
Materno Infantile / Departement of Traslational Medical Sciences
Principal Investigator Name
Letizia Capasso
Principal Investigator Email
letizia.capasso@unina.it
Contact Person Name
Letizia Capasso
Contact Person Email
letizia.capasso@unina.it
Site Name
ASST Fatebenefratelli Sacco
Department Name
Department of the mother and the neonate
Principal Investigator Name
Francesca Castoldi
Principal Investigator Email
francesca.castoldi@asst-fbf-sacco.it
Contact Person Name
Francesca Castoldi

Germany

Earliest CTIS Part Ii Submission Date
06-02-2026
Latest Decision Or Authorization Date
11-02-2026
Processing Time Days
5
Number Of Sites
7
Number Of Participants
18

Sites

Site Name
Universitaetsklinikum Wuerzburg AöR
Department Name
Children's Hospital
Principal Investigator Name
Christoph Härtel
Principal Investigator Email
Haertel_c1@ukw.de
Contact Person Name
Christoph Härtel
Contact Person Email
Haertel_c1@ukw.de
Site Name
Universitaetsklinikum Heidelberg AöR
Department Name
Neonatology
Principal Investigator Name
Christian Gille
Principal Investigator Email
christian.gille@med.uni-heidelberg.de
Contact Person Name
Christian Gille
Site Name
University Medical Center Hamburg-Eppendorf
Department Name
Dpt of Pediatrics & Adolescent Medicine, Division of Neonatology & Pediatric Intensive Care Medicine
Principal Investigator Name
Irwin Reiss
Principal Investigator Email
i.reiss@uke.de
Contact Person Name
Irwin Reiss
Contact Person Email
i.reiss@uke.de
Site Name
Klinikum Nuernberg
Department Name
Department of Paediatrics
Principal Investigator Name
Christoph Fusch
Principal Investigator Email
Christoph.fusch@klinikum-nuernberg.de
Contact Person Name
Christoph Fusch
Site Name
Medical Center - University Of Freiburg
Department Name
Center for Pediatrics
Principal Investigator Name
Hans Fuchs
Principal Investigator Email
Hans.fuchs@uniklinik-freiburg.de
Contact Person Name
Hans Fuchs
Site Name
Universitaetsklinikum Regensburg AöR
Department Name
Neonatology
Principal Investigator Name
Sven Matthias Wellmann
Principal Investigator Email
sven.wellmann@ukr.de
Contact Person Name
Sven Matthias Wellmann
Contact Person Email
sven.wellmann@ukr.de
Site Name
Universitaetsklinikum Carl Gustav Carus Dresden an der Technischen Universitaet Dresden AöR
Department Name
Department of Pediatrics, Division of Neonatology and Pediatric Intensive Care
Principal Investigator Name
Lars Mense
Principal Investigator Email
Lars.Mense@ukdd.de
Contact Person Name
Lars Mense
Contact Person Email
Lars.Mense@ukdd.de

Sponsor

Primary sponsor

Full Name
Airway Therapeutics Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Third parties

  • {"country":"Belgium","full_name":"Clinigen Clinical Supplies Management","duties_or_roles":"codes: 11,4","organisation_type":"Pharmaceutical company"}
  • {"country":"Germany","full_name":"Clinigen Clinical Supplies Management GmbH","duties_or_roles":"codes: 12,14","organisation_type":"Pharmaceutical company"}
  • {"country":"Croatia","full_name":"Optimapharm d.o.o.","duties_or_roles":"codes: 1,12,2,5,8","organisation_type":"Pharmaceutical company"}

Co-sponsors

  • Airway Therapeutics Spain S.L.

Investigational products

Investigational Product Name
zelpultide alfa
Active Substance
RECOMBINANT HUMAN SURFACTANT PROTEIN-D
Modality
Peptide/protein/enzyme
Routes Of Administration
Endotracheopulmonary instillation
Route
Endotracheopulmonary instillation
Authorisation Status
prodAuthStatus: 1
Orphan Designation
Yes
Starting Dose
4 mg/kg
Dose Levels
4 mg/kg; 6 mg/kg
Maximum Dose
6 mg/kg (max daily); 42 mg/kg (max total)
Dose Escalation Increase
initial 4 mg/kg and following 6 mg/kg
Investigational Product Name
Endotracheopulmonary instillation, room air
Modality
Other
Routes Of Administration
Endotracheopulmonary instillation
Route
Endotracheopulmonary instillation
Combination Treatment
Yes

Related trials

Other published trials that may interest you.