Canadian National PDA Treatment Study

NCT ID: NCT04347720

Last Updated: 2024-06-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

1663 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-01-01

Study Completion Date

2023-12-31

Brief Summary

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Patent ductus arteriosus (PDA) is the most common cardiovascular problem that develops in preterm infants. Persistent PDA may result in higher rates of death, chronic lung disease (CLD), pulmonary hemorrhage, necrotizing enterocolitis (NEC), acute kidney injury (AKI), intraventricular hemorrhage (IVH) and cerebral palsy. Currently available options to treat a PDA include indomethacin, ibuprofen or acetaminophen followed by surgical or interventional closure of the PDA if medical therapy fails.

Wide variation exists in PDA treatment practices across Canada. A survey conducted through the Canadian Neonatal Network (CNN) in 2019 showed that the most common choice of initial pharmacotherapy is standard dose ibuprofen. In view of the high pharmacotherapy failure rate with standard dose ibuprofen, there is a growing use of higher doses of ibuprofen with increasing postnatal age (with 32% of respondents currently adopting this practice) in spite of the fact that effectiveness and safety of higher ibuprofen doses have not been established in extremely preterm infants \[\<29 weeks gestational age (GA)\]. In view of this large practice variation across Canadian neonatal intensive care units (NICUs), we are planning a comparative effectiveness study of the different primary pharmacotherapeutic agents used to treat the PDA in preterm infants.

Aims Primary: To compare the primary pharmacotherapeutic practices for PDA closure and evaluate their impact on clinical outcomes in extremely preterm infants (\<29 weeks GA) Secondary: To understand the relevance of pharmacotherapeutic PDA treatment with respect to clinical outcomes in the real world.

Methods:

Participants: Extremely preterm infants (\<29 weeks gestational age) with an echocardiography confirmed PDA who will be treated according to attending team

Interventions:

1. Standard dose ibuprofen \[10-5-5 regimen, i.e., 10mg/kg followed by 2 doses of 5mg/kg at 24h intervals\]
2. Adjustable dose ibuprofen \[10-5-5 regimen if treated within the first week. Higher doses of ibuprofen up to a 20-10-10 regimen if treated after the postnatal age cut-off for lower dose as per the local center policy\]
3. Intravenous indomethacin \[0.1-0.3mg/kg every 12-24h for a total of 3 doses\].
4. Acetaminophen \[Oral/intravenous\] (15mg/kg every 6h) for 3-7 days

Outcomes:

Primary: Failure of primary pharmacotherapy (Need for further medical and/or surgical/interventional treatment following an initial course of pharmacotherapy).

Secondary: (a) Receipt of 2nd course of pharmacotherapy; (b) Surgical/interventional PDA closure; (c) CLD (d) NEC (stage 2 or greater) (e) Severe IVH (Grade III-IV) (f) Definite sepsis (g) Stage 1 or greater AKI; (h) Post-treatment serum bilirubin; (i) Phototherapy duration; (j) All-cause mortality during hospital stay.

Detailed Description

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In this study, we intend to generate real-world evidence (RWE) by analyzing real-world data (RWD) (defined as data generated during routine clinical practice) from a registry-based Comparative Effectiveness Research study.

The Canadian Neonatal Network (CNN) is a well-established patient registry that includes members from 31 hospitals and 17 universities across Canada. The Network maintains a standardized NICU database and provides a unique opportunity for researchers to participate in collaborative projects. We will use the principles of Hypotheses Evaluating Treatment Effectiveness (HETE) research, which are designed to evaluate the presence or absence of a pre-specified effect and/or its magnitude. The network has recent experience in conducting such a study where one CIHR-funded study to evaluate effectiveness of two modes of non-invasive ventilation in preterm infants is already underway in 20 NICUs across Canada.

The CNN's coordinating facility is located within the Maternal-Infant Care (MiCare) Research Center, Lunenfeld-Tanenbaum Research Institute (LTRI) at Mount Sinai Hospital (Toronto). Each participating site has highly trained abstractors who enter data from patient charts into the CNN database. The abstractors will also enter data specific to our project, which will allow us to obtain real-world data at a minimal cost with easy access to investigators for troubleshooting.

