Inhaled Treatment for Bronchopulmonary Dysplasia

Overview

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

Full Title of Study: “Pilot Study of an Inhaled Treatment for Bronchopulmonary Dysplasia”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: September 1, 2025

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 (5×10-7 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].

Interventions

  • Drug: GSNO
    • 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.

Arms, Groups and Cohorts

  • Experimental: GSNO therapy
    • 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.

Clinical Trial Outcome Measures

Primary Measures

  • Treatment Emergent Adverse Events
    • Time Frame: 7 days
    • Occurrence of >grade 3 adverse events related to the treatment

Secondary Measures

  • S-nitrosoglutathione change
    • Time Frame: 30 minutes
    • Percent change in S-nitrosoglutathione pre/post treatment
  • Change in oxygen saturation index
    • Time Frame: 4 hours
    • 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
  • Change in GSNO catabolism pre/post treatment
    • Time Frame: 30 minutes
    • Percent change in GSNO catabolism pre/post treatment
  • Intermittent hypoxemia as measured by oxygen saturation post treatment
    • Time Frame: 4 hours
    • Incidence, duration, and nadirs of intermittent hypoxemia (SpO2 <80%) as measured by oxygen saturation parameters post treatment
  • Ventilator parameters post treatment
    • Time Frame: 4 hours
    • Change in ventilator parameters post treatment

Participating in This Clinical Trial

Inclusion Criteria

1. Inborn or outborn infants of either sex or any race or ethnicity 2. <32 weeks gestation at birth (best obstetrical dating) 3. Aged 29 to 365 days 4. Refractory hypoxic respiratory failure (average daily FiO2 >35% for 5 days) 5. Requires mechanical ventilation via endotracheal airway Exclusion Criteria:

1. Life-threatening congenital or acquired anomalies (lethal chromosomal, thoracic/cardiac, brain) 2. Unstable condition defined as severe hypoxemia (FiO2 >85% for >24hrs), sepsis, or hypotension 3. Baseline methemoglobin > 3%, congenital methemoglobinemia, or a familial hemoglobinopathy 4. On steroid to facilitate endotracheal extubation 5. 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 6. 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 7. Anemia defined as a hemoglobin of < 9 mg/dL on weekly NICU labs 8. Concerns for pre-existing liver damage defined as an AST/ALT > 50 IU/L or direct bilirubin >1 mg/dL on weekly NICU labs 9. 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 10. Patients that are ventilated with a device not certified for blending of aerosolized solutions into the ventilator circuit 11. 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 12. Subjects that have experienced cardiac arrest with CPR for longer than 30 minutes

Gender Eligibility: All

Minimum Age: 29 Days

Maximum Age: 365 Days

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • University Hospitals Cleveland Medical Center
  • Provider of Information About this Clinical Study
    • Principal Investigator: Thomas Michael Raffay, MD, Sponsor-Investigator – University Hospitals Cleveland Medical Center
  • Overall Official(s)
    • Thomas Raffay, MD, Principal Investigator, University Hospitals Cleveland Medical Center

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