PPHNPINORNCT06043635
Predictors of Inhaled Nitric Oxide Responsiveness in Patients With PPHN
An observational study of PPHN and Hypoxic Respiratory Failure, run by Nationwide Children's Hospital.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 10 days
- Conditions the study is for
- PPHN, Hypoxic Respiratory Failure
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 15 Jul 2021to 1 Dec 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 10 days
- All sexes
- No healthy volunteers
- Child
Study population: Late pre-term and term neonates admitted to NCH NICU with a diagnosis of PPHN/HRF.
Who may be able to join
- 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)
Who cannot join
- 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).
About this study
Official title: Predictors of Inhaled Nitric Oxide (iNO) Responsiveness in Neonates With Persistent Pulmonary Hypertension of the Newborn (PPHN)/Hypoxic Respiratory Failure (HRF)
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.
Read the detailed description
A single-center, prospective observational cohort study evaluating potential biochemical, clinical, and/or genetic biomarkers in neonates admitted with a diagnosis of PPHN that may predict iNO response. Thus, it is important to collect data prior to the initiation of iNO to be able to identify biomarkers that can predict response. For patients who meet the screening criteria and when consent is not attainable prior to initiation of the potential life-saving iNO therapy, then samples will be obtained with deferred written informed parental consent obtained within the first 4 hours after enrollment. These patients will have one sample of blood, urine, and tracheal aspirate (if available) collected. If the patient meets the inclusion and exclusion criteria, the patient will be recruited for study participation. To ensure no delay in care or in the initiation of the only FDA-approved vasodilator for this population, iNO, a deferred written informed parental consent is requested to allow for the collection of blood, urine, tracheal aspirate specimens (if available) prior to the initiation of iNO. Only one sample of blood and tracheal aspirate specimens will be collected prior to the deferred consent. Samples are often collected during the routine collections that are obtain from patients who are critically ill such as in our study participants, therefore, no additional discomfort will be expected from this study. Urine collection will be from cotton ball collection that would normally be discarded and is noninvasive. All data and biospecimens obtained from eligible infants whose parent(s) decline consent or do not qualify for the study will be discarded.
- Conditions
- PPHN, Hypoxic Respiratory Failure
What is being measured
The primary outcome is the main question the study is designed to answer.
Biomarker Identification
Identification of a biomarker that is associated with response to iNO therapy
Measured: 24 hours after enrollment
Secondary outcomes (1)
Biomarker Predication
Identification of a biomarker that may be predictive of the need for iNO therapy
Measured: 24 hours after enrollment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2023
- Study start15 Jul 2021
- Primary completion1 Dec 2022
- Study completion1 Dec 2022
Why it stopped: No patients were enrolled
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PPHNStudies of the same condition, open and under way.
- More trials run by Nationwide Children's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nationwide Children's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Bernadette Chen, Nationwide Children's Hospital
- Investigators
- Bernadette Chen, MD, Principal investigator, Nationwide Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06043635
- Sponsor's protocol ID
- IRB18-00711
- Acronym
- PINOR
- Status last verified
- Sep 2023
- First submitted
- 12 Sep 2023
- Last updated
- 21 Sep 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Predictors of Inhaled Nitric Oxide (iNO) Responsiveness in Neonates With Persistent Pulmonary Hypertension of the Newborn (PPHN)/Hypoxic Respiratory Failure (HRF)
- ClinicalTrials.gov ID
- NCT06043635
- Acronym / sponsor ID
- PINOR / IRB18-00711
- Conditions
- PPHN, Hypoxic Respiratory Failure
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: No patients were enrolled
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Nationwide Children's HospitalUniversity, hospital or other
- Study start
- 15 Jul 2021 (actual)
- Primary completion
- 1 Dec 2022 (actual)
- Study completion
- 1 Dec 2022 (actual)
- First posted
- 21 Sep 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
A single-center, prospective observational cohort study evaluating potential biochemical, clinical, and/or genetic biomarkers in neonates admitted with a diagnosis of PPHN that may predict iNO response. Thus, it is important to collect data prior to the initiation of iNO to be able to identify biomarkers that can predict response. For patients who meet the screening criteria and when consent is not attainable prior to initiation of the potential life-saving iNO therapy, then samples will be obtained with deferred written informed parental consent obtained within the first 4 hours after enrollment. These patients will have one sample of blood, urine, and tracheal aspirate (if available) collected. If the patient meets the inclusion and exclusion criteria, the patient will be recruited for study participation. To ensure no delay in care or in the initiation of the only FDA-approved vasodilator for this population, iNO, a deferred written informed parental consent is requested to allow for the collection of blood, urine, tracheal aspirate specimens (if available) prior to the initiation of iNO. Only one sample of blood and tracheal aspirate specimens will be collected prior to the deferred consent. Samples are often collected during the routine collections that are obtain from patients who are critically ill such as in our study participants, therefore, no additional discomfort will be expected from this study. Urine collection will be from cotton ball collection that would normally be discarded and is noninvasive. All data and biospecimens obtained from eligible infants whose parent(s) decline consent or do not qualify for the study will be discarded.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- Up to 10 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Late pre-term and term neonates admitted to NCH NICU with a diagnosis of PPHN/HRF.
Key inclusion criteria (3 listed)
- 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)
Key exclusion criteria (3 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Biomarker Identification
Time frame: 24 hours after enrollment
Identification of a biomarker that is associated with response to iNO therapy
1 secondary outcome
Biomarker Predication
Time frame: 24 hours after enrollment
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Sep 2023.
- Individual participant dataSharing planned: No
Related trials for PPHN
Active, not recruitingPhase 2
Effect of a Parenteral Emulsion With Omega3 on Neonates With PPHN and CDH
Pulmonary Hypertension of Newborn, Diaphragm Defect
Recruiting
Effect of Melatonin on Pulmonary Hypertension in Full Term Neonates
Congenital Peumonia, Persistent Pulmonary Hypertension of Newborn
Not yet recruitingObservational study
Post Market Clinical Follow-up to Assess the Portable Nitric Oxide Generation and Delivery System (INOwill N200) for the Treatment of Neonatal Hypoxic Respiratory Failure Complicated by Pulmonary Hypertension
Persistent Pulmonary Hypertension of the Newborn, Hypoxic Respiratory Failure
Not yet recruitingObservational study
Predictors of Outcomes in Pulmonary Hypertension of the Newborn
Hypertension, Pulmonary, Infant, Newborn, Diseases
RecruitingPhase 4
Dopamine vs. Norepinephrine for Hypotension in Neonates With Pulmonary Hypertension (DONE)
Hypotension and Shock, Pulmonary Hypertension of the Newborn (PPHN)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bernadette Chen, MD principal investigator
Nationwide Children's Hospital
Questions about this trial
What is this clinical trial studying?
An observational study of PPHN and Hypoxic Respiratory Failure, run by Nationwide Children's Hospital.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 21 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Biomarker Identification (time frame: 24 hours after enrollment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Sep 2023.