Statistical Analysis overview: Since the proposed study is a CER using RWD, we will examine and account for potential confounders at the analyses stage. As recommended for HETE studies using RWD, accuracy of results will be checked by performing complementary sensitivity analyses. The analyses will be conducted in 2 stages: unit-level protocol effectiveness analysis and a secondary drug-dosage effectiveness analysis.

Conditions

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Patent Ductus Arteriosus Extreme Prematurity

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Indomethacin Arm

Intravenous indomethacin at 0.1-0.3 mg/kg IV every 12-24h for a total of 3 doses as choice of initial pharmacotherapy.

Indomethacin

Intervention Type DRUG

Intravenous formulation

Standard dose ibuprofen Arm

Standard dose ibuprofen \[Oral/intravenous\] at 10 mg/kg followed by 2 doses of 5mg/kg at 24 h intervals irrespective of postnatal age as choice of initial pharmacotherapy.

Ibuprofen

Intervention Type DRUG

Intravenous and oral formulations

Adjustable dose ibuprofen Arm

Adjustable dose ibuprofen \[Oral/intravenous\] as choice of initial pharmacotherapy. The dose of ibuprofen will be 10 mg/kg followed by 2 doses of 5 mg/kg at 24 h intervals if treated within the first 7 days after birth. Higher doses of ibuprofen up to 20 mg/kg followed by 2 doses of 10 mg/kg at 24 h intervals if treated after the postnatal age cut-off for lower dose as per the local center policy

Ibuprofen

Intervention Type DRUG

Intravenous and oral formulations

Acetaminophen Arm

Acetaminophen \[Oral/intravenous\] at 15mg/kg every 6h for 3-7 days as choice of initial pharmacotherapy.

Acetaminophen

Intervention Type DRUG

Intravenous and oral formulations

Control group

Infants \<29 weeks GA with echocardiography-confirmed PDA but never received any pharmacotherapy

No interventions assigned to this group

Reference group

Infants \<29 weeks GA who were never diagnosed with PDA

No interventions assigned to this group

Interventions

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Indomethacin

Intravenous formulation

Intervention Type DRUG

Ibuprofen

Intravenous and oral formulations

Intervention Type DRUG

Acetaminophen

Intravenous and oral formulations

Intervention Type DRUG

Other Intervention Names

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indocid Advil NeoProfen paracetamol

Eligibility Criteria

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Inclusion Criteria

* Extremely preterm infants (\<29 weeks gestational age) with an echocardiography confirmed PDA who will be treated according to attending team

Exclusion Criteria

* Any infant who received pharmacotherapy for a clinically symptomatic PDA without prior echocardiographic confirmation of the presence of PDA will be excluded from all analyses.
Maximum Eligible Age

12 Weeks

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Provincial Health Services Authority

OTHER

Sponsor Role collaborator

Children's Hospital of Eastern Ontario

OTHER

Sponsor Role collaborator

CHU de Quebec-Universite Laval

OTHER

Sponsor Role collaborator

Foothills Medical Centre

OTHER

Sponsor Role collaborator

Victoria General Hospital

UNKNOWN

Sponsor Role collaborator

The Hospital for Sick Children

OTHER

Sponsor Role collaborator

Health Sciences Centre, Winnipeg, Manitoba

OTHER

Sponsor Role collaborator

St. Justine's Hospital

OTHER

Sponsor Role collaborator

Queen's University

OTHER

Sponsor Role collaborator

MOUNT SINAI HOSPITAL

OTHER

Sponsor Role collaborator

Royal Alexandra Hospital

OTHER

Sponsor Role collaborator

Regina General Hospital

OTHER

Sponsor Role collaborator

Royal University Hospital Foundation

OTHER

Sponsor Role collaborator

St. Boniface Hospital

OTHER

Sponsor Role collaborator

The Moncton Hospital

UNKNOWN

Sponsor Role collaborator

Horizon Health Network

OTHER

Sponsor Role collaborator

Sunnybrook Health Sciences Centre

OTHER

Sponsor Role collaborator

Windsor Regional Hospital

OTHER

Sponsor Role collaborator

Royal Columbian Hospital Foundation

OTHER

Sponsor Role collaborator

Centre de recherche du Centre hospitalier universitaire de Sherbrooke

OTHER

Sponsor Role collaborator

London Health Sciences Centre

OTHER

Sponsor Role collaborator

Canadian Institutes of Health Research (CIHR)

