Brief Title
Inhaled Treatment for Bronchopulmonary Dysplasia
Official Title
Pilot Study of an Inhaled Treatment for Bronchopulmonary Dysplasia
Brief Summary
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 Phase
Phase 1
Study Type
Interventional
Primary Outcome
Treatment Emergent Adverse Events
Secondary Outcome
S-nitrosoglutathione change
Condition
Bronchopulmonary Dysplasia
Intervention
GSNO
Study Arms / Comparison Groups
GSNO therapy
Description: 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.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
20
Start Date
July 1, 2022
Completion Date
October 1, 2024
Primary Completion Date
September 1, 2024
Eligibility Criteria
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
All
Ages
29 Days - 365 Days
Accepts Healthy Volunteers
No
Contacts
Thomas Raffay, MD, (216)844-3387, [email protected]
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT04619602
Organization ID
BPD
Responsible Party
Principal Investigator
Study Sponsor
University Hospitals Cleveland Medical Center
Study Sponsor
Thomas Raffay, MD, Principal Investigator, University Hospitals Cleveland Medical Center
Verification Date
December 2021