Inhaled Treatment for Bronchopulmonary Dysplasia

NCT04619602 | Withdrawn Phase 1 DMC FDA Drug

• 1 other identifier: BPD
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The primary objective of this study is to provide expanded access of S-nitrosylation therapy for the treatment of bronchopulmonary dysplasia

Conditions

Bronchopulmonary Dysplasia

Keywords

bronchopulmonary dysplasia; respiratory failure

Outcome Measures

Primary Outcomes (1)

• Treatment Emergent Adverse Events

  Occurrence of \>grade 3 adverse events related to the treatment

  7 days

Secondary Outcomes (5)

• S-nitrosoglutathione change

  30 minutes

• Change in oxygen saturation index

  4 hours

• Change in GSNO catabolism pre/post treatment

  30 minutes

• Intermittent hypoxemia as measured by oxygen saturation post treatment

  4 hours

• Ventilator parameters post treatment

  4 hours

Study Arms (1)

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.

Drug: GSNO

Interventions

GSNO DRUG

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.

GSNO therapy

Eligibility Criteria

• Age: 29 Days - 365 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• University Hospitals Cleveland Medical Center: lead

Study Sites (1)

University Hospitals Cleveland Medical Center Rainbow Babies and Children's Hospital

Cleveland, Ohio, 44106, United States

Location

MeSH Terms

Conditions

Bronchopulmonary Dysplasia; Respiratory Insufficiency

Condition Hierarchy (Ancestors)

Ventilator-Induced Lung Injury; Lung Injury; Lung Diseases; Respiratory Tract Diseases; Infant, Premature, Diseases; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Respiration Disorders

Study Officials

• Thomas Raffay, MD

  University Hospitals Cleveland Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Sponsor-Investigator

Study Record Dates

• First Submitted: November 2, 2020
• First Posted: November 6, 2020
• Study Start: August 1, 2025
• Primary Completion: September 1, 2025
• Study Completion: September 1, 2025
• Last Updated: August 22, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)