OTHER_GOV

Sponsor Role collaborator

IWK Health Centre

OTHER

Sponsor Role lead

Responsible Party

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Dr. Souvik Mitra, MD MSc FRCPC

Assistant Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Souvik Mitra, MD, MSc

Role: PRINCIPAL_INVESTIGATOR

IWK Health Center, Halifax, Canada

Amish Jain, MBBS, PhD

Role: PRINCIPAL_INVESTIGATOR

Mount Sinai Hospital, Canada

Prakeshkumar Shah, MD, FRCPC

Role: PRINCIPAL_INVESTIGATOR

Mount Sinai Hospital, Canada

Locations

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Foothills Medical Centre

Calgary, Alberta, Canada

Site Status

Royal Alexandra Hospital

Edmonton, Alberta, Canada

Site Status

Royal Columbian Hospital

New Westminster, British Columbia, Canada

Site Status

British Columbia Women's Hospital

Vancouver, British Columbia, Canada

Site Status

Victoria General Hospital

Victoria, British Columbia, Canada

Site Status

Health Sciences Centre

Winnipeg, Manitoba, Canada

Site Status

St. Boniface General Hospital

Winnipeg, Manitoba, Canada

Site Status

The Moncton Hospital

Moncton, New Brunswick, Canada

Site Status

Saint John Regional Hospital

Saint John, New Brunswick, Canada

Site Status

IWK Health Center

Halifax, Nova Scotia, Canada

Site Status

Kingston Health Sciences Centre

Kingston, Ontario, Canada

Site Status

London Health Sciences Centre

London, Ontario, Canada

Site Status

Children's Hospital of Eastern Ontario

Ottawa, Ontario, Canada

Site Status

Hospital for Sick Children

Toronto, Ontario, Canada

Site Status

Mount Sinai Hospital

Toronto, Ontario, Canada

Site Status

Sunnybrook Health Sciences Centre

Toronto, Ontario, Canada

Site Status

Windsor Regional Hospital

Windsor, Ontario, Canada

Site Status

CHU Sainte-Justine

Montreal, Quebec, Canada

Site Status

Centre Hospitalier Universitaire de Quebec

Québec, Quebec, Canada

Site Status

Centre Hospitalier Universitaire de Sherbrooke

Sherbrooke, Quebec, Canada

Site Status

Regina General Hospital

Regina, Saskatchewan, Canada

Site Status

Royal University Hospital

Saskatoon, Saskatchewan, Canada

Site Status

Countries

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Canada

References

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Mitra S, Jain A, Ting JY, Ben Fadel N, Drolet C, Abou Mehrem A, Soraisham A, Jasani B, Louis D, Lapointe A, Dorling J, Khurshid F, Hyderi A, Kumaran K, Bodani J, Weisz D, Alvaro R, Adie M, Stavel M, Morin A, Bhattacharya S, Kanungo J, Canning R, Ye XY, Hatfield T, Gardner CE, Shah P. Relative effectiveness and safety of pharmacotherapeutic agents for patent ductus arteriosus (PDA) in preterm infants: a protocol for a multicentre comparative effectiveness study (CANRxPDA). BMJ Open. 2021 May 5;11(5):e050682. doi: 10.1136/bmjopen-2021-050682.

Reference Type DERIVED
PMID: 33952559 (View on PubMed)

Related Links

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http://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=410247&lang=en

Project funding information on the Canadian Institutes of Health Research (CIHR) Funding Decision Database

Other Identifiers

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1025627

Identifier Type: -

Identifier Source: org_study_id

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