Inhaled Treatment for Bronchopulmonary Dysplasia

Bronchopulmonary Dysplasia Clinical Trial

Official title:

Pilot Study of an Inhaled Treatment for Bronchopulmonary Dysplasia

Clinical Trial Details — Status: Suspended

Administrative data

• NCT number: NCT04619602
• Other study ID #: BPD
• Status: Suspended
• Phase: Phase 1
• Start date: August 1, 2024
• Est. completion date: September 1, 2025

Study information

• Verified date: August 2023
• Source: University Hospitals Cleveland Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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].

Recruitment information / eligibility

• Status: Suspended
• Enrollment: 20
• Est. completion date: September 1, 2025
• Est. primary completion date: September 1, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 29 Days to 365 Days

Eligibility

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, ß-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

Related Conditions & MeSH terms

• Bronchopulmonary Dysplasia
• Hyperplasia

Intervention

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.

Locations

Country	Name	City	State
United States	University Hospitals Cleveland Medical Center Rainbow Babies and Children's Hospital	Cleveland	Ohio

Sponsors (1)

Lead Sponsor	Collaborator
University Hospitals Cleveland Medical Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Treatment Emergent Adverse Events	Occurrence of >grade 3 adverse events related to the treatment	7 days
Secondary	S-nitrosoglutathione change	Percent change in S-nitrosoglutathione pre/post treatment	30 minutes
Secondary	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	4 hours
Secondary	Change in GSNO catabolism pre/post treatment	Percent change in GSNO catabolism pre/post treatment	30 minutes
Secondary	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	4 hours
Secondary	Ventilator parameters post treatment	Change in ventilator parameters post treatment	4 hours