Clinical trial NCT04007120: Open-label Study of Inhaled RVT-1601 in Preterm Infants
Official title: Open-label Study to Assess the Safety, Tolerability, Feasibility, and Pharmacokinetics of Inhaled RVT-1601 in Preterm Infants at High-risk for Long-term Respiratory Morbidities
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Respiratory Morbidities of Prematurity (RMP)
Lead sponsor: Respivant Sciences Inc.
Sponsor class: INDUSTRY
Collaborators: name: Respivant Sciences Inc.
First posted: 2019-07-05
Enrollment: 8
Sex: ALL
Minimum age (years): 32
Maximum age (years): 35
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: OTHER

Brief summary: Preterm birth predisposes infants to greater risk for respiratory morbidities and the need for pulmonary care compared to term infants both in the short-term and long-term. In the short-term, preterm birth is a high risk factor for development of bronchopulmonary dysplasia (BPD), the second most common chronic pediatric respiratory disease after asthma. In the long-term, following discharge from the neonatal intensive care unit (NICU) and the hospital, preterm birth carries a high risk for respiratory morbidities (e.g., wheezing, cough, doctor visits, and hospitalizations for respiratory infections) and resource use, which in turn predisposes infants to the development of lung diseases in childhood and adulthood, including airway hyperresponsiveness, asthma, and chronic obstructive pulmonary disease (COPD). There is a significant unmet need for safe and efficacious approaches in the prevention and treatment of respiratory morbidities of prematurity.

The study will be conducted in the neonatal intensive care unit (NICU) in preterm infants to determine safety, tolerability and lung delivery performance of RVT-1601, a new inhalation formulation of cromolyn sodium delivered via the eFlow® Closed System (CS) nebulizer/face mask.


Eligibility criteria: Inclusion Criteria:

* Preterm infants between 32 weeks 0 days and 34 weeks 6 days of PMA
* Born between 24 weeks 0 days and 29 weeks 6 days of estimated GA
* Requiring minimal or no respiratory support (i.e., supplemental oxygen with \<2 liters per minute of nasal cannula flow acceptable)
* Body weight appropriate for gestational age
* Written informed consent obtained from at least one of the parents or legal guardians

Exclusion Criteria:

* Requiring invasive or noninvasive respiratory support (e.g., mechanical ventilation, CPAP)
* Clinically unstable (i.e. unable to maintain SpO2 between 90-95 % , escalating respiratory support in the past 24 hours)
* Major congenital anomaly (chromosomal, renal, cardiac, hepatic, neurologic or pulmonary malformations)
* Significant cardiac disorder (i.e., pulmonary hypertension)
* History of major surgical procedure
* Any condition that would preclude receiving study drug or performing any study-related procedures
* Participation in any other investigational drug study
* History of hypersensitivity or intolerance to cromolyn sodium

Primary outcomes:
measure: Change in heart rate
timeFrame: Pre-dose and 15 minutes post-dose
measure: Change in blood pressure
timeFrame: Pre-dose and 15 minutes post-dose
measure: Change in oxygenation
timeFrame: Pre-dose and 15 minutes post-dose
Secondary outcomes:
measure: Peak plasma concentration (Cmax)
timeFrame: 30 minutes post-dose
measure: Total urine excretion
timeFrame: 8 hours post-dose
Other outcomes:
Locations:
city: San Diego
country: United States
facility: Sharp Mary Birch Hospital for Women & Newborns
state: California
Study officials:
References: