Evaluation of frequency occurrence persistent pulmonary hypertension of newborns treated by salbutamol

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Medical University Of Warsaw

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

15 Nov 2021 → 4 Nov 2025

Decision date (initial)

2024-12-16

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Medical Research Agency based on the Medical University of Warsaw proposal no: 2019/ABM/01/00058

External identifiers

EU CT number

2024-515216-46-00

EudraCT number

2020-003913-36

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Therapy

Assessment of frequency occurrence PPHN defined as the need to use ventilation with an additional oxygen fraction in the respiratory mixture (FiO2)> 0.30 and echocardiographic features of increased pulmonary pressure (right - left or bi-directional leakage through an unobstructed arterial duct or oval opening) or the difference in pre- and post-ductal saturation > 10%

Secondary objectives 1

1. Evaluation on the TTN scale (modified Silverman score) in comparison to the concentration of oxygen used during ventilation and acid-base equilibrium parameters: pH, carbon dioxide pressure (pCO2) and alkaline deficiency (BE) in compared groups

Conditions and MedDRA coding

Breathing disorders as a cause of neonatal respiratory failure

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Screening / Eligibility criteria: Newborns born from a pregnancy lasting min. 32 0/7 and no longer than 41 6/7 weeks. Eligibility period from birth until the inclusion criteria are met or 24 hours are reached, whichever comes first.
2. Inclusion criteria: Each Patient in the Study must meet all of the following inclusion criteria:
3. 1. Disturbances of tachypnoe breathing and expiratory bulking persisting for more than 15 minutes after birth or the above mentioned respiratory disturbances occurring up to the age of 6 hours for at least 15 minutes or the need for breathing support at birth before the age of 6 hours.
4. 2. Newborns born from a pregnancy of at least 32 0/7 weeks to 41 6/7 weeks.
5. 3. Chest X-ray image taken up to 6 hours of age.
6. 4. Performed acid-base balance (ABS) test from umbilical cord blood or capillary blood drawn from the heel of a newborn in the first hour of life.

Exclusion criteria 9

1. If the Patient meets one of the following conditions cannot be included in the Study: 1. Need for intubation immediately after birth.
2. 2. Perinatal hypoxia defined on the basis of an abnormal acid-base balance (ABS) result from umbilical cord blood or capillary blood taken from the heel of a newborn in the first hour of life, with a pH < 7.0 or BE below -14 mmol/L.
3. 3. Age over 24 hours from birth.
4. 4. Meconium aspiration syndrome.
5. 5. Neonatal air leak syndrome.
6. 6. Congenital heart defects.
7. 7. Congenital diaphragmatic hernia.
8. 8. Other severe congenital malformations and genetically determined syndromes associated with a higher risk of respiratory failure diagnosed.
9. 9. Necessity for surfactant administrating directly after birth regardless of the method of administration (respiratory distress syndrome - RDS).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Assessment of the incidence of PPHN defined as the need for ventilation with additional oxygen fraction in the respiratory mixture (FiO2) > 0. 30 and echocardiographic features of elevated pulmonary pressure or the difference in pre and postprandial saturation > 10%

Secondary endpoints 6

1. 1. Scoring on the TTN score.
2. 2. Prevalence of failures defined as the need for intubation.
3. 3. Duration of respiratory support.
4. 4. Hospitalisation time.
5. 5. Results of the acid-base balance (ABS) and ionogram.
6. 6. Occurrence of Adverse Events.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Medical University Of Warsaw

Sponsor organisation

Medical University Of Warsaw

Address

Ul. Zwirki I Wigury 61

City

Warsaw

Postcode

02-091

Country

Poland

Scientific contact point

Organisation

Medical University Of Warsaw

Contact name

Renata Bokiniec / Warszawski Uniwersytet Medyczny

Public contact point

Organisation

Medical University Of Warsaw

Contact name

Renata Bokiniec / Warszawski Uniwersytet Medyczny

Third parties 2

Locations

1 EU/EEA country · 10 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 5 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications