Bronchopulmonary DysplasiaPhase 1NCT04619602
Inhaled Treatment for Bronchopulmonary Dysplasia
A Phase 1 study testing GSNO in people with Bronchopulmonary Dysplasia, run by University Hospitals Cleveland Medical Center at 1 site in United States.
Trial summary
The primary objective of this study is to provide expanded access of S-nitrosylation therapy for the treatment of bronchopulmonary dysplasia Read more about this study
- Who is it for?
- Ages 29 to 365 days
- Conditions the study is for
- Bronchopulmonary Dysplasia
- What is being tested?
- GSNO
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Aug 2025to 1 Sep 2025
- Phase
- Phase 1
- 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 29 to 365 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Inborn or outborn infants of either sex or any race or ethnicity
- <32 weeks gestation at birth (best obstetrical dating)
- Aged 29 to 365 days
- Refractory hypoxic respiratory failure (average daily FiO2 >35% for 5 days)
- Requires mechanical ventilation via endotracheal airway
Who cannot join
- Life-threatening congenital or acquired anomalies (lethal chromosomal, thoracic/cardiac, brain)
- Unstable condition defined as severe hypoxemia (FiO2 >85% for >24hrs), sepsis, or hypotension
- Baseline methemoglobin > 3%, congenital methemoglobinemia, or a familial hemoglobinopathy
- On steroid to facilitate endotracheal extubation
- Individuals on inhaled nitric oxide, a phosphodiesterase 5 (PDE-5) inhibitor, taking allopurinol, β-adrenergic blockers, tricyclic antidepressants, meperidine (or related CNS agents), or nitrates
- Thrombocytopenia defined as <50,000 platelets/µL on weekly NICU labs, clinical evidence of bleeding, on an anti-coagulant, or individuals with an inherited or acquired coagulation disorder
Show all 12Show fewer
- Anemia defined as a hemoglobin of < 9 mg/dL on weekly NICU labs
- Concerns for pre-existing liver damage defined as an AST/ALT > 50 IU/L or direct bilirubin >1 mg/dL on weekly NICU labs
- Concerns for acute kidney injury defined as a serum creatinine > 0.7 mg/dL on weekly NICU labs or 24-hr urine output <1.0 ml/kg/hr during preceding 4 days
- Patients that are ventilated with a device not certified for blending of aerosolized solutions into the ventilator circuit
- Physician of record opposed to enrolling the patient due to perceived safety concerns; or any condition that does not allow the protocol to be followed safely
- Subjects that have experienced cardiac arrest with CPR for longer than 30 minutes
Trial locations and contacts
1 site in 1 country.
United States1 site
University Hospitals Cleveland Medical Center Rainbow Babies and Children's Hospital
Cleveland, Ohio, 44106
About this study
Official title: Pilot Study of an Inhaled Treatment for Bronchopulmonary Dysplasia
The primary objective of this study is to provide expanded access of S-nitrosylation therapy for the treatment of bronchopulmonary dysplasia
Read the detailed description
Open label study with 20 participants, open-label, with block dose escalation of 3 subjects/dose (0.5 mL/kg of 0.25 mM, 0.5 mM, or 1 mM). A minimum of seven days of surveillance will separate dosing blocks. An additional 11 subjects will be enrolled at the maximum 1 mM block (5x10-7 moles/kg). The primary outcomes are safety during 30 minutes of inhalation, and for 4 hours after inhalation, as measured by occurrence of adverse events related to the treatment + time period [during administration and tracked for next 7 days].
- Conditions
- Bronchopulmonary Dysplasia
- Being tested
- GSNO
- Keywords
- bronchopulmonary dysplasia, respiratory failure
- Medical subject headings
- Bronchopulmonary Dysplasia, Respiratory Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GSNODrug
Intervention will be 30 minutes of inhaled GSNO agent in enrollment blocks of three subjects/dose (0.5 mL/kg of 0.25 mM, 0.5 mM, or 1 mM) to infants.
| Study group | Role | What they receive |
|---|---|---|
| GSNO therapy | Experimental | Intervention will be 30 minutes of inhaled GSNO agent in enrollment blocks of three subjects/dose (0.5 mL/kg of 0.25 mM, 0.5 mM, or 1 mM) to infants. Receives: Drug: GSNO |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment Emergent Adverse Events
Occurrence of >grade 3 adverse events related to the treatment
Measured: 7 days
Secondary outcomes (5)
S-nitrosoglutathione change
Percent change in S-nitrosoglutathione pre/post treatment
Measured: 30 minutes
Change in oxygen saturation index
Percent change in oxygen saturation index (O.S.I.) pre/post treatment. oxygen saturation index [O.S.I. = (FiO2) x (mean airway pressure) x 100 / (SpO2)]. High OSI scores indicate worse respiratory failure (high OSI = bad, low OSI = good).Scale range: zero - infinity
Measured: 4 hours
Change in GSNO catabolism pre/post treatment
Percent change in GSNO catabolism pre/post treatment
Measured: 30 minutes
Intermittent hypoxemia as measured by oxygen saturation post treatment
Incidence, duration, and nadirs of intermittent hypoxemia (SpO2 <80%) as measured by oxygen saturation parameters post treatment
Measured: 4 hours
Ventilator parameters post treatment
Change in ventilator parameters post treatment
Measured: 4 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Nov 2020
- Study start1 Aug 2025 (estimated)
- Primary completion1 Sep 2025 (estimated)
- Study completion1 Sep 2025 (estimated)
Why it stopped: FDA hold
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bronchopulmonary DysplasiaStudies of the same condition, open and under way.
- More trials run by University Hospitals Cleveland Medical CenterThe 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospitals Cleveland Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Thomas Michael Raffay, MD, University Hospitals Cleveland Medical Center
- Investigators
- Thomas Raffay, MD, Principal investigator, University Hospitals Cleveland Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04619602
- Sponsor's protocol ID
- BPD
- Status last verified
- Aug 2025
- First submitted
- 2 Nov 2020
- Last updated
- 22 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of an Inhaled Treatment for Bronchopulmonary Dysplasia
- ClinicalTrials.gov ID
- NCT04619602
- Acronym / sponsor ID
- BPD
- Conditions
- Bronchopulmonary Dysplasia
- Keywords
- bronchopulmonary dysplasia, respiratory failure
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: FDA hold
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University Hospitals Cleveland Medical CenterUniversity, hospital or other
- Study start
- 1 Aug 2025 (estimated)
- Primary completion
- 1 Sep 2025 (estimated)
- Study completion
- 1 Sep 2025 (estimated)
- First posted
- 6 Nov 2020 (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.
The primary objective of this study is to provide expanded access of S-nitrosylation therapy for the treatment of bronchopulmonary dysplasia
Detailed description
Open label study with 20 participants, open-label, with block dose escalation of 3 subjects/dose (0.5 mL/kg of 0.25 mM, 0.5 mM, or 1 mM). A minimum of seven days of surveillance will separate dosing blocks. An additional 11 subjects will be enrolled at the maximum 1 mM block (5x10-7 moles/kg). The primary outcomes are safety during 30 minutes of inhalation, and for 4 hours after inhalation, as measured by occurrence of adverse events related to the treatment + time period [during administration and tracked for next 7 days].
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GSNO therapy | Experimental | Drug: GSNOIntervention will be 30 minutes of inhaled GSNO agent in enrollment blocks of three subjects/dose (0.5 mL/kg of 0.25 mM, 0.5 mM, or 1 mM) to infants. |
Who may qualify
- Ages
- 29 to 365 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Inborn or outborn infants of either sex or any race or ethnicity
- <32 weeks gestation at birth (best obstetrical dating)
- Aged 29 to 365 days
- Refractory hypoxic respiratory failure (average daily FiO2 >35% for 5 days)
- Requires mechanical ventilation via endotracheal airway
Key exclusion criteria (12 listed)
- Life-threatening congenital or acquired anomalies (lethal chromosomal, thoracic/cardiac, brain)
- Unstable condition defined as severe hypoxemia (FiO2 >85% for >24hrs), sepsis, or hypotension
- Baseline methemoglobin > 3%, congenital methemoglobinemia, or a familial hemoglobinopathy
- On steroid to facilitate endotracheal extubation
- Individuals on inhaled nitric oxide, a phosphodiesterase 5 (PDE-5) inhibitor, taking allopurinol, β-adrenergic blockers, tricyclic antidepressants, meperidine (or related CNS agents), or nitrates
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GSNOIntervention will be 30 minutes of inhaled GSNO agent in enrollment blocks of three subjects/dose (0.5 mL/kg of 0.25 mM, 0.5 mM, or 1 mM) to infants. | Drug | GSNO therapy |
Primary outcome
Treatment Emergent Adverse Events
Time frame: 7 days
Occurrence of >grade 3 adverse events related to the treatment
5 secondary outcomes
S-nitrosoglutathione change
Time frame: 30 minutes
Change in oxygen saturation index
Time frame: 4 hours
Change in GSNO catabolism pre/post treatment
Time frame: 30 minutes
Intermittent hypoxemia as measured by oxygen saturation post treatment
Time frame: 4 hours
Ventilator parameters post treatment
Time frame: 4 hours
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 22 Aug 2025.
- Individual participant dataSharing planned: No
Related trials for Bronchopulmonary Dysplasia
Recruiting
Optimization of Saturation Targets And Resuscitation Trial (OptiSTART)
Premature Infants, Bronchopulmonary Dysplasia
Recruiting
Controlled Remote Monitoring and Optimization of Oxygen Therapy in Preterm Infants
BPD - Bronchopulmonary Dysplasia, Pulmonary Hypertension
Not yet recruitingPhase 1/2
Little Lungs Study
Invasive Mechanical Ventilation, Severe Respiratory Distress Syndrome
Not yet recruiting
Strap To Reduce Apnea in Preterm Infants: A Randomized Crossover Trial
Apnea of Prematurity, Intermittent Hypoxemia
Recruiting
Ventilator Pressure and Optimization of Compliance and Hemodynamics
Bronchopulmonary Dysplasia, Pulmonary Hypertension Due to Lung Diseases and Hypoxia
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.
Thomas Raffay, MD principal investigator
University Hospitals Cleveland Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing GSNO in people with Bronchopulmonary Dysplasia, run by University Hospitals Cleveland Medical Center at 1 site in United States.
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 5 inclusion and 12 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 22 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Treatment Emergent Adverse Events (time frame: 7 days). 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 22 Aug 2025.