Predictors of Inhaled Nitric Oxide Responsiveness in Patients With PPHN

NCT06043635 | Withdrawn

• 1 other identifier: IRB18-00711
• Study Type: observational
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

To identify biochemical, clinical, or genetic biomarkers that may predict responsiveness to iNO in neonates with PPHN/HRF. The primary outcome will be identification of any biomarker(s) associated with response to iNO therapy. We will evaluate related biomarkers at various time-points during disease progression and in response to therapy, including single nucleotide polymorphisms in the cyclic adenosine monophosphate/cyclic guanosine monophosphate-Phosphodiesterase (PDE) pathway, indicators of metabolic dysregulation and inflammation, as well as biochemical markers of heart strain. We will perform targeted neonatal echocardiograms to evaluate severity of PPHN and heart function both as an added clinical biomarker and to follow disease progression.

Conditions

PPHN; Hypoxic Respiratory Failure

Outcome Measures

Primary Outcomes (1)

• Biomarker Identification

  Identification of a biomarker that is associated with response to iNO therapy

  24 hours after enrollment

Secondary Outcomes (1)

• Biomarker Predication

  24 hours after enrollment

Eligibility Criteria

• Age: Up to 10 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Late pre-term and term neonates admitted to NCH NICU with a diagnosis of PPHN/HRF.

You may qualify if:

• Greater than 34 weeks gestational age.
• Less than or equal to 10 postnatal days of age.
• Echocardiographic evidence of PPHN (right-to-left or bidirectional shunt at the foramen ovale or the ductus arteriosus and/or severe tricuspid regurgitation)

You may not qualify if:

• Lethal congenital anomalies, obvious syndromic or chromosomal disorders.
• Congenital diaphragmatic hernia.
• Congenital heart defect (except a small atrial septal defect, ventricular septal defect, and/or PDA).

Sponsors & Collaborators

• Nationwide Children's Hospital: lead

Biospecimen

Retention: SAMPLES WITH DNA

blood, urine, tracheal aspirate.

Study Officials

• Bernadette Chen, MD

  Nationwide Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: September 12, 2023
• First Posted: September 21, 2023
• Study Start: July 15, 2021
• Primary Completion: December 1, 2022
• Study Completion: December 1, 2022
• Last Updated: September 21, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share