The COllaborative Neonatal Network for the First CPAM Trial

NCT05701514 | Recruiting DMC

• 1 other identifier: CONNECT-study
• Study Type: interventional
• Target: 176
• Locations: 1 country
• Sites: 2

Brief Summary

The goal of this clinical trial is to compare conservative wait-and-see management to elective surgical intervention, in asymptomatic Congenital Pulmonary Airway Malformation (CPAM) children. Children assigned to the intervention group will undergo surgical resection of the CPAM between 6 and 9 months of age. Children assigned to the control group will be monitored conservatively. The follow-up scheme will be uniform for both treatment groups and last for 5 years. The primary outcome is the difference in maximal endurance at five years of age between the surgical and conservative group. Secondary outcome measures are molecular genetic diagnostics, validated questionnaires - on parental anxiety, quality of life and health care consumption -, repeated imaging, and pulmonary morbidity during follow-up, as well as surgical complications and histopathology.

Conditions

Congenital Pulmonary Airway Malformation

Keywords

Asymptomatic CPAM; Optimal management; Conservative vs surgical; Randomized

Outcome Measures

Primary Outcomes (1)

• Exercise tolerance

  BRUCE treadmill test protocol. Total endurance time will be converted to a sex and age matched percentile score based on pre-defined reference values.

  5 years

Secondary Outcomes (13)

• Pulmonary morbidity during follow-up

  through study completion, a total of 5 years

• Surgical intervention due to pulmonary morbidity

  Through study completion, a total of 5 years

• CPAM characteristics on prenatal ultrasound images, according to standardized structured report

  20 weeks gestation

• CPAM characteristics on postnatal CT-scan, according to standardized structured report

  3-9 months of age

• CPAM development / post-surgical appearance on repeated CT imaging, according to standardized structured report

  2.5 years of age

Study Arms (2)

Conservative arm

NO INTERVENTION

Children assigned to this treatment arm will be conservatively managed, and will continue to be followed until the age of 5 years (end of study inclusion).

Surgical arm

ACTIVE COMPARATOR

Children assigned to this treatment arm will undergo elective surgical resection at the age of 6-9 months, and will continue to be followed until the age of 5 years (end of study inclusion).

Procedure: Elective surgical resection of CPAM between 6 and 9 months of age

Interventions

Elective surgical resection of CPAM between 6 and 9 months of age PROCEDURE

Surgical resection of the CPAM between 6 and 9 months of age

Surgical arm

Eligibility Criteria

• Age: 1 Month - 1 Year
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Lesion detected during routine prenatal ultrasound screening
• Delivery at term: gestational age ≥37 weeks
• Birthweight \> -2SD or \>P10
• Asymptomatic at birth defined as no prolonged respiratory distress or oxygen support (\< 24 hours)
• Confirmation of CPAM on postnatal chest CT-scan at 3-9 months of age, according to structured report form (34)
• Unilateral lesion occupying no more than one lung lobe as assessed on chest CT-scan at 3-9 months of age

You may not qualify if:

• Bilateral lesion
• Development of symptoms before randomization, considered by treating physician as caused by CPAM with reasonable certainty
• Complicated pregnancy defined as (pre-)eclampsia, pregnancy diabetes in mother, foetal hydrops or severe polyhydramnios on prenatal ultrasound
• Syndrome associated anomalies on genetic analysis confirmed by genetic expert
• Major associated malformations. Anomalies include cardiac malformations requiring surgical correction or follow-up by a paediatric cardiologist, congenital malformations requiring major surgical intervention, and anomalies that affect normal lung growth and development.
• Suspicion of malignancy on chest CT scan evaluation at the age of 3-9 months
• Participation in another randomised controlled trial

Sponsors & Collaborators

• Erasmus Medical Center: lead

Study Sites (2)

Radboud University Medical Centre

Nijmegen, Gelderlanf, 6525GA, Netherlands

NOT YET RECRUITING

Erasmus MC Sophia Children's Hospital

Rotterdam, South Holland, 3015GD, Netherlands

RECRUITING

Related Publications (3)

• Hermelijn S, Kersten C, Mullassery D, Muthialu N, Cobanoglu N, Gartner S, Bagolan P, Mesas Burgos C, Sgro A, Heyman S, Till H, Suominen J, Schurink M, Desender L, Losty P, Ertresvag K, Tiddens HAWM, Wijnen RMH, Schnater M; CONNECT study consortium COS development group; CONNECT study consortium COS development group. Development of a core outcome set for congenital pulmonary airway malformations: study protocol of an international Delphi survey. BMJ Open. 2021 Apr 12;11(4):e044544. doi: 10.1136/bmjopen-2020-044544.

  PMID: 33846152 BACKGROUND

• Kersten CM, Hermelijn SM, Mullassery D, Muthialu N, Cobanoglu N, Gartner S, Bagolan P, Mesas Burgos C, Sgro A, Heyman S, Till H, Suominen J, Schurink M, Desender L, Losty P, Steyaert H, Terheggen-Lagro S, Metzelder M, Bonnard A, Sfeir R, Singh M, Yardley I, Rikkers-Mutsaerts NRVM, van der Ent CK, Qvist N, Cox DW, Peters R, Bannier MAGE, Wessel L, Proesmans M, Stanton M, Hannon E, Zampoli M, Morini F, Tiddens HAWM, Wijnen RMH, Schnater JM. The Management of Asymptomatic Congenital Pulmonary Airway Malformation: Results of a European Delphi Survey. Children (Basel). 2022 Jul 30;9(8):1153. doi: 10.3390/children9081153.

  PMID: 36010044 BACKGROUND

• Kersten CM, Hermelijn SM, Dossche LWJ, Muthialu N, Losty PD, Schurink M, Rietman AB, Poley MJ, van Rosmalen J, Zanen-van den Adel TPL, Ciet P, von der Thusen J, Brosens E, Ijsselstijn H, Tiddens HAWM, Wijnen RMH, Schnater JM. COllaborative Neonatal Network for the first European CPAM Trial (CONNECT): a study protocol for a randomised controlled trial. BMJ Open. 2023 Mar 17;13(3):e071989. doi: 10.1136/bmjopen-2023-071989.

  PMID: 36931672 DERIVED

MeSH Terms

Conditions

Cystic Adenomatoid Malformation of Lung, Congenital

Interventions

Aging

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Respiratory System Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Growth and Development; Physiological Phenomena

Study Officials

• Rene MH Wijnen, professor

  Head of department Pediatric Surgery, Erasmus MC Sophia Children's Hospital, Rotterdam

  STUDY DIRECTOR

Central Study Contacts

J Marco Schnater, MD, PhD

CONTACT

0031 107036923; j.schnater@erasmusmc.nl

Rene MH Wijnen, professor

CONTACT

0031 107036923; r.wijnen@erasmusmc.nl

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: dr. J.M. Schnater, Principal Investigator

Model Details: Randomised, controlled, multicentre superiority trial. Randomisation will take place as blocked randomisation with stratification by centre, with a 1:1 allocation.

Study Record Dates

• First Submitted: December 15, 2022
• First Posted: January 27, 2023
• Study Start: January 1, 2023
• Primary Completion: December 31, 2025
• Study Completion (Estimated): December 31, 2030
• Last Updated: March 4, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: Starting 6 months after publication
• Access Criteria: Open access

Locations

NL (2)